Category: FDA

It was the end of April — just as the U.S. confirmed its millionth covid-19 case and 50,000 deaths — when White House adviser Dr. Anthony Fauci announced “highly significant” news about a drug called remdesivir.

That was surprising because the antiviral drug, owned by Gilead Sciences and developed with investment from the federal government, had languished for years with no apparent commercial use. It had struck out as a treatment for hepatitis C and Ebola.

But early in 2020, when the first global cases of a new pneumonia-like viral illness emerged in China, Gilead resurfaced the compound, branded as Veklury, and shared it with scientists across the globe. From the Oval Office, Fauci, director of the National Institute of Allergy and Infectious Diseases, said remdesivir would be the “standard of care” for treating coronavirus disease.

Its emergency-use approval by the Food and Drug Administration immediately drew a storm of criticism. Clinical trials suggested it was only modestly helpful to covid patients. The World Health Organization recommended against its use. Politicians railed at its $3,120 price tag.

Yet, nine months later, it appears Fauci was right: As U.S. infections climb above 24 million, doctors with no other FDA-approved treatment options are now prescribing remdesivir to half the covid patients in U.S. hospitals.

“We want to shorten their hospital stay,” said Dr. Jade Le, an infectious diseases specialist with Access Physicians in Texas, where hospitals have been at capacity for weeks. Le prescribes remdesivir on average three to five times a day, always in concert with a low-cost steroid.

This month, Gilead chief executive Daniel O’Day raised the company’s profit estimates, noting that remdesivir alone would deliver about $2.8 billion in 2020 sales, bolstered by its use in U.S. and European markets. The company is proud of the role the treatment is playing during the pandemic, he said.

Still, controversies abound. Tension mounted last summer as shortages of remdesivir taxed the global medical community and raised urgent questions about how the federal government could step in. While doctors rationed the drug, politicians and advocates said U.S. taxpayers had invested enough in remdesivir’s development to merit a lower price.

“The remdesivir story is actually a story that is all too common,” said Zain Rizvi, law and policy researcher at the consumer advocacy group Public Citizen. Rizvi — and other advocates — say the drug would not have come to market if it wasn’t for federal grants and the help of U.S. scientists.

They said the rags-to-riches story of remdesivir, a losing prospect turned blockbuster, holds lessons in how Americans end up paying more for medicine than anyone else in the world. Remdesivir used taxpayer dollars to be developed but, in a public health emergency, patients have no option but to pay whatever the pharmaceutical giant demands.

Rizvi has estimated that federal grants of “at least” $70 million supported the scientific discovery of remdesivir. He pointed to early research done, including a collaboration between Gilead and university scientists, using federal grants, to test remdesivir’s antiviral compounds against coronaviruses, such as MERS and SARS. Others figure the investment could be much higher.

Kathryn Ardizzone, legal counsel for the patent rights group Knowledge Ecology International, is among those, though she agreed the amount is at least $70 million and declined to estimate the total amount of federal dollars used for remdesivir’s discovery. There is no publicly available database of how much the government has spent to develop any drug, including remdesivir.

The NIH said in an emailed statement that it “did not develop remdesivir.” The agency confirmed it funded research on the drug’s uses as well as providing money to institutions that worked with Gilead to develop the drug.

KEI filed a Freedom of Information Act request to view the grants and clinical trials related to remdesivir. In October, the group sued the National Institutes of Health because it had failed to respond. Ardizzone said she expects the records will counter Gilead’s justification of remdesivir’s price: “When the government has played such a critical role at every step of the way, that argument falls apart.”

Gilead, in an emailed statement, said its investment in the drug predates any government involvement and “disagrees with the premise that the government has any rights to Gilead’s remdesivir intellectual property.” No federal scientists are named on remdesivir’s patents.

The company has defended the price of the drug, saying it invested more than $1 billion in 2020 to support clinical trials as well as to manufacture and distribute remdesivir. Gilead priced it at $3,120 for a five-day course of treatment in the U.S. market, and $2,340 for other developed countries. Some smaller U.S. government entities, like the Indian Health Service, pay the lower price as well. Noting the price for developed countries, Gilead spokesperson Arran Attridge said the drug is priced “significantly below the potential value” it delivers.

But U.S. Sen. Ron Wyden, a top-ranking Democrat from Oregon on the Senate Finance Committee, said he has been concerned about Gilead’s price for a five-day course of treatment since it was announced. “My previous investigative work … has shown Gilead’s willingness to put profits over patients,” Wyden said. Wyden, along with Sen. Chuck Grassley (R-Iowa), released an investigation in 2015 of Gilead’s hepatitis C drug prices and marketing.

At its core, remdesivir is a simple, “small molecule” drug. Generics manufacturers in India have copied the design and priced it at $53.34 per vial, or $320 for a course of treatment.

The U.S. government, so far, has not exerted its intellectual property rights. But there are signals that could change.

In late summer, California Attorney General Xavier Becerra led a group of more than 30 attorneys general calling for the federal government to license remdesivir to another manufacturer, such as a lower-cost provider from overseas. President Joe Biden nominated Becerra to lead the Department of Health and Human Services.

Another Biden pick, Dr. Rochelle Walensky, an infectious diseases specialist who has been tasked to lead the Centers for Disease Control and Prevention, co-authored a New York Times opinion piece suggesting that HHS could buy the drug from another company (including a generics maker overseas) and pay royalties to Gilead. This fall, U.S. Rep. Lloyd Doggett (D-Texas) held a news conference supporting the approach Walensky and others proposed.

Neither maneuver, however, was meant as a lever to lower its price for patients, said Adam Mossoff, a law professor at George Mason University. Mossoff doubted that either of the strategies would hold up in court and noted there are plenty of congressional proposals to lower drug prices and sometimes “people get ahead of themselves.” They try to use the law to advocate for what should be a policy goal, he said.

Underlying Becerra’s and Walensky’s proposals is the deeper, nagging question of whether Gilead should fully own the rights to remdesivir if the U.S. funded research and its scientists worked with Gilead to discover the drug.

Doggett put it this way: “Gilead is overcharging on a drug that was saved from the scrap heap of failed drugs only because of taxpayer-funded research.”

Sen. Debbie Stabenow (D-Mich.) and Rep. Carolyn Maloney (D-N.Y.) have asked the government’s federal watchdog agency, the Government Accountability Office, to investigate “what legal rights do federal agencies have” in relation to remdesivir. Gilead said it is cooperating with the investigation; government officials expect the review to be completed this spring.

Gilead, in an emailed statement in response to a question about the GAO review, called the government’s involvement “limited.” In response to questions about the patents, Gilead said its own investments predate any government involvement and its inventors identified the drug’s antiviral activity, optimized the formula and scaled up the manufacturing process.

Gilead confirmed it has eight listed patents on remdesivir, with the last expiring in 2038.

In the abstracts of two patents, filed in 2014 and 2015, the CDC and USAMRIID, the U.S. Army Medical Research Institute of Infectious Diseases, are mentioned as places studies were conducted. Each patent emphasizes its focus on treating the Ebola virus and other filoviruses that cause fatal hemorrhagic fevers.

Christopher Morten, deputy director of technology law and policy clinic at New York University School of Law, said those two patents should list government scientists as co-inventors. Referring to one patent (No. 9,724,360), Morten said its earliest filing was October 2014. “Which makes perfect sense, because the U.S. government collaboration started in 2013 with Ebola and the CDC,” Morten said. While not attaching specific government investment dollars to its descriptions, Gilead confirms the collaboration, as well, saying it “worked with the U.S. government to confirm remdesivir’s preclinical activity against Ebola.”

Morten, who previously represented pharmaceutical companies as a patent lawyer, said he believes the question of inventorship should be raised in court, using the legal tactic proposed by CDC chief Walenksy. Morten co-authored a white paper on remdesivir with the HIV advocacy group PrEP4All.

Notably, PrEP4All also challenged Gilead’s patents for the HIV drug Truvada. In 2019, the U.S. Departments of Justice and Health and Human Services sued Gilead over patent rights for Truvada. The case is ongoing.

Back on the pandemic’s front lines, U.S. doctors administer remdesivir even after the World Health Organization recommended against the drug in November. The WHO’s recommendation, which referenced results from an international trial called Solidarity, found remdesivir did not improve a covid patient’s chance of survival.

The Food and Drug Administration gave remdesivir full regulatory approval in October, making it the only approved U.S. treatment for the deadly disease.

Dr. Rajesh Gandhi, a member of the Infectious Diseases Society of America’s panel on covid-19 treatment guidelines, pointed to another clinical trial of more than 1,000 hospitalized patients run by the National Institutes of Health. The trial, called ACTT-1, showed hospital stays of about 10 days for those who received remdesivir compared with 15 days for those who did not, he said.

“Many of us believe remdesivir has a role,” Gandhi said, “though we wish it had a greater effect.” He noted that the trial showed one group of patients saw the biggest benefit: those who need supplemental oxygen but are not yet on a ventilator. It’s clear, he said, “we need better drugs than remdesivir.”

Remdesivir — along with a steroid — was enough for Shirley Lewis.

The 69-year-old Florida resident said she was sick for about a week when she found herself unable to breathe and went to the hospital.

“I’m telling you, I was like half-dead and half-alive … all I could do was pray,” Lewis said. Doctors put her on supplemental oxygen and began IV bags of remdesivir paired with steroids. Lewis said she felt the difference right away.

“I said, Oh, thank God,” Lewis recalled about a month after being released from the hospital. “I thought it was some kind of miracle, I really did. So don’t let them say it doesn’t work, because it did.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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A new report from the U.S. Food and Drug Administration (FDA) notes that the Center for Drug Evaluation and Research (CDER) approved 53 novel drugs last year. These figures are in addition to innovative medicines that are approved through FDA’s Center for Biologics Evaluation and Research—which include for example, cell therapies, gene therapies, vaccines and other biologic medicines.

Shots:

• The approval is based on the pivotal AURORA P-III study and AURA-LV P-II study involves assessing of Lupkynis + SoC in 533 patients to treat adult patients with LN
• The study demonstrated significantly improved renal response rates vs SoC, improved response rates in all parameters across immunologically-active classes, 50 % reduction in UPCR twice as fast as SoC, complete renal response @24 wks vs SoC @1year
• Lupkynis is the 1^st FDA-approved oral therapy for LN and is now commercially available in the US

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Aurinia Pharmaceuticals

The post Aurinia Lupkynis (voclosporin) Receives US FDA’s Approval to Treat Adult Patients with Active Lupus Nephritis first appeared on PharmaShots.

Shots:

• The approval is based on results from CheckMate -9ER, P-III pivotal trial involves assessing of Cabometyx + Opdivo vs sunitinib in 651 patients previously untreated advanced or metastatic RCC.
• Results: @ median follow-up of 18.1 mos. patients treated with the combination achieved PFS of 16.6 mos vs 8.3 mos for Sutent arm. Opdivo + Cabometyx also significantly reduced the risk of death by 40% vs Sutent alone , ORR 56% in combination and 27% sunitinib
• Application approved prior to PDUFA action date of Feb 20, 2021 and reviewed under the Real-Time Oncology Review pilot program

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Fierce Pharma

The post Exelixis Cabometyx (cabozantinib) + Opdivo (nivolumab) Receives US FDA’s Approval as a 1L Treatment for Patients with Advanced Renal Cell Carcinoma first appeared on PharmaShots.

President Joe Biden looks likely to ditch the previous administration’s move to create a quicker route to market for digital health products and other devices following the pandemic emergency.

After he took office on Wednesday, Biden quickly moved to freeze a raft of Trump policies, including the moves to loosen regulations on devices including digital health products.

Only last week, the FDA had posted plans with the US Department of Health and Human Services (HHS) to allow certain medical devices including “low risk” digital health apps to be exempt from its usual premarket notification requirements.

The regulatory process known as 510(k) had been temporarily waived during the COVID-19 emergency and the proposal would have made the move permanent.

It was part of a plan to create a fast route to market for products that have helped to support the US health system during the COVID-19 crisis.

The 510(k) process already gave considerable regulatory leeway for new products, allowing the FDA to wave products through to market that it found to be “substantially equivalent” to another legally marketed predecessor.

But it looks like the Biden administration is taking a more long-term view about the regulations and how they apply to digital health.

Legal experts from the law firm Hogan Lovells noted that the proposals to drop the process “fly in the face” of an action plan to regulate artificial intelligence (AI) in healthcare.

That called for an expanded regulatory framework for machine learning devices and a more careful review of products that rely on AI.

The team from Hogan Lovells said that the exemptions remain in place for seven of the devices, all of which are gloves.

But the proposal will not take effect regarding 83 other devices unless the Biden administration decides to pursue it.

Filings for the kinds of devices covered by the notice are still being submitted and accepted by the FDA and meanwhile the HHS is still accepting comments on ways to improve the premarket approval process known until 16 March.

The post Biden likely to ditch Trump’s plans to relax digital health regulations appeared first on .

Shots:

• The approval is based on pivotal phase III ATLAS and FLAIR study assessing Cabenuva in 1,100+ HIV-1 adults to replace the current ARV regimen in those who were virologically suppressed (HIV-1 RNA less than 50 copies/mL) on a stable regimen
• Prior to initiating treatment of Cabenuva, oral dosing of cabotegravir and rilpivirine should be administered for ~1mos. to assess the tolerability of each therapy. The therapy reduces the treatment dosing days from 365 days to 12days/ yr
• The company will begin shipping of Cabenuva to wholesalers and specialty distributors in the US in Feb’2021

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: ViiV Healthcare

The post ViiV’s Cabenuva (cabotegravir and rilpivirine) Receives the US FDA’s Approval as the First and Only Complete Long-Acting Regimen for HIV treatment first appeared on PharmaShots.

Shots:

• The approval is based on SUSTAIN FORTE trial assessing Ozempic (2.0mg, qw) vs Ozempic (1.0mg) in 961 people with T2D in need of treatment intensification
• Result: 2.0 mg dose achieved significant and superior reduction in HbA1c, both doses are safe and well-tolerated profiles
• Ozempic is a glucagon-like peptide-1 (GLP-1) analogue and is currently approved in the US for 0.5 mg and 1.0 mg doses to treat T2D in adults and to reduce the risk of MACE in adults with T2D mellitus and established CV disease

Click here ­to­ read full press release/ article | Ref: GlobeNewswire| Image: Business Medical Dialogues

The post Novo Nordisk Report Submission of label Expansion Application to US FDA’s for Ozempic (semaglutide) for T2D first appeared on PharmaShots.

The agency published its first action plan last week for how it plans to regulate machine learning-based software as a medical device. To start, the FDA said it will issue guidance on how changes to algorithms should be regulated as they “learn.”

Bayer and Merck & Co’s heart failure drug vericiguat has been approved by the FDA under the brand name Verquvo, in an increasingly competitive market.

Verquvo has been approved to reduce risk of cardiovascular death and heart failure hospitalisation in adults with symptomatic chronic heart failure and ejection fraction less than 45%.

The drug can be used after hospitalisation for heart failure or in patients in need of intravenous diuretics.

But the drug is entering an increasingly competitive market, where Novartis and AstraZeneca are vying for supremacy.

Entresto (sacubitril+valsartan) was FDA-approved in patients with reduced ejection fraction five years ago.

Meanwhile, AstraZeneca’s Farxiga (dapagliflozin), originally a diabetes drug, was approved in the US last year in heart failure with reduced ejection fraction.

The FDA is also quickly reviewing Boehringer Ingelheim’s Jardiance (empagliflozin) in heart failure, AZ’s big rival in the market for SGLT2 inhibitor class drugs.

Verquvo’s approval is the first for patients following a hospitalisation for heart failure or who need for outpatient IV diuretics and is based on the results of the pivotal phase 3 VICTORIA trial and follows a priority regulatory review.

VICTORIA’s main efficacy goal was to determine whether Verquvo is superior to placebo, both in combination with other heart failure therapies, in reducing the risk of cardiovascular death or heart failure hospitalisation in adults with symptomatic chronic heart failure and ejection fraction less than 45% following a worsening heart failure event.

Verquvo met the primary efficacy objective, with the study showing there was a 4.2% reduction in annualised absolute risk in the treatment group compared with placebo.

Therefore, 24 patients would need to be treated over an average of one year to prevent one death or heart failure hospitalisation.

VICTORIA is a large phase 3 trial involving 5,050 patients with heart failure with ejection fraction less than 45%.

Dr Paul W. Armstrong, cardiologist and Distinguished University Professor of Medicine at the Canadian VIGOUR Centre, University of Alberta, and study chair of the VICTORIA trial, said: “Patients with symptomatic chronic heart failure and reduced ejection fraction have a high risk for hospitalisation after experiencing symptoms of heart failure requiring outpatient IV diuretic treatment or hospitalisation.

“By some estimates, more than half of these patients are rehospitalised within a month of discharge due to a worsening event and approximately one in five die within two years.

“The approval of Verquvo provides doctors, health care professionals, and patients with a welcome new option to current available therapies.”

The post FDA approves Bayer/Merck & Co heart failure drug appeared first on .

Shots:

• The sNDA submission is based on a P-II study that involves assessing Esbriet vs PBO in patients aged ≥18-85yrs. with progressive fibrosing UILD for 24wks. The anticipated PDUFA date is May’2021
• Results: Over 24wks. predicted median change in FVC measured by home spirometry (-87.7 vs -157.1 mL); change in percent predicted DLco and 6MWD are in favor of Esbriet. Additionally, less loss to lung function and exercise capacity was observed
• Esbriet is an oral therapy for IPF and is available in more than 60+ countries globally. FDA has granted ODD and BTD to the therapy in 2020

Click here ­to­ read full press release/ article | Ref: Roche| Image: The Indian Express

The post Roche Reports the US FDA’s Acceptance of sNDA and Granted Priority Review for Esbriet (pirfenidone) to Treat UILD first appeared on PharmaShots.

Agency veteran Dr Janet Woodcock is the new interim FDA commissioner appointed by president Joe Biden to replace outgoing Trump appointee Stephen Hahn.

Woodcock has most recently been working with the Operation Warp Speed coronavirus vaccine and drug project started by the Trump administration.

While this work will continue, the Operation Warp Speed name has been dropped and Woodcock will take a leading role at the agency she first joined in 1984.

Dr Janet Woodcock

Holding a Bachelor of Science in chemistry from Bucknell University and a Doctor of Medicine at Northwestern University Medical School, Woodcock first served as director of the division, covering new drugs at the FDA’s Center for Biologics Evaluation and Research (CBER).

She held several other roles at CBER before being named as director of the FDA’s Center for Drug Evaluation and Research in 1994 and staying in that role until 2005.

Between 2005 and 2008 she had several other roles at the FDA commissioner’s office, including deputy commissioner, chief medical officer and chief operating officer, before returning as head of CDER in 2008.

According to press reports, the Biden administration has not yet nominated a permanent commissioner.

In a tweet, Woodcock said that she will continue to recuse herself from work relating to the therapeutics developed by Operation Warp Speed, although this does not apply to vaccines.

She added that Julia Tierney, a 12-year veteran of the agency, has agreed to serve as acting chief of staff.

 

According to the New York Times, advisers to the Biden administration’s transition team, Woodcock is one of the candidates under consideration for the permanent position.

Insiders said that other candidates under review are principal deputy commissioner Dr Amy Abernethy and former agency official Dr Joshua Sharfstein, who is vice dean for public health practice and community engagement at Johns Hopkins University.

Hahn’s resignation is a formality as senior political appointees are expected to leave their roles when a new administration takes over.

In a farewell note to FDA staff, Hahn praised the organisation’s employees for their response to the coronavirus crisis.

He noted the scientific advances that have been achieved since the pandemic began, such as the authorisation of the first non-prescription over-the-counter coronavirus test, authorisation of the first antiviral agent and the first two FDA authorised COVID-19 vaccines.

But Hahn’s tenure was marked by miss-steps as the FDA came under political pressure from the previous administration, such as the Emergency Use Authorization for the drug hydroxychloroquine despite a lack of scientific evidence.

The post Biden appoints veteran Woodcock as interim FDA commissioner appeared first on .

Shots:

• The approval is based on pivotal P-III VICTORIA trial involves assessing of Verquvo (2.5mg, 5mg & 10mg) vs PBO in 5,050 adult patients with symptomatic CHF and LVEF less than 45%, following a worsening HF event
• The study met the primary efficacy objective based on a time-to-event analysis & showed a 4.2% reduction in annualized absolute risk. The 1EPs is time to the first event of CV death or hospitalization for HF @median follow-up of 11 mos.
• Verquvo is the first soluble guanylate cyclase stimulator, approved to treat HF

Click here ­to­ read full press release/ article | Ref: Business Wire| Image: Financial Times

The post Bayer and Merck’s Verquvo (vericiguat) Receives the US FDA’s Approval to Treat Chronic Heart Failure first appeared on PharmaShots.

What You Should Know:

– Tyto Care announces the launch of its fingertip Pulse
Oximeter device, allowing users to measure their own blood oxygen saturation
level and heart rate from the comfort and safety of home.

– Expanding its clinic-quality remote capabilities, TytoCare users will be able to perform a remote blood oxygen exam, allowing clinicians to better monitor patients with COVID-19 and chronic lung or heart conditions.

---

 Tyto Care, a New York City-based all-in-one modular device and examination platform for AI-powered, on-demand, remote medical exams, today announced the release of its FDA-cleared fingertip Pulse Oximeter (SpO2) medical device. The Pulse Oximeter enables TytoCare users to check blood oxygen saturation levels and heart rate, which are crucial for the monitoring of chronic conditions and COVID-19.

Fingertip At-Home Pulse Oximeter Measures Blood Oxygen and Heart Rate

The Pulse Oximeter connects by cable to the TytoCare device.
Users who choose to perform the blood oxygen exam are prompted to place their
finger in the SpO2 device for 15 seconds. Upon completion of the
exam, the blood oxygen and heart rate results appear immediately on the
handheld TytoCare device and the Pulse Oximeter adaptor itself and are also
recorded within the TytoCare platform for review by the clinician and sent to
the patients’ EHR
(Electronic Health Record). If the examination is performed in real-time
during a live telehealth visit, the clinician will see the results immediately
in the TytoCare Clinician Dashboard. Clinicians are able to assess the
reliability of the results recorded by viewing the heart rhythm graph generated
during the exam.     

Why It Matters

This expansion of Tyto Care’s virtual examination solution
is bringing the company even closer to full remote, clinic-level testing
capabilities from the comfort of home. The Pulse Oximeter is a key tool for
monitoring high-risk or infected individuals during the COVID-19 pandemic, as
well as for post-discharge care and home hospitalization. The company’s Pulse
Oximeter device also enables seamless monitoring of patients with chronic lung
and heart conditions, critical during routine times as well.

“We’re excited to announce the release of our Pulse Oximeter, providing patients with more tests that bring the clinic directly to them,” said Dedi Gilad, CEO and Co-Founder of Tyto Care. “The COVID-19 pandemic thrust telehealth into the spotlight, and we are constantly enhancing the TytoCare platform to ensure users have access to the most comprehensive telehealth solution available. The pandemic will eventually be behind us, yet telehealth will remain a key component in the future of healthcare, providing patients with the best possible remote care and clinicians with actionable insights into their patients’ health.”

Availability

The SpO₂ device is available in the
United States and Israel through partnering health systems. In the future, the
device will also be available in Europe, South Africa, and Asia.

Shots:

• The US FDA has accepted the BLA and granted the PR with an anticipated PDUFA date as Jul 17, 2021
• The BLA filing marks the milestone on the path to commercialization of narsoplimab
• Narsoplimab is an investigational mAb targeting MASP-2 has received the US FDA’s BTD and ODD to the FDA for each of HSCT-TMA and IgA nephropathy

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Omeros

The post Omeros Reports the US FDA’s Acceptance and Priority Review of BLA for Narsoplimab (OMS721) to Treat HSCT-TMA first appeared on PharmaShots.

Shots:

• The approval is based on P-III ANDROMEDA (AMY3001) study involves assessing of Darzalex Faspro + bortezomib, cyclophosphamide, and dexamethasone (VCd) vs VCd alone in 388 patients with newly diagnosed AL amyloidosis
• Genmab to receive $30M as milestones with the first commercial sale of Darzales faspro in this indication. The US FDA reviewed the submission of data for approval in this indication under RTOR pilot program and Project Orbis
• Darzalex faspro is the SC formulation of daratumumab and is the only therapy for newly diagnosed Light-chain (AL) amyloidosis in the US

Click here ­to­ read full press release/ article | Ref: GlobeNewswire | Image: Medwatch

The post Genmab’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) Receives the US FDA’s Approval for Patients with Newly Diagnosed Light-chain (AL) Amyloidosis first appeared on PharmaShots.

Shots:

• The approval is based on pivotal P-II DESTINY-Gastric01 trial involves assessing of Enhertu (6.4 mg/kg, q3w) vs CT in a ratio (2:1) in adult patients with LA or metastatic HER2 positive gastric or GEJ adenocarcinoma who have received a prior trastuzumab-based regimen
• In a pre-specified interim analysis, it showed 41% reduction in the risk of death vs patients with mOS (12.5 vs 8.4mos); PFS (5.6 vs 3.5mos); ORR (40.5% vs 11.3%); CR (7.9% vs 0%); PR (32.5% vs 11.3%); DoR (11.3 vs 3.9mos.)
• Enhertu is a HER2-directed ADC and has received the US FDA’s PR & BTD for HER2+ m-gastric cancer and ODD for gastric cancer

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: BioSpace

The post Daiichi Sankyo and AstraZeneca’s Enhertu Receive the US FDA’s Approval for Previously Treated HER2-Positive Advanced Gastric Cancer first appeared on PharmaShots.

Developers of digital health tools have benefited from a truncated route to market during the COVID-19 crisis, and that could be made permanent if new FDA guidance is adopted.

Pandemic emergency provisions were put in place in March last year to allow certain medical devices to be launched for use by patients without having to go through the FDA’s usual premarket notification requirements – also known as the 510(k) process.

That included some low-risk (Class IIa) digital health apps under the software as a medical device (SaMD) category, which can be used to minimise direct contact between patients and healthcare professionals at a time when non-essential visits are best avoided.

Now, the FDA is seeking comment on plans to make that emergency regulatory flexibility permanent, by doing away with the premarket review requirements altogether  “for some or all of [these] devices”, according to a Federal Register notice (0991–ZA52).

The document is likely to be one of the last signed by Health and Human Services (HHS) Secretary Alex Azar, who has resigned from the role as preparations continue for the start of the new administration under President-elect Joe Biden on Wednesday.

Last April, the FDA temporary waiver covered digital health tools used specifically for psychiatric disorders, along with low-risk general wellness and digital health products for mental health or psychiatric conditions. The discretion also covered digital pathology tools and other medical devices like thermometers and ventilators.

Pandemic lockdowns mean patients with mental illness are less able to maintain contact with healthcare and support workers, placing them under added strain, and when it announced the measure the FDA said remote tools could help them cope until some degree of normal life can be restored.

The waiver meant that a number of apps were launched earlier than expected in the US, including Pear Therapeutics’ smartphone-based Pear-004 for people living with schizophrenia and Akili Therapeutics’ attention deficit hyperactivity disorder (ADHD) app Endeavor (AKL-T01), as well as computerised cognitive assessment aids.

The experience so far seems to have been a success, given the FDA is considering making the exemption permanent for 91 devices and SaMD tools out of 221 Class I and Class II medical device types covered by the waiver categories.

“The exemptions provided for and proposed under this notice for these 91 device classes could eliminate anywhere from $9.1 to $364 million in startup costs if there were one new entrant into each device market,” says the guidance.

“At the same time, should these waivers go into effect as proposed, patients stand to gain more immediate access to new products that would otherwise be required to obtain a 510(k) clearance prior to marketing.”

The proposal would also spare FDA’s “scarce review resources” as a result of the pandemic and allow it to redeploy these elsewhere, including devices intended for use in managing CPOVID-19 patients.

The post US eyes permanent change to digital health regulatory path appeared first on .

Shots:

• The US FDA has granted BTD for Ligelizumab for the treatment of CSU in patients with an inadequate response to H1-antihistamine treatment
• The therapy is currently being evaluated in ongoing P-III program including PEARL 1 & -2 that assess Ligelizumab vs Xolair (omalizumab) in ~2000 patients across the globe with its anticipated results in H2’21
• Ligelizumab is a next generation monoclonal anti-IgE Ab, that demonstrated more patients experienced complete resolution of wheals (hives) in a P-IIb dose-finding trial. The company is anticipating the US regulatory submission in 2022

Click here ­to­ read full press release/ article | Ref: Novartis | Image: Medical, Marketing and Media

The post Novartis’ Ligelizumab (QGE031) Receives the US FDA’s Breakthrough Designation for Patients with Chronic Spontaneous Urticaria first appeared on PharmaShots.

Shots:

• The approval is based on the ADVL0912 study assessing Xalkori in 121 patients aged 1-21yrs. that included 26 patients with r/r, systemic ALK+ ALCL prior treated with at least one systemic treatment
• The study showed 88% ORR. Among 23 patients, who achieved a response, 39% maintained their response for at least 6mos. and 22% maintained their response for at least 12mos.
• Xalkori is a TKI and has received the FDA’s BTD for ALK+ ALCL in May’2018. Additionally, EMA has agreed to a PIP for the therapy to treat pediatric patients with r/r systemic ALK+ ALCL

Click here ­to­ read full press release/ article | Ref: Pfizer | Image: The Bangkok Post

The post Pfizer’s Xalkori (crizotinib) Receives the US FDA’s Approval for ALK-Positive Anaplastic Large Cell Lymphoma In Children And Young Adult first appeared on PharmaShots.

The agency has finalized a rule that allows it to provide immediate Medicare coverage for FDA-approved products that are deemed “breakthrough devices.” The new coverage process would enable seniors to get access to these devices more quickly, but some provider and payer groups are concerned that this could cause patient harm.

AI tools increasingly occupy a regulatory gray area in healthcare. For clinicians to assess whether they are trustworthy, they need transparency on how they work, said panelists at CES.

What are some strategies to reduce arsenic exposure from rice?

Those who are exposed to the most arsenic in rice are those who are exposed to the most rice, like people who are eating plant-based, gluten-free, or dairy-free. So, at-risk populations are not just infants and pregnant women, but also those who may tend to eat more rice. What “a terrible irony for the health conscious” who are trying to avoid dairy and eat lots of whole foods and brown rice—so much so they may not only suffer some theoretical increased lifetime cancer risk, but they may actually suffer arsenic poisoning. For example, a 39-year-old woman had celiac disease, so she had to avoid wheat, barley, and rye, but she turned to so much rice that she ended up with sky-high arsenic levels and some typical symptoms, including “diarrhea, headache, insomnia, loss of appetite, abnormal taste, and impaired short-term memory and concentration.” As I discuss in my video How Much Arsenic in Rice Is Too Much, we, as doctors, should keep an eye out for signs of arsenic exposure in those who eat lots of rice day in and day out.

As you can see at 1:08 in my video, in its 2012 arsenic-in-rice exposé, Consumer Reports recommended adults eat no more than an average of two servings of rice a week or three servings a week of rice cereal or rice pasta. In its later analysis, however, it looked like “rice cereal and rice pasta can have much more inorganic arsenic—a carcinogen—than [its] 2012 data showed,” so Consumer Reports dropped its recommendation down to from three weekly servings to a maximum of only two, and that’s only if you’re not getting arsenic from other rice sources. As you can see from 1:29 in my video, Consumer Reports came up with a point system so people could add up all their rice products for the week to make sure they’re staying under seven points a week on average. So, if your only source of rice is just rice, for example, then it recommends no more than one or two servings for the whole week. I recommend 21 servings of whole grains a week in my Daily Dozen, though, so what to do? Get to know sorghum, quinoa, buckwheat, millet, oatmeal, barley, or any of the other dozen or so common non-rice whole grains out there. They tend to have negligible levels of toxic arsenic.

Rice accumulates ten times more arsenic than other grains, which helps explain why the arsenic levels in urine samples of those who eat rice tend to consistently be higher than those who do not eat rice, as you can see at 2:18 in my video. The FDA recently tested a few dozen quinoa samples, and most had arsenic levels below the level of detection, or just trace amounts, including the red quinoas that are my family’s favorite, which I was happy about. There were, however, still a few that were up around half that of rice. But, overall, quinoa averaged ten times less toxic arsenic than rice. So, instead of two servings a week, following the Consumer Reports recommendation, you could have 20. You can see the chart detailing the quinoa samples and their arsenic levels at 2:20 in my video.

So, diversifying the diet is the number-one strategy to reduce exposure of arsenic in rice. We can also consider alternatives to rice, especially for infants, and minimize our exposure by cooking rice like pasta with plenty of extra water. We found that a 10:1 water-to-rice ratio seemed best, though the data suggest the rinsing doesn’t seem to do much. We can also avoid processed foods sweetened with brown rice syrup. Is there anything else we can do at the dining room table while waiting for federal agencies to establish some regulatory limits?

What if you eat a lot of fiber-containing foods with your rice? Might that help bind some of the arsenic? Apparently not. In one study, the presence of fat did seem to have an effect, but in the wrong direction: Fat increased estimates of arsenic absorption, likely due to the extra bile we release when we eat fatty foods.

We know that the tannic acid in coffee and especially in tea can reduce iron absorption, which is why I recommend not drinking tea with meals, but might it also decrease arsenic absorption? Yes, by perhaps 40 percent or more, so the researchers suggested tannic acid might help, but they used mega doses—17 cups of tea worth or that found in 34 cups of coffee—so it isn’t really practical.

What do the experts suggest? Well, arsenic levels are lower in rice from certain regions, like California and parts of India, so why not blend that with some of the higher arsenic rice to even things out for everybody?

What?!

Another wonky, thinking-outside-the-rice-box idea involves an algae discovered in the hot springs of Yellowstone National Park with an enzyme that can volatize arsenic into a gas. Aha! Researchers genetically engineered that gene into a rice plant and were able to get a little arsenic gas off of it, but the rice industry is hesitant. “Posed with a choice between [genetically engineered] rice and rice with arsenic in it, consumers may decide they just aren’t going to eat any rice” at all.

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This is the corresponding article to the 11th in a 13-video series on arsenic in the food supply. If you missed any of the first ten videos, watch them here:

• Where Does the Arsenic in Chicken Come From?
• Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?
• The Effects of Too Much Arsenic in the Diet
• Cancer Risk from Arsenic in Rice and Seaweed
• Which Rice Has Less Arsenic: Black, Brown, Red, White, or Wild?
• Which Brands and Sources of Rice Have the Least Arsenic?
• How to Cook Rice to Lower Arsenic Levels
• Arsenic in Infant Rice Cereal
• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup
• How Risky Is the Arsenic in Rice?

You may also be interested in Benefits of Turmeric for Arsenic Exposure.

Only two major questions remain: Should we moderate our intake of white rice or should we minimize it? And, are there unique benefits to brown rice that would justify keeping it in our diet despite the arsenic content? I cover these issues in the final two videos: Is White Rice a Yellow-Light or Red-Light Food? and Do the Pros of Brown Rice Outweigh the Cons of Arsenic?.

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

Boehringer and Eli Lilly have moved closer to a heart failure indication for their SGLT2 inhibitor Jardiance, as the FDA starts a fast-track review of the drug in its first use beyond diabetes.

The US regulator is looking at data from the EMPEROR-Reduced trial of Jardiance (empagliflozin), which found that the drug achieved a 25% reduction in the combined primary endpoint of cardiovascular death or hospitalisation compared to placebo.

Lilly and Boehringer claim almost 60% market share for Jardiance among SGLT2 drugs used to treat type 2 diabetes, driving blockbuster sales for the brand.

It was the top-selling drug in the SGLT2 inhibitor class in 2019, with sales of almost $3 billion, helped by side-effect problems that have afflicted first-to-market rival Invokana (canagliflozin) from Johnson & Johnson.

However its rivals – which also include AstraZeneca’s Farxiga (dapagliflozin)  – have moved more swiftly into areas like heart failure and chronic kidney disease (CKD) which have made large numbers of new patients eligible for treatment with the class.

The new FDA review – covering Jardiance as a treatment for adults with heart failure with reduced ejection fraction (HFrEF) in patients with and without diabetes – is Lilly and Boehringer’s first chance to fight back.

Farxiga won FDA approval for adults with HFrEF in May 2020, which helped to drive its third-quarter sales up by a third to $525 million. AZ picked up EU approval for the same indication the following November.

Lilly and Boehringer will be hoping for a swift FDA review so that Jardiance will not fall too far behind its competitor in the heart failure category, and that looks likely as the benefit in HFrEF increasingly appears to be an SGLT2 class effect.

GlobalData has said that heart failure could add billions to the sales of the two SGLT2 inhibitors, particularly if they also claim approvals in heart failure with preserved ejection fraction (HFpEF), a larger patient population.

It says Farxiga will reach peak sales of $9 billion in 2028, with Jardiance forecast to reach $4.6 billion, assuming a launch for HFrEF this year. The bulk of those sales will come from HFpEF, however,  as in this form of heart failure there is a huge unmet need and no approved therapies.

Boehringer and Lilly are waiting for the results of the EMPEROR-Preserved later in 2021, while AZ should also Farxiga in the DELIVER trial in HFpEF, with additional data on both HFrEF and HFpEF due from the DETERMINE study, before year-end.

Meanwhile, EMPEROR-Reduced also showed a slowdown in the rate of decline in kidney function among patients with HFrEF, an effect that Lilly and Boehringer are exploring in the CKD patient population in the EMPA-KIDNEY trial due to generate results in 2022.

Invokana was the first mover among the SGLT2 drugs in the kidney area, winning FDA approval towards the end of 2019 for diabetic kidney disease. After a couple of years of declining sales due to concern about a risk of lower limb amputation, Invokana managed a 25% gain to $224 million in third-quarter 2020.

Farxiga meanwhile has already been filed for CKD on the back of the DAPA-CKD trial, with a verdict due in the second quarter.

The post Boehringer, Lilly’s Jardiance ties to match AZ’s Farxiga with heart failure label appeared first on .

The new tests can open a window into the duration of immunity and to the antibody levels needed to sustain it. But first, a universal standard is needed to ensure results are comparable between tests.

Getting rice down to the so-called safe water limit for arsenic would still allow for roughly 500 times greater cancer risk than is normally considered acceptable. Given the level of arsenic in rice, how could we figure out how much rice is too much? There are no U.S. standards for arsenic in rice, even though “food sources are the main source of exposure.” There are limits on arsenic in apple juice and tap water, though. To calculate those, experts must have sat down, determined out how much arsenic a day was too much—too risky—then figured people typically drink about four to eight cups of water a day, and set the limits that way, right? Okay, well, can’t we just use their how-much-arsenic-a-day-is-too-much-arsenic-a-day number, and, based on the average arsenic content in rice, figure out how-much-rice-a-day-is-too-much-rice? I discuss this in my video How Risky Is the Arsenic in Rice?.

“The allowable level established by the FDA for arsenic in bottled water is 10 ppb,” assuming people might drink a liter a day. So, based on that daily 10 ppb limit, how much rice is that?

“Each 1 g increase in rice intake was associated with a 1% increase in urinary total arsenic, such that eating 0.56 cups [a little over a half cup] of cooked rice was considered comparable with drinking 1 L/d,” one liter per day, of that maximally contaminated water. Well, if you can eat a half cup a day, why does Consumer Reports suggest eating just a few servings of rice a week? You could eat nearly a serving every day and still stay within the daily arsenic limits set for drinking water.

Well, Consumer Reports felt the 10 ppb water standard was too lax, so, it went with the “most protective standard in the country,” at 5 ppb. Guess where it came from? New Jersey. Good for New Jersey! So, by using 5 ppb instead of 10 ppb in the calculation, you can see how Consumer Reports got to its only-a-few-servings-of-rice-a-week recommendation. Presumably, that’s based on average arsenic levels in rice. If you choose a lower-arsenic rice, one with only half the level of arsenic, can you have four servings a week instead of two? And, if you boil rice like pasta and drain off the excess water, doesn’t that also cut levels in half? If so, then you are up to about eight servings a week. Based on the water standard, apparently, you could still safely eat a serving of rice a day if you choose the right rice and cook it right. I assumed the water limit is ultra-conservative since people are expected to drink water every day of their lives, whereas most people don’t eat rice every day, seven days a week. I made that assumption, but I was wrong. It turns out the opposite is true.

All this time, I had been assuming the current drinking guideline exposure would be safe, which in terms of carcinogens, is usually “1 in a million chances of getting cancer over a lifetime.” I’ve mentioned this before. It’s how cancer-causing substances are typically regulated. If a company wants to release some new chemical, it has to show that it doesn’t cause more than one in a million excess cancer cases. Of course, there are 300 million people in this country, so that one-in-a-million doesn’t make the 300 extra families who have to deal with cancer feel any better, but that’s just the kind of agreed upon “acceptable risk.”

The problem, according to the National Research Council, is that with the current federal drinking water standard for arsenic of 10 μg/L, we are not talking about an excess cancer risk of 1 in a million people, but as high as 1 case in 300 people. Those 300 extra cases of cancer just turned into a million more cases? A million more families dealing with a cancer diagnosis? “This is 3000 times higher than a commonly accepted cancer risk for an environmental carcinogen of 1 case in 1 000 000 people.” If we were to use the normally accepted 1 in a million odds of cancer risk, the water standard would have to be 500 times lower, .02 instead of 10. Even the New Jersey standard is 250 times too high. “While this is a rather drastic difference… it underlines just how little precaution is instilled in the current guidelines.”

Hold on. So why isn’t the water standard .02 instead of 10? Because that “would be nearly impossible to implement” as we just don’t have the technology to get arsenic levels in water that low. The technologically feasible level has been estimated at 3. Okay, so why is the limit 10 and not 3? The decision to use a threshold of 10 instead of 3 was “mainly a budgetary decision.” A threshold of three would cost a lot of money.

So, the current water “safety” limit “is more motivated by politics than by technology.” Nobody wants to be told they have toxic tap water. If they did, they might demand better water treatment and that would be expensive. “As a result, many people drink water at levels very close to the current guideline… and may not be aware that they are exposed to an increased risk of cancer.” Even worse, millions of Americans drink water exceeding the legal limit, as you can see at 5:10 in my video. But, even the people living in areas that meet the legal limit “must understand that current arsenic guidelines are only marginally protective.”

Perhaps we should tell people who drink water—i.e., everyone—“that current arsenic regulations are a cost-benefit compromise and that, based on usual health risk paradigms, the standards should be much lower… People must be made aware that regulatory targets for arsenic should be as close to zero as possible,” and, when it comes to water, we should aim for the reachable limit of 3. What does this mean for rice, though?

Well, first of all, so much for just trying to get rice down to the so-called safe water limit, since that “already exceeds standard [carcinogen] risks and is based on feasibility and cost-benefit compromises,” which “allows for a roughly 500 times higher risk of cancer” than is normally considered acceptable. So, “while authorities ponder when and how they will regulate arsenic concentration in rice,” perhaps we should “curtail or strongly limit our consumption of rice.”

This is the corresponding blog post to the pivotal video in my 13-part series on arsenic in the food supply. The final three videos focus on how to deal practically with the repercussions:

• How Much Arsenic in Rice Is Too Much?
• Is White Rice a Yellow-Light or Red-Light Food?
• Do the Pros of Brown Rice Outweigh the Cons of Arsenic?

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If you missed any of the first nine videos, see:

• Where Does the Arsenic in Chicken Come From?
• Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?
• The Effects of Too Much Arsenic in the Diet
• Cancer Risk from Arsenic in Rice and Seaweed
• Which Rice Has Less Arsenic: Black, Brown, Red, White, or Wild?
• Which Brands and Sources of Rice Have the Least Arsenic?
• How to Cook Rice to Lower Arsenic Levels
• Arsenic in Infant Rice Cereal
• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup

You may also be interested in Benefits of Turmeric for Arsenic Exposure.

My arsenic series reminds me of the extensive video series I did on lead:

• How the Lead Paint Industry Got Away with It
• Lead in Drinking Water
• How the Leaded Gas Industry Got Away with It
• “Normal” Blood Lead Levels Can Be Toxic
• The Effects of Low-Level Lead Exposure in Adults
• How to Lower Lead Levels with Diet: Thiamine, Fiber, Iron, Fat, Fasting?
• How to Lower Lead Levels with Diet: Breakfast, Whole Grains, Milk, Tofu?
• Best Foods for Lead Poisoning: Chlorella, Cilantro, Tomatoes, Moringa?
• Best Food for Lead Poisoning: Garlic
• Can Vitamin C Help with Lead Poisoning?
• Yellow Bell Peppers for Male Infertility and Lead Poisoning?

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

Shots:

• The US FDA has accepted the PR for NDA of mirabegron (oral suspension) and sNDA for Myrbetriq (mirabegron, tablets) for neurogenic detrusor overactivity (NDO) in pediatric patients aged ≥ 3yrs. with anticipated PDUFA date as Mar 28, 2021
• The NDA & sNDA is based on P-III study assessing efficacy, safety, tolerability and PK of mirabegron in children and adolescents aged 3-<18yrs. with NDO and using clean intermittent catheterization
• In 2012, Myrbetriq tablets were initially approved in the US for adults with overactive bladder with symptoms of urge urinary incontinence, urgency and urinary frequency

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Financial Times

The post Astellas Reports the US FDA’s Acceptance of Priority Review for its NDO Therapy first appeared on PharmaShots.

In a recent interview with PharmaShots, Ted Omachi, Global Development Leader for Xolair, and Senior Medical Director of Product Development for Immunology, Genentech shared his views on the approval of Xolair in the US.

Shots:

• The approval is based on P-III POLYP 1 & 2 trials assessing Xolair vs PBO in 138 & 127 adult patients with nasal polyps who had an inadequate response to nasal corticosteroids respectively
• Results: @24wks. improvement in NPS (-1.1 vs 0.1 & -0.9 vs -0.3); improvement in NCS (-0.9 vs -0.4 & -0.7 vs -0.2); no new or unexpected safety signals were identified respectively
• Xolair is the first biologic for the treatment of nasal polyps that targets and blocks IgE. In the US, Novartis & Genentech work together to develop and co-promote Xolair

Tuba: Can you please shed some light on Nasal Polyps? (causes, symptoms, epidemiology, etc.)

Ted: Approximately 13 million people in the U.S. are impacted by nasal polyps, a commonly occurring condition in adults that may be refractory to treatments such as nasal corticosteroids and even surgery. Nasal polyps present as noncancerous growths on the lining of the nasal sinuses or nasal cavity associated with irritation and inflammation and, as such, they can block normal airflow. Nasal polyps may also co-occur with other respiratory conditions, such as allergies and asthma. They may become quite large and develop in both nostrils, leading to a loss of smell, nasal congestion, chronic runny nose, and post-nasal drip. This condition can cause significant long-term symptoms and impact on patients’ lives. While the pathophysiology of nasal polyps is not entirely elucidated, we know that it is an inflammatory condition in which immunoglobulin E (IgE) plays an important role.

Tuba:  A quick highlight of clinical data submitted for the approval of Xolair in nasal polyps to the U.S. FDA.

Ted: The FDA’s approval is based on results from the Phase III POLYP 1 and POLYP 2 pivotal trials conducted in adult patients. To be enrolled in the study, patients needed to have large polyps in both nostrils and significant symptoms, with persistent symptoms and large polyps even after treatment with nasal steroids. Patients were then given either Xolair or placebo, in a blinded fashion, while continuing to receive nasal steroids. Patients who received Xolair had statistically significantly greater improvements, as compared to placebo, over the approximate six-month duration of the study, from baseline to Week 24, in both of the co-primary endpoints: Nasal Polyp Score (NPS) and Nasal Congestion Score (NCS). NPS is an objective measure of the size of the polyps, as determined by endoscopy, while NCS is a measure of the degree of nasal blockage, as determined by patients’ assessment of their own symptoms. The greater improvements in NPS and NCS in the Xolair group as compared to the placebo group were observed as early as the first assessment at Week 4 in both studies. They also had statistically significant improvements in smell, post-nasal drip, and runny nose. The safety profile in POLYP 1 and POLYP 2 was consistent with the established safety profile for Xolair, which is based on more than 17 years of real-world experience in allergic asthma and more than 1.3 million patient-years of usage in clinical practice. 

Tuba: Can you explain how Xolair works for nasal polyps (mechanism of action)?

Ted: Xolair is the first biologic for the treatment of nasal polyps that targets and blocks immunoglobulin E (IgE), a key driver of inflammation. By reducing free IgE, down-regulating high-affinity IgE receptors and limiting mast cell degranulation, Xolair minimizes the release of mediators throughout the allergic inflammatory cascade.

Tuba: Can you provide some insights on RoA for Xolair in Nasal Polyps?

Ted: We do not comment on sales forecasts or projections.

Tuba:  Can our readers have more details on any ongoing and upcoming patients support programs and efforts for patient adherence?

Ted: As a physician, I know firsthand that adherence and access to medicines are some of the most important factors to ensure the safety and effectiveness of treatment for patients. At Genentech, we are committed to helping patients access to the medicines prescribed by their physician, even if they can’t afford them. For more than 20 years, we have helped more than 2.2 million people get the medicine they need through patient assistance programs like Genentech Access Solutions and the Genentech Patient Foundation.

Tuba: Xolair is now approved in multiple indications including allergic asthma, CIU and nasal polyps. What’s next?

Ted: We are committed to exploring the full potential of Xolair across a range of respiratory diseases.

In August 2020, the FDA accepted our sBLA for a new self-injection option for Xolair prefilled syringe formulation across all approved US indications, with a decision on approval anticipated in Q1 2021.

Additionally, in 2018 the FDA granted Breakthrough Therapy Designation to Xolair as a potential treatment for food allergies. Xolair is currently being investigated as a potential treatment for multiple food allergies in Phase III clinical trial, Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen Oral Immunotherapy in Food Allergic Children and Adults, or OUtMATCH trial. The trial is supported by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, Genentech, and Novartis. We are hopeful about the potential to address this area of the significant need for patients as there are limited FDA-approved treatments that help prevent severe reactions due to food allergies.

Tuba: Are you focusing on approvals in different countries?

Ted: Xolair is approved for nasal polyps-related conditions in several countries outside of the U.S. In August 2020, the European Commission approved Xolair as add-on therapy with intranasal corticosteroids for the treatment of adults with severe chronic rhinosinusitis with nasal polyps (CRSwNP) for whom therapy with intranasal corticosteroids does not provide adequate disease control. Additionally, Xolair is approved in more than 10 other countries throughout the world for nasal polyps or CRSwNP. Further regulatory reviews of Xolair to treat patients with CRSwNP (often referred to as nasal polyposis) are currently underway in multiple countries.

Tuba: What kind of pricing difference shall be expected for Xolair for Nasal Polyps vs. Xolair for other indications?

Ted: When determining the prices of our medicines, we take into consideration a number of factors including clinical benefit, patient access, investments required for future medications, and our responsibility to patients, society, and shareholders. The dosing of Xolair for nasal polyps and allergic asthma depends on the patient’s weight and serum IgE levels, which affects the cost of therapy.

About Ted Omachi:

Ted Omachi is a Medical Director in the Product Development Immunology group at Genentech, focusing on late-stage development in respiratory and allergic diseases. He joined Genentech in 2013, starting in the Medical Affairs group before transitioning to Product Development in 2015.

Related Post:  ViewPoints Interview: Genentech’s Ted Omachi Shares Insights on Xolair (omalizumab) PFS

The post ViewPoints Interview: Genentech’s Ted Omachi Shares Insight on the US FDA’s Approval of Xolair in Nasal polyps first appeared on PharmaShots.

What You Should Know:

– TigerConnect has announced an expansion in their suite
through the acquisition of Critical Alert, a leading provider of
enterprise-grade middleware for hospitals and health systems.

– For the hundreds of thousands of nurses that currently
use TigerConnect, these new capabilities will deliver real-time, contextual
information to their mobile device or desktop to allow them to work smarter,
prioritize responses, and efficiently coordinate care, all within the same
reliable TigerConnect platform they use every day for enterprise messaging.

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 TigerConnect®,
a care team collaboration solution, today announced the acquisition
of Critical Alert, a Jacksonville,
FL-based leading provider of enterprise-grade middleware for hospitals and
health systems. Critical Alert’s product suite consists of a middleware suite
of products as well as traditional nurse call hardware servicing over 200
hospitals in North America. Financial details of the acquisition were not
disclosed.

Real-Time Care Team Collaboration for Hospitals

Founded in 1983, Cloud-native and mobile-first, Critical
Alert’s middleware solution enables any health system to combine nurse call,
alarm and event management, medical device interoperability, and clinical
workflow analytics.  TigerConnect will integrate Critical
Alert’s middleware stack into its platform to power a wide range of alert types
and alarm management enhancements for TigerConnect’s customers. Critical
Alert’s Nurse Call hardware business will continue to operate under its
namesake as a standalone business unit.  

When combined with Critical Alert’s middleware, TigerConnect dramatically
enhances the value proposition to nursing, IT leadership, and end-users. This ‘dream
suite’ of capabilities comes at a time when nurse burnout is at a record high
and chronic nurse shortages are severely challenging organizations’ ability to
deliver the best quality care.

“We see the Critical Alert acquisition as highly strategic and
a natural evolution of our already-robust collaboration
platform,” said Brad Brooks, CEO and co-founder of TigerConnect. “For the
hundreds of thousands of nurses that currently use TigerConnect, these new
capabilities will deliver real-time, contextual information to their mobile
device or desktop to allow them to work smarter, prioritize responses, and
efficiently coordinate care, all within the same reliable TigerConnect platform
they use every day for enterprise messaging.”

Post-Acquisition Plans

Joining TigerConnect is Critical Alert CEO John
Elms, who will assume the role as TigerConnect Chief Product Officer,
guiding the integration of the two companies’ technologies and leading the
development of all future product offerings. Wil Lukens, currently VP of Sales
for Critical Alert, will assume the role of General Manager of Critical Alert’s
traditional Nurse Call hardware unit. 

“The timing of the deal and the fit of these two companies aligned perfectly,” said John Elms, CEO of Critical Alert. “Two best-in-class, highly complementary solutions coming together to solve some of the chronic challenges—alarm fatigue, response prioritization, resource optimization—that have driven nurse teams to the brink. Together, these unified technologies will make care professionals’ lives easier, not harder, and I couldn’t be more excited to lead the TigerConnect product organization into this next chapter.”

Critical Alert Integration with TigerConnect Plans

TigerConnect’s robust product suite, which includes care
team collaboration (TigerFlow®), on-call scheduling (TigerSchedule®), virtual
care/telemedicine (TigerTouch®), and now virtualized nurse call and
alerts/alarm management (Critical Alert middleware), will help transform
hospitals and healthcare organizations into the real-time health systems of the
future. 

Hardware-free Middleware Forms the Foundation

With a shared cloud-native approach, Critical Alert’s
advanced middleware seamlessly fuses TigerConnect’s care team
collaboration with alarm management and event notifications. Deep
enterprise-level integrations with hospital systems enable the centralization
of clinical workflow management and real-time analytics. Integrating these
systems will have a sizable impact on customer organizations’ productivity and
patient care.

Next Generation Nurse Call

Critical Alert’s nurse call solution brings a modern, badly
needed upgrade to legacy systems, extending both their life and feature-set. A
single mobile- or desktop-enabled user-interface brings vital contextual
information about requests while allowing for centralized answering of nurse
call alerts and management of workflows and assignments. These streamlined
workflows reduce noise and clinical interruptions while improving
responsiveness.

Physiological Monitoring – Less Noise, More Signal

The FDA-cleared offering intelligently routes context-rich
alarm notifications from clinical systems to TigerFlow+. An easy-to-use
workflow builder ensures alerts are prioritized accordingly and are routed to
the appropriate caregiver, suppressing unnecessary noise. The filtering,
mobilization, and escalation of alerts pairs with TigerConnect Teams,
allowing for prompt responses in critical situations.

Smart Bed Alarms for Enhanced Patient Safety

Integrations with popular smart bed systems provide remote
monitoring of bed status details, informing nurses whether they should walk or
run to a patient’s room. Staff can review and adjust bed compliance settings
from their mobile device and receive fall prevention notifications if safe-bed
configuration is compromised.

Real-time Location System (RTLS) Measures What Matters

The integration of RTLS with a deployed nurse call
application greatly enhances the data available to clinical leadership. The
combined TigerConnect/Critical Alert offering enables real-time tracking
of staff location (presence) and time spent on tasks, providing deeper insights
into resource planning, workflow effectiveness and ongoing process improvement
initiatives.

Advanced Analytics for Deeper Workflow Insights

A better understanding of patient behavior and workflows
helps reveal areas for optimization that can lead to improved patient care and
staff efficacy. The new combined platform capabilities centralize the
collection and tracking of patient event data and nurse task efficiency,
turning insights into action. Advanced analytics also allow for identifying,
documenting, and benchmarking responsiveness, compliance, resource allocation,
and patient throughput across the health system. 

Availability

This new integrated functionality is expected to be
available to TigerConnect customers in Q1 of 2021.

Shots:

• The US FDA has granted EUA to Nirmidas’ COVID-19 rapid antibody IgG/IgM test for use in POC settings, delivering results in 15-20min
• Using a fingerstick serology test, the MidaSpot COVID-19 Ab combo detection kit checks for Ab against the RBD antigen and showed 100% sensitivity for IgG after 14 days post symptom onset and 100% sensitivity for IgM after 7 days post symptom onset
• With this, Nirmidas became the first US company to receive EUA for a COVID-19 POC fingerstick Ab test. The kit can now be administered in CLIA-waived settings for POC testing such as doctor’s offices, pharmacies, ERs

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Yahoo Finance

The post Nirmidas Receives the US FDA’s EUA for its COVID-19 Rapid Antibody Fingerstick Test first appeared on PharmaShots.

Elsevier, the data analytics business specialized
in science and health, and Heel, a pharmaceutical company specialized in
developing and manufacturing medicines made from natural ingredients, have
recently completed a series of research projects with a focus on improving
exploratory preclinical studies.

“We at Heel are pioneers in the field
of systems research and have a strong commitment to scientific excellence and
the generation of evidence. Our aim is to find out how these medicines work in
the body and to develop therapies which are tailored even more to patients’
medical needs,’’ said Dr. Kathrin Hemmer, a scientist at Heel. “We chose to
partner with Elsevier because of its expertise in scientific information search.
Assistance from the Professional Services group allowed us to get a single
access to all the Elsevier’s R&D solutions advancing our exploratory
research programs.”

“Research success requires connecting, combining
and harmonizing data from different sources. We are helping researchers to select
the best evidence-based strategy for mechanism-based drug action”, said Dr.
Taisiya Bezhaeva, Professional Services Consultant at Elsevier.

“Together with Heel, we designed a
series of projects to find preclinical models for drug action discovery,
identify key biomarkers, and research platforms validated by the international
research community. We focused on the broad range of disease areas, overall
covering more than 10000 literature sources, as well as FDA and EMA drug
approval documents. We also supported researchers by providing key opinion leaders
(KOL) and potential academic and commercial partners helping Heel to direct and
facilitate the course of their studies”, said Dr. Maria Shkrob, Senior Consultant
in Professional Services at Elsevier.  

ELSEVIER SOURCES AND METHODS

• Embase (https://www.embase.com) provides unparalleled coverage of the biomedical literature
• Elsevier Text Mining (https://demo.elseviertextmining.com) uses Natural Language Processing and deep taxonomy-based indexing to find concepts and relations between them
• PharmaPendium (https://www.pharmapendium.com) provides access to comprehensive drug safety information, drug metabolism, clinical efficacy data and FDA and EMA drug approval documents
• Scopus (https://www.elsevier.com/solutions/scopus) uses complex algorithms of author and institution disambiguation to identify key opinion leaders and key research institutions for a specific area
• SciVal (https://www.elsevier.com/solutions/scival) allows users to visualize research performance, develop strategic partnerships, and identify and analyze new, emerging research trends

KEY BENEFITS

• Connecting, combining and harmonizing data from different
  sources
• Big-data & evidence-based approach to identify complex molecular
  mechanisms and biological networks for natural active compounds
• Such approach combined with advanced text-mining technologies and
  statistics is a powerful, feasible
  and universal analytic method to select the best strategy for exploratory
  research to demonstrate pharmacodynamic actions, safety and efficacy
• Support to strengthen scientific credibility and
  facilitate market positioning

In an unusual move, the Food and Drug Administration issued an alert about false negatives in startup Curative’s Covid-19 test. The test was being used by the County of Los Angeles, as well as Congress.

Tinkering with the dosing schedule of COVID-19 vaccines runs a “significant risk” to public health, the FDA has warned in a statement after the UK announced plans to prioritise the first doses of two approved shots.

The regulator made its comments after the UK announced a policy of stretching the gap between doses of the vaccine to up to 12 weeks to ensure maximum coverage of vulnerable groups.

The UK’s Joint Committee for Vaccines and Immunisation (JCVI) has recommended prioritising the first shot of the vaccines from Pfizer/BioNTech and AstraZeneca – but said that the follow-up booster dose can be delayed by up to 12 weeks.

According to the UK’s drug regulator the Pfizer/BioNTech vaccine doses should be taken at least 21 days apart while AZ vaccine’s booster doses should be given between four and 12 weeks apart.

But the FDA has issued a statement warning against such a strategy and reminded the public about the importance of receiving their second dose on time.

In the US, the Pfizer and BioNTech vaccine and a rival from Moderna is approved with a four-week gap between doses, but the AstraZeneca vaccine is not.

The FDA said it had been monitoring discussions and news reports about the policy and concluded that extending the time between doses, changing the dose and mixing and matching vaccines are “reasonable questions” to consider and evaluate.

But in a strongly-worded statement it said that changing the vaccine schedule without evidence could “undermine the historic vaccination efforts to protect the population from COVID-19.”

There are concerns elsewhere that the extended dosing schedule could also lead to resistant forms of the virus taking hold at a time when there is a new and highly infectious variant spreading through the UK.

In the US, doctors pointed out that the slow roll-out of the vaccines is due to logistical challenges rather than supply and urged authorities to focus on this rather than delaying the dosing schedule.

In the meantime, the UK government has pushed ahead with plans to vaccinate everyone in the four highest-priority groups by mid-February.

This will require the NHS to start delivering at least two million shots a week from next week.

AZ’s chief executive Pascal Soriot said it would be possible to manufacture the required vaccines, although it remains to be seen whether output will be increased in time to hit the target.

The government said it already has a batch of 530,000 doses and a further 450,000 are reportedly due to be available by the end of week according to Oxford University’s regius professor of medicine Sir John Bell.

Several million more doses of the vaccine are understood to have been manufactured but have not yet been bottled in vials.

The post FDA and doctors warn against extending COVID-19 vaccine dose gap appeared first on .

Shots:

• The BLA for teplizumab to delay or prevention of clinical T1D in at-risk individuals has been filed to the US FDA. The FDA has also granted Provention’s request for Priority Review and assigned anticipated PDUFA date as Jul 02, 2021
• The company is actively working with the FDA to support their review while planning to launch the therapy in Q3’21
• Teplizumab is an investigational anti-CD3 mAb and has previously received the US FDA’s BT designation. If approved, teplizumab will be the first disease-modifying therapy for T1D

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: ProventionBio

The post Provention Bio Reports Submission of BLA and Priority Review to the US FDA for Teplizumab to Prevent T1D first appeared on PharmaShots.

When it comes to rice and rice-based products, pediatric nutrition authorities have recommended that arsenic intake should be as low as possible.

“The US Food and Drug Administration (FDA) has been monitoring the arsenic content in foods” for decades, yet despite the “well-established science describing the health risks associated with arsenic exposure, no standards have been set limiting the amount of arsenic allowable in foods” in the United States. In 2001, the EPA “adopted a new stricter standard for arsenic in drinking water,” and in 2013, the FDA proposed a legal limit for apple juice. “There are still no standards for arsenic in food products despite the fact that food sources are our main source of exposure.”

Unlike the United States, China has standards. As of 2014, China set a maximum threshold of inorganic arsenic at 150 parts per billion, stricter than the World Health Organization’s limit of 200 ppb. In the United States, a 200 ppb limit wouldn’t change the cancer risk much. If we had China’s safety limits of 150 ppb, though, cancer risk would be reduced up to 23 percent and a maximum threshold of 100 ppb would lower cancer risk up to 47 percent—but that could seriously affect the rice industry. In other words, U.S. rice is so contaminated with arsenic that if a safety standard that really cut down on cancer risk were set, it “would wipe out the U.S. rice market.” However, with no limits, what’s the incentive for the rice industry to change its practices? Setting arsenic limits would not only directly protect consumers but also encourage the industry to stop planting rice paddies on arsenic-contaminated land.

Those cancer estimates are based on arsenic-contaminated water studies. Might the arsenic in rice somehow have a different effect? You don’t know…until you put it to the test. We know rice has a lot of toxic arsenic that urine studies have shown we absorb into our body, but there hadn’t been any studies demonstrating “deleterious health impacts” specific to rice arsenic—until now. Since arsenic causes bladder cancer, the researchers figured they would see what kind of DNA mutations the urine of rice eaters can have on human bladder cells growing in a petri dish. And, indeed, they clearly demonstrated that eating a lot of arsenic-contaminated rice every day can “give rise to significant amounts of genetic damage,” the kind that‘s associated with cancer. Yes, but the study used pretty contaminated rice. However, only about 10 percent of the rice in certain parts of Asia might ever reach those levels of contamination, though a quarter of rice in parts of Europe might and more half in the United States, making for considerable public health implications.

So, “there remains little mystery surrounding the health risks associated with arsenic levels in rice. The remaining mystery is why long-overdue standards for arsenic levels in rice have not been set by the FDA” in the United States, but that may be changing. In 2016, the FDA proposed setting a limit on toxic arsenic—at least in infant rice cereal, which I discuss in my video Arsenic in Infant Rice Cereal.

As you can see at 3:24 in my video, infants and children under four years of age average the highest rice intake, in part because they eat about three times the amount of food in relation to their body size, so there’s an especially “urgent need for regulatory limits” on toxic arsenic in baby food.

Pediatric nutrition authorities have recommended that when it comes to rice and rice-based products, “arsenic intake should be as low as possible,” but how about as early as possible? Approximately 90 percent of pregnant women eat rice, which may end up having “adverse health effects” on the baby.

You can estimate how much rice the mother ate while pregnant by analyzing arsenic levels in the infant’s toenail clippings. “Specifically, an increase of 1/4 cup of rice per day was associated with a 16.9% increase in infants toenail [arsenic] concentration,” which indicates that arsenic in rice can be passed along to the fetus. What might that arsenic do? A quarter cup of rice worth of arsenic has been associated with low birth weight, increased respiratory infections, and, above that, a 5- to 6-point reduction in IQ, among other issues. So, “based on the FDA’s findings, it would be prudent for pregnant women to consume a variety of foods, including varied grains (such as wheat, oats, and barley),” which is code for cut down on rice. Saying eat less of anything, after all, is bad for business.

Once the baby is weaning, “what’s a parent to do?” Asks Consumer Reports, “To reduce arsenic risks, we recommend that babies eat no more than 1 serving of infant rice cereal per day on average. And their diets should include cereals made of wheat, oatmeal, or corn grits, which contain significantly lower levels of arsenic”—that is, rely on other grains, which are much less contaminated than rice. As the American Academy of Pediatrics has emphasized, “there is no demonstrated benefit of rice cereal over those made with other grains such as oat, barley, and multigrain cereals, all of which have lower arsenic levels than rice cereal.” As you can see at 5:28 in my video, reducing consumption of infant rice cereal to just two servings per week could have an even more dramatic effect on reducing risk.

 The proposed limit on toxic arsenic in infant rice cereals would end up removing about half of the products off the shelves. The FDA analyzed more than 500 infant and toddler foods, and the highest levels of toxic arsenic were found in organic brown rice cereals and “Toddler Puffs.” Based on the wording in the report, these puffs appear to be from the Happy Baby brand. Not-so-happy baby if they suffer brain damage or grow up to get cancer. A single serving could expose infants to twice the tolerable arsenic intake set by the EPA for water. I contacted the Happy Baby company and was told they “are not able to provide any comments” on the FDA’s results.

“Eliminating all rice and rice products from the diets of infants and small children up to 6 years old could reduce the lifetime cancer risk from inorganic arsenic in rice and rice products by 6% and 23% respectively.” That is, there would be a 6 percent lower chance of developing lung or bladder cancer later in life if infants stopped, and a 23 percent lower chance if young kids stopped. However, switching to other grains is a move described as “drastic and dramatic,” creating “a huge crisis”—for the rice industry, presumably—and therefore “not feasible at all.”

I was hoping Happy Baby, upon learning of the concerning FDA arsenic toddler puffs data (regardless of whether the data were about its brand or not) would have kicked its own testing and potential remediation into high gear like Lundberg did (see Which Brands and Sources of Rice Have the Least Arsenic?). But, unfortunately, in my email correspondence with the company, I got no sense that it did.

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For more videos on this topic, see:

• Benefits of Turmeric for Arsenic Exposure
• Where Does the Arsenic in Chicken Come From?
• Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?
• The Effects of Too Much Arsenic in the Diet
• Cancer Risk from Arsenic in Rice and Seaweed
• Which Rice Has Less Arsenic: Black, Brown, Red, White or Wild?
• How to Cook Rice to Lower Arsenic Levels

And here are five more:

• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup
• How Risky Is the Arsenic in Rice?
• How Much Arsenic in Rice Is Too Much?
• Is White Rice a Yellow-Light or Red-Light Food?
• Do the Pros of Brown Rice Outweigh the Cons of Arsenic?

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

What You Should Know:

– The American Medical Association (AMA) announced the addition of three Current Procedural Terminology (CPT) codes for AstraZeneca’s COVID-19 vaccine.

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The American Medical Association (AMA) today announced that
the Current Procedural Terminology (CPT®) code set is
being updated by the CPT Editorial Panel to include immunization and
administration codes that are unique to the COVID-19 vaccine under development
by AstraZeneca and University of Oxford.

The new CPT codes will be effective for use on the condition
that the AstraZeneca vaccine receives approval or emergency use authorizations
from the Food and Drug Administration (FDA). The AMA is publishing the new CPT
codes now to ensure electronic systems across the U.S. health care
system are updated and prepared for the prospect of FDA approval or
authorization for the AstraZeneca vaccine.

Growing List
of CPT Codes to Support COVID-19 Vaccines

The AstraZeneca vaccine joins two other COVID-19 vaccines that were previously issued unique CPT codes to report vaccine-specific immunizations once FDA approval or authorization has been granted. On Nov. 10, the AMA announced that COVID-19 vaccines developed by Pfizer and Moderna had been issued unique CPT codes to clinically distinguish each vaccine for better tracking, reporting, and analysis that supports data-driven planning and allocation

AstraZeneca COVID-19 Vaccine CPT Codes Overview

The new
Category I CPT code and long descriptor for the AstraZeneca vaccine are:

91302: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) (coronavirus disease [COVID-19]) vaccine, DNA, spike protein, chimpanzee adenovirus Oxford 1 (ChAdOx1) vector, preservative-free, 5×10¹⁰ viral particles/0.5mL dosage, for intramuscular use

In addition to the new vaccine-specific product CPT code, the AstraZeneca vaccine has been issued vaccine administration CPT codes that are distinct to its two-dose immunization schedule. These CPT codes report the actual work of administering the vaccine, in addition to all necessary counseling provided to patients or caregivers and updating the electronic record.

For quick reference, the new vaccine administration CPT codes and long descriptors for the AstraZeneca vaccine are:

0021A: Immunization administration by intramuscular injection of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) (coronavirus disease [COVID-19]) vaccine, DNA, spike protein, chimpanzee adenovirus Oxford 1 (ChAdOx1) vector, preservative-free, 5×10¹⁰ viral particles/0.5mL dosage; first dose 

0022A: second dose

In addition to the long descriptors, short and medium descriptors for all the new vaccine-specific CPT codes can be accessed on the AMA website, along with several other recent modifications to the CPT code set that have helped streamline the public health response to the SAR-CoV-2 virus and the COVID-19 disease.

“A mass vaccination effort with the first available COVID-19 vaccines presents enormous logistical challenges,” said AMA President Susan R. Bailey, M.D. “The ability to correlate each COVID-19 vaccine with its own unique CPT code provides analytical and tracking advantages that ensures optimal vaccine distribution and administration, especially for patients who will need to complete the two-dose immunization schedule.”

---

What You Should Know:

– Amazon, CVS Health, Thermo Fisher Scientific join forces to
promote employer-based testing As part of a comprehensive COVID-19 testing strategy.

– The coalition, named Workplace Employers Alliance for
COVID-19 Testing (WE ACT), believes
that employer-based testing programs are essential to keeping employees safe
during the current public health emergency.

– WE ACT aims to
advance a comprehensive national testing strategy that includes clear guidance
for the implementation of testing programs and results reporting; to ensure
access to high-quality, FDA authorized COVID-19 tests for employers; and to
serve as a resource for any employer who wishes to launch or expand an
employer–based testing program.

– As a nonpartisan coalition, WE ACT and its partners
believe that combating the COVID-19
pandemic requires an all-hands-on-deck approach.

What You Should Know:

– Philips and BioIntelliSense has been selected by the
U.S. Army Medical Research and Development Command (USAMRDC) to receive nearly $2.8M
from the U.S. Department of Defense (DoD) to validate BioIntelliSense’s
FDA-cleared BioSticker device for the early detection of COVID-19 symptoms.

– Working with the University of Colorado Anschutz
Medical Campus, the clinical study will consist of 2,500 eligible participants
with a recent, known COVID-19 exposure and/or a person experiencing early
COVID-19 symptoms.

---

Royal
Philips and BioIntelliSense,
Inc., a continuous health monitoring and clinical intelligence company, today
announced they have been selected by the U.S. Army Medical Research and
Development Command (USAMRDC) to receive nearly $2.8M from the U.S. Department
of Defense (DoD) through a Medical Technology Enterprise Consortium (MTEC)
award to validate BioIntelliSense’s FDA-cleared BioSticker device for the early
detection of COVID-19
symptoms. The goal of the award is to accelerate the use of wearable
diagnostics for the benefit of military and public health through the early
identification and containment of pre-symptomatic COVID-19 cases.

Medical-Grade Wearable for Early COVID-19 Detection

As millions of individuals have been screened and tested, the emerging research on traditional screening methods is revealing how challenging it is to detect the risk of COVID-19 infections early. Temperature checks have proven to be unreliable and even amplified testing (PCR) has proven to be ineffective in identifying the virus in the early days of infection.

The FDA-cleared BioSticker is an advanced on-body sensor
that allows for effortless continuous monitoring of temperature and vital signs
combined with advanced analytics, enables the BioSticker to identify
statistically meaningful trends and screen for early potential COVID-19
infection.

“The medical-grade BioSticker wearable, combined with advanced diagnostic algorithms, may serve as the basis for identifying pre- and very early symptomatic COVID-19 cases, allow for earlier treatment for infected individuals, as well as reduce the spread of the virus to others,” said James Mault, MD, Founder and CEO of BioIntelliSense.

Clinical Trial Details

Working with the University of Colorado Anschutz Medical Campus, the
clinical study will consist of 2,500 eligible participants with a recent, known
COVID-19 exposure and/or a person experiencing early COVID-19 symptoms.
Individuals may learn more about the study eligibility and enroll online
at www.BioStickerCOVIDstudy.com.
The research will focus on the validation of BioIntelliSense’s BioSticker for
early detection of COVID-like symptoms, as well as assessment of scalability,
reliability, software interface, and user environment testing. 

Turning Data into Actionable Insights

While previous studies have shown potential using consumer wearables in relation to COVID-19, this study will leverage BioIntelliSense’s medical-grade wearable, the BioSticker, which enables continuous multi-parameter vital signs monitoring for 30 days and captures data across a broad set of vital signs, physiological biometrics and symptomatic events, including those directly associated with COVID-19.  With its integration into Philips’ remote patient monitoring offerings, this is another example of how cloud-based data collection takes place seamlessly, across multiple settings, from the hospital to the home. Allowing data to be turned into actionable insights and care interventions, while providing connected, patient-centered care across the health continuum. 

Dr. Vik Bebarta, the Founder and Director of the CU Center for COMBAT Research and Professor of Emergency Medicine on the CU Anschutz Medical Campus added: “The University of Colorado School of Medicine and the CU Center for COMBAT Research in the Department of Emergency Medicine are excited to be a lead in this effort that will change how we care for our service members in garrison and our civilians in our communities.  The COMBAT Center aims to solve the DoD’s toughest clinical challenges, and the pandemic is certainly one example. With this progressive solution, we aim to detect COVID in the pre-symptomatic or early symptomatic phase to reduce the spread and initiate early treatment. This trusted military-academic-industry partnership is our strength, as we optimize military readiness and reduce this COVID burden in our community and with frontline healthcare workers.” 

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It can be republished for free.

The recent rollout of two newly authorized COVID-19 vaccines is a bright ray of hope at the pandemic’s darkest hour.

We now have a path that can lead us to happier times — even as we watch and suffer from the horrible onslaught of new infections, hospitalizations and deaths that mark the end of this regrettable year.

Health care workers and nursing home residents have already begun to get shots in the first phase of the rollout. Vaccinations should start to be available to the general public sometime in the first few months of next year.

The two vaccines — one developed by Pfizer and BioNTech, the other by Moderna — use the same novel genetic approach. Their development in under a year, shattering all records, is a marvel of science. It’s also a cause for concern for millions of Americans who fear the uncertainty of an unknown technology.

The clinical trial data for the Pfizer and Moderna vaccines show that when both shots of the dual-injection immunization are taken, three weeks to a month apart, they are about 95% effective — at least at preventing severe COVID illness.

However, “a vaccine that remains in the vial is 0% effective no matter what the data show,” says Dr. Walter Orenstein, a professor of infectious diseases at the Emory University School of Medicine in Atlanta and associate director of the Emory Vaccine Center.

Hence, the imperative of persuading millions of people, across racial, cultural, religious, political and generational lines, to get immunized when a vaccine becomes available to them. A survey published this month showed 45% of respondents are taking a wait-and-see approach to vaccination.

Because the vaccines were developed under duress as the coronavirus exacted its deadly toll, the premium was on speed — “warp speed.” So although the number of people in the trials is as large as or larger than in previous vaccine trials, some key questions won’t be answered until millions more are vaccinated.

For example, we don’t know to what extent the vaccines will keep us from transmitting or contracting the virus — though the protection from potentially fatal illness they are likely to confer is in itself something of a miracle.

We don’t know whether irreversible side effects might emerge, or who is at higher risk from them. And we don’t know whether we’ll need to get vaccinated every year, every three years, or never again.

These unknowns add to the challenges faced by the federal government, local health authorities, medical professionals and private sector entities as they seek to persuade people across the broadest possible swath of the population to get a vaccine.

Skepticism resides in many quarters, including among African Americans, many of whom have a long-standing mistrust of the medical world; the vocal “anti-vaxxers”; and people of all stripes with perfectly understandable doubts. Not to mention communities with language barriers and immigrants without documents — more than 2 million strong in California — who may fear coming forward.

Here are answers to some questions you might be asking yourself about the new vaccines:

Q: How can I be sure they’re safe?

There’s no ironclad guarantee. But the federal Food and Drug Administration, in authorizing the Moderna and Pfizer vaccines, determined that their benefits outweighed their risks.

The side effects observed in trial participants were common to other vaccines: pain at the injection site, fatigue, headache, muscle pain and chills. “Those are minor side effects, and the benefit is not dying from this disease,” says Dr. George Rutherford, a professor of epidemiology at the University of California-San Francisco.

Saturday, the Centers for Disease Control and Prevention reported six cases of anaphylactic allergic reaction in the first 272,000 people who got the Pfizer vaccine outside the clinical trials. This has led the CDC to recommend that people receiving the vaccine be observed for up to 30 minutes afterward.

It’s possible other unexpected adverse effects could pop up down the road. “The chances are low, but they are not zero,” says Orenstein. There’s not enough data yet to know if the vaccines pose an elevated risk to pregnant or lactating women, for example, or to immunocompromised people, such as those with HIV. And we know very little about the effects in children, who were not in the initial trials and for whom the vaccines are not authorized.

Q: Why should my family and I take it?

First of all, because you will protect yourselves from the possibility of severe illness or even death. Also, by getting vaccinated you will be doing your part to achieve a vaccination rate high enough to end the pandemic. Nobody knows exactly what percentage of the population needs to get inoculated for that to happen, but infectious disease experts put the number somewhere between 60% and 70% — perhaps even a little higher. Think of it as a civic duty to get your shots.

Q: So, when can I get mine?

It depends on your health status, age and work. In the first phase, already underway, health care workers and nursing home residents are getting vaccinated. The 40 million Moderna and Pfizer doses expected to be available by year’s end should immunize most of them.

Next in line are people 75 and older and essential workers in various public-facing jobs. They will be followed by people ages 65-74 and those under 65 with certain medical conditions that put them at high risk. Enough vaccine could be available for the rest of the population by late spring, but summer or even fall is more likely. Already, some distribution bottlenecks have developed.

On the bright side, two other vaccines — one from Johnson & Johnson, the other from AstraZeneca and Oxford University — could win FDA authorization early next year, significantly increasing the supply.

Q: Once I’m vaccinated, can I finally stop wearing a mask and physical distancing?

No. Especially not early on, before a lot of people have been vaccinated. One reason for that is self-protection. The Moderna and Pfizer vaccines are 95% effective, but that means you still have a 5% chance of falling ill if you are exposed to someone who hasn’t been vaccinated — or who has been but is still transmitting the virus.

Another reason is to protect others, since you could be the one shedding virus despite the vaccination.

Q: I’ve already had COVID-19, so I don’t need the vaccine, right?

We don’t know for sure how long exposure to the virus protects you from reinfection. Protection probably lasts at least a few months, but public health experts say it’s a good idea to get vaccinated when your turn comes up — especially if it’s been many months since you tested positive.

There’s been some talk among health officials of pushing those who’ve been infected in the last 90 days or so toward the back of the line, to ensure adequate supply for those who might be at higher risk.

Q: How long before our lives get back to normal?

“If everything goes well, next Thanksgiving might be near normal, and we might be getting close to that by the summer,” says Dr. William Schaffner, a professor of infectious diseases at the Vanderbilt University School of Medicine in Nashville, Tennessee. ”But there would have to be substantial acceptance of the vaccine and data showing the virus moving in a downward direction.”

This story was produced by KHN, which publishes California Healthline, an editorially independent service of the California Health Care Foundation.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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This story can be republished for free (details).

One tray of COVID-19 vaccine from pharmaceutical giant Pfizer contains 975 doses — way too many for a rural hospital in Arkansas.

But with the logistical gymnastics required to safely get the Pfizer vaccine to rural health care workers, splitting the trays into smaller shipments has its own dangers. Once out of the freezer that keeps it at 94 degrees below zero, the vaccine lasts only five days and must be refrigerated in transit.

In Arkansas — where over 40% of its counties are rural and COVID infections are climbing — solving this distribution puzzle is urgently critical, said Dr. Jennifer Dillaha, the state’s epidemiologist.

“If their providers come down with COVID-19,” Dillaha said, “there’s no one there to take care of the patients.”

Such quandaries resonate with officials in Georgia, Kentucky, Utah, Indiana, Wisconsin and Colorado. The first push of the nation’s mass COVID vaccination effort has been chaotic, marked by a lack of guidance and miscommunication from the federal level.

With Washington punting most vaccination decisions, each state and county is left to weigh where to send vaccines first and which of two vaccines authorized by the Food and Drug Administration for emergency use makes the most sense for each nursing home, hospital, local health department and even school. And after warning for months that they lacked the resources to distribute vaccines, state officials are only now set to receive a major bump in funding — $8.75 billion in Congress’ latest relief bill, which lawmakers are likely to pass this week.

The feat facing public health officials has “absolutely no comparison” in recent history, said Claire Hannan, executive director of the Association of Immunization Managers.

Officials who thought the H1N1 swine flu shot in 2009 was a logistical nightmare say it now looks simple in comparison. “It was a flu vaccine. It was one dose. It came at refrigerator-stable temperatures,” Hannan said. “It was nothing like this.”

Within just a few days, the logistical barriers of the vaccine made by Pfizer and BioNTech were laid bare. Many officials now hang their hopes on Moderna, whose vaccine comes in containers of 100 doses, doesn’t require deep freezing and is good for 30 days from the time it’s shipped.

The federal government had divvied up nearly 8 million doses of Pfizer and Moderna’s vaccines to distribute this week, on top of roughly 3 million Pfizer shots that were sent last week, said Army Gen. Gustave Perna, chief operating officer of the Trump administration’s Operation Warp Speed effort.

Perna said he took “personal responsibility” for overstating how many Pfizer doses states would receive.

Federal delays have led to confusion, Dillaha said: “Sometimes we don’t have information from CDC or Operation Warp Speed until right before a decision needs to be made.”

Officials in other states painted a mixed picture of the rollout.

Georgia’s Coastal Health District, which oversees public health for eight counties and has offices in Savannah and Brunswick, spent more than $27,000 on two ultra-cold freezers for the Pfizer vaccine, which it’s treating “like gold,” said Dr. Lawton Davis, its health director. Health care workers are being asked to travel, some up to 40 minutes, to get their vaccinations, because shipping them would risk wasting doses, he said. Vaccination uptake has been lower than Davis would like to see. “It’s sort of a jigsaw puzzle and balancing act,” he said. “We’re kind of learning as we go.”

In Utah, sites to vaccinate teachers and first responders starting in January had no capability to store the Pfizer vaccine, although officials are trying to secure some ultra-cold storage, a state department of health spokesperson said. Very few of Kentucky’s local health offices could store the Pfizer shots, because of refrigeration requirements and the size of shipments, said Sara Jo Best, public health director of the Lincoln Trail District. Indiana’s state health department had to identify alternative cold storage options for 17 hospitals following changes in guidance for the vaccine thermal shippers.

And in New Hampshire, where the National Guard will help administer vaccines, officials last week were still finalizing details for 13 community-based sites where first responders and health care workers are due to get vaccinated later this month. Jake Leon, a state Health and Human Services spokesperson, said that while the sites will be able to administer both companies’ vaccines, most likely they’ll get Moderna’s because of its easier transport. Even as the earliest vaccines are injected, much remains up in the air.

“It’s day to day and even then hour by hour or minute by minute — what we know and how we plan for it,” Leon said Friday. “We’re building the plane while flying it.”

In all, the Trump administration has bought 900 million COVID vaccine doses from six companies, but most of the vaccines are still in clinical studies. Even the front-runners whose shots have received FDA emergency authorization— Pfizer and BioNTech on Dec. 11, Moderna on Dec. 18 — will require months to manufacture at that scale. The Trump administration plans to distribute 20 million vaccine doses to states by early January, Perna said Saturday.

By spring, officials hope to stage broader vaccine deployment beyond top-priority populations of health care workers, nursing home residents and staff, as well as first responders.

During the effort to vaccinate Americans against H1N1, Dillaha said, health departments set up mass vaccination clinics in their counties and delivered doses to schools. But hospitals are taking charge of parts of the initial COVID immunization campaign, both because health care workers are at highest risk of illness or death from COVID-19, and to pick up the slack from health departments overwhelmed by case investigations and contact tracing from an unending stream of new infections.

Best said her workforce is struggling to keep up with COVID infections alone, much less flu season and upcoming COVID vaccinations. Public health department personnel in Kentucky shrank by 49% from 2009 to 2019, according to state data she supplied. Across the country, 38,000 state and local health positions have disappeared since the 2008 recession. Per capita spending for local health departments has dropped by 18% since 2010.

Nationally, Pfizer and Moderna have signed contracts with the federal government to each provide 100 million vaccine doses by the end of March; Moderna is set to deliver a second tranche of 100 million doses by June. States were playing it safe last week, directing Pfizer vials mainly to facilities with ultra-cold freezers, Hannan said.

“A lot of that vaccine is destined for institutional facilities,” Sean Dickson, director of health policy for West Health Policy Center, said of the Pfizer shots. The center, with the University of Pittsburgh School of Pharmacy, found that 35% of counties have two or fewer facilities to administer COVID vaccines.

The analysis found tremendous variation in how far people would need to drive for the vaccine. Residents of North Dakota, South Dakota, Montana, Wyoming, Nebraska and Kansas face the longest drives, with more than 10% living more than 10 miles from the closest facility that could administer a shot.

Counties with long driving distances between sites and a low number of sites overall “are going to be the hardest ones to reach,” said Inmaculada Hernandez, an assistant professor at the University of Pittsburgh School of Pharmacy and lead author of the analysis.

Certain vaccines could be better suited for such places, including Johnson & Johnson’s potential offering, which is a single shot, and health departments could distribute in rural areas through mobile units, she said. The company is expected to apply for FDA emergency authorization in February, Operation Warp Speed chief scientific adviser Moncef Slaoui said this month.

Until then, Pfizer and Moderna are the companies supplying doses for the country, and they’re not considered equal even though each is more than 90% effective at reducing disease.

In Wisconsin, the Moderna vaccine “gives us many more options” and “allows for us to get doses to those smaller clinics, more-rural clinics, in a way that reduces the number of logistics” needed for ultra-cold storage, Dr. Stephanie Schauer, the state’s immunization program manager, told reporters Wednesday.

Alan Morgan, head of the National Rural Health Association, echoed that the Moderna vaccine is being looked to as a “rural solution.” But he said states including Kansas have shown that a Pfizer rural rollout can be done.

“It’s where these states put a priority — either they prioritize rural or they don’t,” he said. “It’s a cautionary tale of what we may see this spring, of rural populations perhaps being second-tier when it comes to vaccination.”

Virginia, too, has a plan for getting the Pfizer vaccine to far-flung places. It’s shipping the vaccines to 18 health facilities with ultra-cold freezers across the state. The hubs are distributed widely enough so vaccinators can bring shots from there to health workers even in thinly populated areas before they spoil, said Brookie Crawford, spokesperson for the Virginia Department of Health’s central region.

Washington, on the other hand, allows hospitals without ultra-cold freezers to temporarily store Pfizer vaccines in the thermal boxes they arrive in, said Franji Mayes, spokesperson for the state’s health department. That means a box needs to be used quickly, before doses expire. “We are also working on a policy that will allow hospitals who don’t expect to vaccinate 975 people to transfer extra vaccine to other enrolled facilities,” she said. “This will reduce wasted vaccine.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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Novartis’ cholesterol drug inclisiran has hit a speed bump with the FDA, which has rejected the potential blockbuster because a manufacturing facility has yet to be inspected.

Matters appear to have been complicated by the pandemic and the travel disruption that it has caused, which is preventing an FDA team from visiting the plant in Europe.

In a statement the Swiss pharma said that the FDA has not raised any concerns about the safety or efficacy of the drug, which Novartis added to its pipeline after buying The Medicines Company for $9.7 billion late last year.

A first-in-class treatment discovered by Alnylam and then licensed to The Medicines Company, the drug is being developed for treatment for hyperlipidaemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy.

The FDA was due to make a regulatory decision on inclisiran by Wednesday but this has been delayed because of the unresolved “facility inspection-related conditions”.

Novartis said it will receive these requirements at the European manufacturing facility within 10 business days.

The FDA has not yet conducted a facility inspection and the FDA will set out a schedule if it deems one necessary, once safe travel may resume.

The European Commission has already approved the drug under the brand name Leqvio earlier this month following backing from the CHMP regulatory committee.

Inclisiran is a small interfering RNA (siRNA) therapy that works by inhibiting PCSK9 – the same target as Amgen’s Repatha (evolocumab) and Sanofi/Regeneron’s Repatha (alirocumab) – but is dosed only twice a year rather than every month.

Novartis hopes that the drug will make an impact where these antibody drugs have failed, with Repatha and Praluent lacking sales momentum despite being on the market for more than five years.

Payers had deemed the drugs to be too expensive and even price cuts failed to expand their use.

In January, Novartis and NHS England forged a pact to accelerate access to inclisiran for primary prevention of cardiovascular disease.

The post FDA rejects Novartis’ cholesterol drug after factory inspection issue appeared first on .

As expected, the FDA has moved swiftly ahead with emergency approval of Moderna’s COVID-19 vaccine after a positive assessment at its vaccines advisory committee.

The authorisation means that around six million more vaccine doses can now be rolled out in the US coronavirus immunisation programme, adding to the almost three million doses of Pfizer/BioNTech’s already-approved shot which started to be administered a few days ago.

The emergency use authorisation (EUA) for mRNA-1273 came the day after the FDA’s advisory panel voted 20-0 with one abstention that the benefits of the vaccine outweighed the risks for people aged 18 and over.

Moderna said delivery of supplies of mRNA-1273 will begin immediately, and reiterated its intention to provide 20 million doses to the US government out of its total order of more than 200 million before the end of this month.

The biotech now expects to have between 100 and 125 million doses available in the first quarter of 2021, of which 85 to 100 million have been claimed by the US.

On Friday meanwhile, the European Commission said it had exercised an option for an additional 80 million doses of Moderna’s product, taking its total order to 160 million doses. The UK has ordered seven million doses, Japan 50 million and South Korea 20 million.

The new supplies come as a quarter of a million new cases are being recorded each day in the US, the highest national rate in the world, with daily deaths averaging around 2,500 in the last couple of weeks.

Some US states re already saying they are unable to get access to promised supplies of Pfizer and BioNTech’s BNT-162b, as federal officials suggested manufacturing issues were at risk of holding up supplies of the shot – although that has been disputed by Pfizer. The US government’s objective is to have 100 million people vaccinated by April.

“It has been less than a year since the world first learned of SARS-CoV-2 and the terrible disease it can cause,” said Francis Collins, director of the US National Institutes of Health (NIH) which carried out clinical trials of mRNA-1273 and also contributed a technology used to stabilise the jab.

“To have not one but two safe and highly effective COVID-19 vaccines ready for deployment to the American public is truly a remarkable scientific achievement, and a significant step toward ending the pandemic that has caused so much suffering,” he added.

Moderna’s vaccine should be easier to distribute than the Pfizer/BioNTech shot as it requires temperatures of around -20 C for shipping – similar to a normal freezer – rather than -70 C.

The company has also updated its handling guide for the distribution for mRNA-1273 to include local transport under controlled conditions in a liquid state at 2-8 C, noting that “this important update eases the logistical burden of transporting the vaccine to more remote locations and ensures that the barriers to being vaccinated are lowered.”

The post Moderna’s COVID-19 vaccine is second for US after FDA green light appeared first on .

An in-depth look at twelve recently released COVID-19 vaccine management solutions as COVID-19 vaccines are being distributed nationwide.

1. Microsoft

Microsoft
launches a COVID-19 vaccine management platform with partners Accenture and
Avanade, EY, and Mazik Global to help government and healthcare customers
provide fair and equitable vaccine distribution, administration, and monitoring
of vaccine delivery.

Microsoft Consulting Services (MCS) has deployed over 230 emergency COVID-19 response missions globally since the pandemic began in March, including recent engagements to ensure the equitable, secure, and efficient distribution of the COVID-19 vaccine.

2. Accenture

Accenture recently rolled out a comprehensive vaccine management solution to help government and healthcare organizations rapidly and effectively plan and develop COVID-19 vaccination programs and related distribution and communication initiatives. Expanding on Accenture’s contact tracing capability that leverages Salesforce’s manual contact tracing solution, the platform is rapidly deployable and designed to securely track a resident’s vaccination journey, from registration and appointment scheduling to final vaccine administration and symptom follow-ups.

3. VigiLanz

VigiLanz, a clinical surveillance company launched their new mass vaccination support software, VigiLanz Vaccinate provides end-to-end management of the entire vaccination process, enabling hospitals to maximize the success of mass vaccination events for healthcare workers. VigiLanz Vaccinate streamlines vaccine administration and management by making it easy for staff to register and provide consent while automating workflows for program administrators. Its real-time insights into volume needs to reduce vaccine waste, while analytics give visibility into vaccination and immunity rates at the individual, department, hospital, and system-level.

4. BioIntelliSense

UCHealth recently deployed BioIntelliSense BioButton™ Vaccine
Monitoring Solution, an FDA-cleared medical-grade wearable for continuous
vital sign monitoring for up to 90-days (based on configuration) to healthcare
workers receiving COVID-19 vaccine UCHealth’s staff and providers will wear the
BioButton device for two days prior and seven days following a COVID-19 vaccine dose
to detect potential adverse vital sign trends. Together with a daily
vaccination health survey and data insights, the wearer may be alerted of signs
and symptoms to guide appropriate follow-up actions and further medical management.

5. VaxAtlas

VaxAtlas launches a
digital platform to support the COVID-19 vaccination process making it easy for
anyone to schedule and manage their vaccinations. Through a comprehensive suite
of on-demand tools, VaxAtlas manages the process of getting COVID vaccinations
from beginning to end. The platform provides access to a national certified
pharmacy network for local appointment scheduling, recall alerts, second dose
reminders, as well as QR clearance passes for vaccine validation. VaxAtlas
alleviates the complexity associated with vaccine logistics and helps to get
people back to work and back to living their lives.

6. DocASAP

DocASAP launches COVID-19
Vaccination Coordination Solution to help healthcare providers and payers meet
the urgent demand for vaccinating the nation. DocASAP’s COVID-19 Vaccination
Coordination Solution will help providers and payers guide people through the
vaccination process with pre-appointment engagement, online appointment scheduling
and reminders, and post-appointment wellness tracking. This will help reduce
the burden on staff and call centers to manage the sheer volume and complexity
of these appointments, and better coordinate the influx so providers can
effectively deliver the needed care. DocASAP will support the phased approach
to rolling out vaccinations, beginning with front-line healthcare staff.

7. Allied Identity

Allied Identity announced the launch of Vaxtrac, comprehensive vaccination management and credentialing platform designed to aid in the local, national and international response to COVID-19 and other communicable diseases. Vaxtrac uses SICPA’s proprietary CERTUS™ service in order to ensure the security of vaccination records and credentials.

8. Net Health

Net Health has developed a proprietary web-based Mobile Immunization Tracking platform to more efficiently manage on-site
immunizations. To ensure compliance, Net Health’s Mobile Immunization
Tracking platform tracks verification and enables employee consent forms to be
electronically recorded. Immunization data and the Vaccine Information Sheet
(VIS) are pulled directly from the Centers for Disease Control (CDC) database
and fields are auto-populated so clinicians do not have to manually enter data.
This ensures information in the employee record is accurate and saves time as
the clinician moves from one employee to the next.

9. Traction on Demand

Vancouver tech company, Traction on Demand,
has developed a COVID-19 Vaccine Clinic Accelerator. The accelerator helps
health authorities track all the critical details of their clinics including
type, location, staff members, and cold storage units available on-site and
applies CDC’s COVID-19 Temporary Clinic Best Practices to a
Salesforce-based mobile app, providing organizations with a digitized CDC
checklist, auditable clinic administration including a permanent auditable
record of all vaccination clinics an organization holds, critical risk
identification, and shift tracking.

10. MTX Group

MTX Group launches a
comprehensive end-to-end COVID-19 vaccine administration, management, and
distribution Solution for state and local public health agencies built on
Salesforce. The MTX vaccine management solution brings together the various
components of a COVID-19 vaccination program, including vaccine administration
and inventory management. MTX also works with public health departments to
identify necessary steps to promote vaccination adoption within a community.
The vaccine management solution is secure, portable, interoperable, and
provides data-driven vaccination program management capabilities.

11. Infosys

The Infosys
Vaccination Management (IVM) Salesforce Solution is an end-to-end offering
for automating tasks, integrating data sources, and delivering a seamless
vaccination program that offers supply chain visibility and future demand
forecasting. Disparate systems won’t work for this unprecedented health crisis.

12. Phresia

Phresia provides an end-to-end COVID-19 vaccine management solution for outreach, intake, reminder, and recall tools to increase vaccine uptake. Key features include communicating with patients about vaccine availability, send appointment reminders and boost recall, manage your waitlist, automate patient intake for vaccine visits, including consents, questionnaires, and patient education, and screen patients for vaccine hesitancy and maximize uptake by delivering personalized messaging based on those survey results.

The FDA is looking to quickly approve Moderna’s COVID-19 vaccine after it was unanimously backed by a panel of experts.

Yesterday’s advisory panel meeting voted 20-0 in favour of approving Moderna’s vaccine and although the vote is non-binding, you could probably bet your house on the FDA backing the shot as it rarely goes against the advice of its experts after such strong backing.

The vaccine was also given a glowing review by FDA staffers in a briefing document posted ahead of the meeting and looks set to become the second COVID-19 vaccine to hit the US market after Pfizer/BioNTech’s rival was approved last week.

Pfizer’s vaccine was approved within a day of a positive vote from the Vaccines and Related Biological Products Advisory Committee.

Roll-out of the Pfizer vaccine has already begun and FDA commissioner Stephen Hahn said the agency will work “rapidly” towards issuing an Emergency Use Authorization for Moderna’s shot.

The US has agreed to buy 200 million doses and there are six million doses ready move to ship as soon as the vaccine is officially approved.

Yesterday’s panel vote heard that the vaccine worked in 94% of cases, based on clinical data gathered so far, and is safe.

It is also slightly easier to move around – although it is an mRNA-based shot like Pfizer the storage temperature is around -20C, considerably less demanding than temperatures of around -75C required to maintain the integrity of its already-approved rival.

Moderna’s vaccine is also given in two shots, with the injections four weeks apart compared with the three-week interval required for Pfizer’s.

The UK has also pre-ordered seven million doses of the Moderna vaccine, which is also being reviewed by the country’s regulator the Medicines and Healthcare products Regulatory Agency (MHRA).

Canada also plans to get two million doses by March, part of a total 56 million doses ordered from Moderna.

The European Union has also announced a contract to buy 80 million doses and an option to buy 80 million more once the vaccine is formally approved.

The post FDA aims for fast approval of Moderna’s COVID-19 shot after panel vote appeared first on .

GlaxoSmithKline’s Benlysta has been on the market for almost a decade, but it still has some tricks up its sleeve – it’s just become the first and only FDA-approved treatment for lupus nephritis.

The US regulator has cleared both intravenous and subcutaneous formulations of Benlysta (belimumab) for the new indication, extending the use of the drug beyond its earlier label covering the treatment of active systemic lupus erythematosus (SLE) in combination with other medicines.

Originally approved in 2011 as an IV therapy for adults with SLE, a debilitating autoimmune disease, GSK got a green light for the subcutaneous formulation in 2017 and last year extended the label of the antibody to include paediatric patients aged over five.

Over that period sales have grown steadily to reach £514 million (almost $700 million) in the first nine months of 2020, boosted by its position as the only biologic approved to treat SLE. Approval in lupus nephritis is expected to push the product above the $1 billion threshold and into blockbuster sales territory.

Lupus nephritis is a severe form of SLE that can lead to late-stage renal failure and require dialysis or even a kidney replacement in the most severe cases. GSK estimates that around 60% of the 300,000-plus severe SLE patients in the US suffer from lupus nephritis each year, and a quarter of those develop end-stage renal disease.

The FDA approval comes on the back of the BLISS-LN trial results, which showed that 43% of patients on Benlysta hit the target of a significant improvement in kidney function, compared with 32% of those in the control arm.

GSK’s drug also improved secondary outcome measures including time to death or a kidney-related complication.

“What is interesting is more than 80% of eligible patients remain untreated with Benlysta in the US and of course even more around the world,” said GSK’s head of global pharma Luke Miels on the firm’s third-quarter results call.

“The number treated will increase further with the lupus nephritis indication and so there’s plenty of opportunity for growth,” he added.

GSK has been ramping up its manufacturing for Benlysta in anticipation of the new approval, including in-house capacity at its Rockville plant in Maryland, US, and through a contract manufacturing deal with Samsung Biologics.

GSK is also running a phase 3 combination study of Benlysta with rituximab, BLISS-BELIEVE, and the hope is that using the duo could drive the disease into clinical remission, according to Miels.

Both drugs work by targeting B cells, which are thought to be central to the disease process in SLE although the precise cause of the disease is unknown. Benlysta binds and neutralises the B cell survival factor BAFF, while rituximab targets CD20, a protein found on the surface of B cells.

The anti-CD20 mechanism could also lead to some competition for GSK in SLE and lupus nephritis. While Roche wasn’t able to show efficacy for its Rituxan brand of rituximab in trials, follow-up Gazyva (obinutuzumab) did better than placebo when added to standard care in the NOBILITY trial.

Another potential lupus nephritis rival is Aurinia Pharma’s voclosporin, which hit the mark last year in the phase 3 AURORA trial. AstraZeneca wasn’t so fortunate with its anifrolumab candidate, which flunked a pivotal trial in SLE in 2018, but still thinks it has potential.

The post GSK’s Benlysta claims first FDA okay for lupus kidney damage appeared first on .

While countries around the world continue to flounder with regards to COVID-19 testing, everyone is wondering how national governments could get things so horribly wrong. It’s true that governments have acted incompetently, leaders can make poor judgment calls, and optimistic testing targets are rarely achieved.

But the claims of various ambitious big pharma companies—about their ability to deliver on capacity and accuracy alike—have created a smokescreen for decision-makers, one that has severely undermined crucial efforts to monitor the spread. They say the hunt for profit leads to innovation, but it can also lead to chaos.

As Jonathan Quick, author of The End of Epidemics put it: “Virus biology and vaccines technology could be the limiting factors [to COVID-19], but politics and economics are far more likely to be the barrier to immunization.”

The time it takes for people to get tested and receive results (end-to-end) has been a significant point of contention in the testing sphere. Some solutions, especially in the pandemic’s infant stages, were keen to position themselves as the go-to protocol, particularly at a time when governments were frantically pouring money into testing, hoping it would give people the confidence to return to work and reopen parts of the economy.

In their quest to promote their own protocols in the best possible light, some companies have publicized processing times that don’t span the time it takes to actually complete the entire testing process. Others have shone a light on their own testing advantages. 

Disadvantages have not only been obscured but sometimes outright manipulated or fudged to appear more appealing and competitive to the average Joe, whose statistical interests lie more in Fantasy Football than the intricacies of testing methods. For instance, in our test comparison study, Abbott’s RealTime SARS-CoV-2 test took approximately 7 hours of end-to-end processing time. It’s the claim of 90-minute completion, however, had been widely publicized, leading several budding admirers, including the White House staff, to quickly commit to its protocol. This 90-minute-only accounted for one stage of its testing protocol. 

Testing companies have also recognized the value of promoting high-capacity protocols. Testing capacity ultimately boils down to the volume capabilities of each testing machine, versus the availability, cost, and a number of these machines required to complete a test.

When some companies promote their testing capacities, the figures produced don’t always mention the cost attached to each machine or the number of machines required for the full testing process. PCR tests tend to have low throughput, laborious process, and often false-negative results, making it overwhelmingly challenging to meet testing needs even in industrialized countries. But, as the world has come to know all too well, statistics can be massaged when presented in a preferred light.

Initially, testing companies prioritized promoting low-turnaround times and enhanced sensitivity. It was only after the preliminary stages of the pandemic when capacity became more of a trait to consider. This has led some test makers to highlight testing capacity in rather vague terms, presented as daily output as opposed to capacity per machine. As a result, certain companies utilizing PCR protocols have veered away from disclosing their throughput numbers. 

False negatives pose an enormous problem because they literally mean people who have COVID-19 will go out into the world in full confidence that they don’t have the disease, and infect others. Such people are less likely to self-isolate or even exercise very basic precautions, such as wearing a mask because they are convinced they aren’t contagious. 

In its own COVID-19 testing policy for labs and commercial manufacturers, the FDA says a diagnostic test should correctly identify at least 95 percent of positive samples. Yet, even 95 percent does not give us the scientific precision we might require to truly quell the spread. It gives us a probable threshold, but false negatives are still likely to leak through, causing further potential spread as a consequence. 

When the White House itself began screening its staff using a rapid coronavirus test made by Abbott Laboratories, they didn’t expect post-analysis studies to conclude the test may return a high percentage of false-negative results. In mid-May, the Food and Drug Administration issued a rare public warning about an Abbott Laboratories COVID-19 test: test results could be wrong. The lower 80 percent threshold for the Abbott and other point-of-care tests’ sensitivity proved insufficient. As such, governments fell into the trap of buying into solutions with plenty of initial hype, but less verifiable agency. 

For some of us, much of this information can seem completely contradictory, just like the old “Coffee is good for you… no, wait it’s bad for you… no, wait, it’s good for you again,” repeated ad nauseam. Understandably, media coverage of the COVID-19 pandemic has continued on full blast since the onset, this has inevitably led to a sense of informational fatigue for certain sections of society.

Tidal waves of medical statistics and information crash over us each and every day. During a global pandemic, this vulnerability to medical gossip only intensifies, meaning health authorities, as well as providers, should be re-doubling efforts to create clarity with regards to prevention; safety restrictions, testing protocols, and limitations. More clarity must be prioritized by competent legislators making vital decisions regarding institutional/national testing protocols.

More needs to be done to separate state and big pharma medicine, that’s because medical research is often financially backed by private entities. E.g. Antidepressants can be life-savers for some people. But drug company-funded studies have overplayed their benefits and downplayed their harms, contributing to overuse and unnecessary side effects.

It would be naive to think that financial gains are not a factor for budding testing companies, it would also be naive to expect that when testing companies recognize their own protocol shortcomings in the market, they will readily disclose it. Their aim is to move their product forward, not necessarily by deception, but also not always by full disclosure either. While this might be common ground for many industries, life-saving testing companies need to do better.

As Gerald Posner, author of Pharma: Greed, Lies, and the Poisoning of America, puts it: “Pharmaceutical companies view COVID-19 as a once-in-a-lifetime business opportunity,” adding that, “the worse the pandemic gets, the higher their eventual profit.” With potentially lucrative government contracts up for grabs, the clamor to present your testing solution as the answer to COVID-19 grew.

What many testing companies seem to have forgotten is that initiatives focusing on the protection of the public from a potentially terminal virus should, in no way, shape, or form, be employing misleading statistics and marketing tactics when the alternatives to effective prevention are so grave.

These companies carry a crucial responsibility: They are not simply providing the public with non-essential products for which you can choose to enjoy or not, the product here is medically crucial, and the fate of the health and livelihoods of millions of people across the globe depend on it.

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About Pawel Czech

Pawel has led market entry for three global tech companies into 45 countries. Pawel specializes in helping prepare companies to scale into multiple markets with a full stack offer. Pawel founded Nex.D to be the company he would have wanted to work with as a founder. Pawel has held three global roles in the technology sectors in the last 15 years working extensively in business development, operations, and sales capacity actively serving in a governance and leadership role with those founders and management teams.

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With the US and other major countries poised to begin national
distribution of multiple FDA-approved COVID-19 vaccines, the cybersecurity threats
to secure COVID19
vaccine distribution is imminent. Earlier this month, IBM released a report on malicious cyber actors targeting
the COVID-19 cold chain—an integral part of delivering and storing a vaccine at
safe temperatures.

Impersonating a biomedical company, cyber actors are sending phishing and spear-phishing emails to executives and global organizations involved in vaccine storage and transport to harvest account credentials. The emails have been posed as requests for quotations for participation in a vaccine program. In the report, IBM urges companies in the COVID-19 supply chain — from research of therapies, healthcare delivery to the distribution of a vaccine — to be vigilant and remain on high alert during this time

We recently sat down with Nigel Thorpe, Technical Director, SecureAge, an enterprise data security and encryption company to talk about the cybersecurity risks involved with COVID-19 vaccine distribution.

HITC: What type of information are hackers trying to
seize to disrupt the vaccine distribution process?

Thorpe: Hackers will try to obtain all the data they can muster, but specifically, they are looking for data around the distribution logistics together with details of the vaccine and its packaging. Using this they could attempt to replicate and profit from a counterfeit vaccine. In addition, cybercriminals are looking for all sorts of personal information about people involved in the vaccine distribution process, plus members of the public, so they can attempt identity theft and phishing attacks.

What are the dangers and implications if foreign actors
weaponize this information?

Thorpe: One of the biggest problems that already exist is an apprehensive public who is concerned with taking the vaccine because of fears that the approval process has been rushed and circumvented. These fears can be exploited by cybercriminals simply through the use of disinformation. In terms of cybersecurity, any attack on the distribution chain feeds into the fear of those already uncertain about the whole program.

In addition, bad actors could launch ransomware and spear-phishing attacks to get into the corporate network. Here, they can steal information concerning the “cold chain” and use this to build an illegal channel for counterfeit vaccine delivery. Not only would this result in unauthorized, unsafe vaccines being distributed but also reinforce fears of vaccines that many Americans already have. Any data, no matter how small or seemingly innocuous, could be used and exploited by cyber attackers.

How can health facilities remain protected?

Thorpe: The most important aspect is to ensure
that data is encrypted at all times so even if it is stolen, hackers won’t be
able to access this scrambled information. In addition, organizations should
make sure that unauthorized processes don’t run. This can be done by blocking
any application that attempts to execute, but which is not on an authorized
list. These measures will stop the problems of both phishing messages and data
theft – even by insiders.

What other information do you think hackers will target
in the future as we head into 2021?

Thorpe: Outside of exploiting the vaccine distribution network, hackers will attempt to capitalize on the continued remote working situation that is likely to last for most of 2021. Cybercriminals will try to exploit a situation where workers are not all using secure devices, resulting in data being stolen and exploited by bad actors.

In addition, we can expect combination attacks, where
something technical and something human will be combined in ways that the
confines and physical security of office spaces would have prevented. Notices
sent by mail to homes, phone calls, and possibly even personal visits by repair
technicians will be facilitated through stolen information and credentials
online, upping the ante of the scams and other illegal shenanigans.

Medical device design has been going through sweeping
changes over the last decade.  Ten years ago,
medical device companies weren’t concerned with delivering consumer-level
design: Devices that are both attractive and intuitively easy to use by a wide
variety of users.  Then the Affordable Care
Act was passed, and adherence and healthy behavior change became a
regulatory requirement.  

Our firm, which has been a long-time proponent of the
“consumerization” of medical product design, saw a steady uptick in business
based on our ability to deliver product experiences that a consumer expects
while also meeting regulatory requirements of the FDA.  And yet we still had to do a fair amount of
convincing to engineering teams about the importance of design that not only
works for physicians, but also makes life easier for caregivers and
patients. 

Our goal has always been to make design a priority for and
deliver great experiences to every voice in the ecosystem.  As tragic as COVID-19 has
been for millions of people, it has accelerated the consumerization of medical
device design:  the pandemic has
radically changed medical products for the better, forever. 

In the last six months, we have had many traditional device
companies and startups approach us to design COVID-19 testing products.  They want clinically effective medical
devices that are as easy to use as at-home pregnancy tests.  Companies are also coming to us with
non-COVID medical device ideas, and even the conversations around those
products have changed:  there is a
realization that medical devices must address a multi-layered audience. 

While all medical products must integrate the emotional,
physical and cognitive needs of the health consumer to create a holistic
experience, to really achieve consumer-level design companies need to go well
beyond human factors and useability studies and truly push the design
boundaries.  Medical device companies
that can’t integrate these four elements into their medical products are not
making scalable products, and will underachieve in today’s marketplace:

1. Improve Convenience:  Consumers today are accustomed to
convenience. We expect the world to operate at the speed of a Google search,
with the customizability of meal planning on sites like Plated or Blue Apron,
and the responsiveness of booking a ride on Uber. Healthcare rarely works this
way so a medical device must integrate it into the design.  In the medical world, the laws of consumer
design also apply:  with Axonics
Modulation Technology system, we
transformed an innovative technology into a complete ecosystem of physical
and digital products that improve the experience and work hard in the background
to return normal daily lives to people suffering from incontinence.

2. Aim to Delight: 
Creating delight can transform an experience and build relationships
that keep customers engaged with your brand. 
Most medical solution providers look at users in terms of physical and
cognitive usability. But this is only the beginning. We believe there are four
additional dimensions that will help companies develop a qualitative
understanding of health consumers and their motivations—emotional, social,
contextual, and developmental. Exploring these dimensions at the front end of
the product development process will reveal what patients need and desire from
a health experience and enable companies to respond with meaningful innovation
that gains adoption and changes health outcomes.  We use these motivations to create delight in
the medical device.

3. Provide Personalized Experiences: Personalization
is a growing trend in the consumer product world, and it needs to become one in
healthcare. Those at the forefront are using data to make predictions that
anticipate customers’ needs and desires. Entertainment platforms, like Netflix,
make recommendations that introduce users to new content based on their
previous consumption. Virgin America’s in-flight screens address their
passengers by name and provide personalized information about their itinerary,
in addition to personalized dining and entertainment recommendations. In
healthcare, targeted, personal experiences can be a tipping point to meaningful
behavior change. Information has the power to engage health consumers in
moments where their decisions have a direct impact on their health and
wellbeing. With a majority of people carrying or wearing smart devices, it’s
possible to have continuous data about their location. This data can be used to
generate relevant, real-time recommendations. 
With COVID-19 or any future pandemic, real-time information can save
countless lives.

4. Be Emotional: 
The goal of consumer-driven product innovation is to create an emotional
connection between users and brands—a delightful experience or perception that
keeps people coming back. This is an important goal in healthcare as well, as
more complex factors start to influence choice, and continued engagement plays
a growing role in health outcomes. Although the medical product development
process is more burdened by engineering, technology, and regulation, medical
solution providers can adopt some best practices from consumer companies to
help their products connect. Consumer giants apply numerous resources toward
developing a deep understanding of their user. To capture health consumers’
interest and loyalty, it’s necessary for medical device makers to develop a
knowledge that goes deeper than a medical record or hospital survey. This
holistic understanding of consumers and their health journeys will breed
empathy—something that only comes from first-hand emotional transactions—and
help companies uncover many opportunities for meaningful innovation and
differentiation.

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About Stuart Karten

Stuart Karten is the principal of Karten Design, a
product innovation consultancy creating positive experiences between people and
products specializing in health technology. 

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Novartis’ Entresto is on course to become the first drug to be approved in the US for a form of heart failure that is notoriously hard to treat effectively, despite missing the mark in a phase 3 trial.

An FDA advisory committee 12 to 1 in favour of approving Entresto (sacubitril/valsartan) for heart failure with preserved ejection fraction (HFpEF), which accounts for around half of all heart failure cases but proves highly resistant to drug treatment. In HFpEF, the heart muscles pump normally but the organ is too stiff to fill properly.

Entresto is already approved to treat heart failure with reduced ejection fraction (HFrEF), caused by the heart muscles not pumping effectively, and has revitalised the treatment of patients with this form since its launch in 2015.

After a slow start, it has grown to become a $1.7 billion product last year, and that represented a surge from around $1 billion in 2018 revenues.

Analysts have predicted that approval in HFpEF – which affects around 3 million people in the US alone – could more than double Entresto’s sales, perhaps driving them as a high as $5 billion a year. There’s also plenty of upside in HFrEF as three out of four eligible patients are still not being treated with the drug, according to Novartis.

The prospect of adding HFpEF to Entresto’s label looked shaky last year however, when the drug missed its primary objective in the phase 3 PARAGON-HF trial.

The 4,822-patient study missed statistical significance for a composite primary endpoint of reducing cardiovascular death and total heart failure hospitalisations by 13% compared with valsartan alone, but only by a whisker, and Novartis has been upbeat since about the chances of approval.

The published data from the study suggested that the drug performed better in women, people with structural abnormalities in the left ventricles of their hearts, and those with very low ejection fractions – the amount of blood pushed out of the heart each beat.

The positive vote by FDA advisors came after the FDA reviewer acknowledged the narrow miss for statistical significance and pointed to the pressing need for a drug treatment for HFpEF.

The agency’s own expert said that “various pre-specified and post-hoc analyses suggest that sacubitril/valsartan compared to valsartan reduces HF events” in HFpEF, and of course Entresto’s long track record of safety stands in its favour.

While the FDA doesn’t have to follow its advisory committee’s advice it generally does, and Novartis is now eyeing approval of Entresto in HFpEF in the first quarter of 2021.

The main question now is exactly how the FDA will word the label if it approves the drug, with panellists debating the use of ejection fractions percentages to guide treatment with little agreement.

PARAGON-HF in included patients with left ventricular ejection fraction (LVEF) of 45% or more, but earlier studies have suggested the drug can have a benefit in people with scores below 40%.

The post Entresto set for big sales hike after FDA panel endorsement appeared first on .

Moderna looks odds on to claim emergency use authorisation from the FDA for its COVID-19 vaccine this week, after the regulator published a report endorsing its safety and rating its efficacy at 94.5%.

The document has been published just after the US started the rollout of Pfizer and BioNTech’s vaccine after it got an emergency green light last week, and ahead of an expert panel due to consider Moderna’s shot on Thursday.

If the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) agrees with the agency’s assessment it could be available before the end of the week, accelerating the pandemic immunisation programme.

Like Pfizer/BioNTech’s BNT-162b, Moderna’s mRNA-1273 is based on messenger RNA coding for the SARS-CoV-2 spike protein, and will be administered in a two-dose regimen given a few weeks apart.

The FDA says no specific safety concerns with mRNA-1273 have been seen in the 30,400-patient COVE trial that underpins the EUA application, with minor effects like fever, headache and fatigue common but manageable, a serious side effects rare.

The overall 94.5% efficacy rating two weeks after the second dose is in line with interim data from the trial, although the FDA notes that it seems to be less effective in older people.

For the 18 to 64 age group efficacy comes in at 96%, but drops to 86% in the over-65s – both values are  way above the threshold that should be needed to support emergency use during the pandemic.

The shot also worked equally well in white, black and Hispanic subjects, men and women, and those with conditions like obesity and diabetes that increase the risk of severe COVID-19.

Importantly, there was also some preliminary data pointing to a reduction in asymptomatic SARS-CoV-2 infections – something that hasn’t yet been demonstrated with other vaccines – as well as prevention of severe disease.

All told, 38 trial participants in the placebo arm of the trial tested positive for asymptomatic COVID-19 at the time of their second dose, well above the 14 positives in the mRNA-1273 arm.

If the VRBPAC votes in favour of mRNA-1273 shipments are expected to begin within 24 hours, and Moderna has said it expects to be able to provide up to 6 million doses in the initial rollout, adding to around 3 million doses of the Pfizer/BioNTech jab.

The first doses of BNT-162b are being used to treat healthcare workers and elderly people in care homes, and it will be many months before vaccinations are available for all America’s 330 million population. Both vaccines will be provided free of charge to recipients.

The federal government has already signed supply agreements with Moderna and Pfizer/BioNTech for 300 million doses in 2021, enough to dose 150 million people. mRNA-1273 requires less intensive refrigeration that BNT-162b, so could be more suitable for distribution to more remote areas of the US.

There is also hope that vaccines from Johnson & Johnson and AstraZeneca could also be available in the first quarter of 2021.

The post FDA backs Moderna COVID-19 shot ahead of emergency use vote appeared first on .

The FDA authorized an at-home antigen test for Covid-19 developed by Ellume. It does not require a prescription.

Brown rice contains more arsenic than white rice, but the arsenic in brown rice is less absorbable, so how does it wash out when you compare the urine arsenic levels of white-rice eaters to brown-rice eaters?

Arsenic in rice is a cause for concern, according to a consensus statement by the European and North American societies for pediatric nutrition. At the very least, “in areas of the world where rice consumption is high in all ages, authorities should be prompted to declare which of the rice [types] have the lowest arsenic content and are, therefore, the least harmful for use during infancy and childhood.” I look into the arsenic content of different rices in my video Which Rice Has Less Arsenic: Black, Brown, Red, White, or Wild?.

Extensive recent testing by the FDA found that long grain white rice, which is what most people eat, appears to have more arsenic than medium or short grain rice, but this may be because most of the shorter grains are produced in California, which has significantly less contaminated rice paddies than those in the South, such as in Texas or Arkansas, where most of the long grain rice is grown. So, it’s less long grain versus short grain than white rice versus brown rice, as the mean concentration of inorganic arsenic in parts per billion of long grain white rice is 102.0 and 156.5 in short, medium, and long grain brown rice, as you can see at 0:54 in my video.

What about some of the naturally pigmented varieties like red rice or black rice, which may be even healthier than brown? As you can see at 1:08 in my video, they may contain even less arsenic than white rice. One sample of black rice from China that was purchased in Kuwait had higher levels for total arsenic, so the toxic inorganic portion may only be half that, putting it on par with U.S. brown rice. The study’s red rice sample from Sri Lanka was even more extraordinary, with less than a fifth of the arsenic of the Chinese black rice. But, the Sri Lankan red rice sample had a ridiculous high amount of cadmium, evidently attributed to the cadmium content of widely used Sri Lankan fertilizers.

Colored rice samples purchased mostly in the United States were better than brown or white, and a dozen samples of red rice purchased in Europe were as bad, or even worse, as brown rice. I was hoping that wild rice would have little or no arsenic because it’s a totally different plant, but an average of eight samples showed it to be nearly comparable to white, though the wild rice samples contained only half as much toxic arsenic as brown rice.

As you can see at 2:06 in my video, the arsenic found in a daily serving of white rice carries 136 times the acceptable cancer risk, but brown rice is even riskier at 162. Brown rice averages two-thirds more toxic arsenic than white rice. But, is that just because brown rice tends to be a different strain or grown in different places? No. If you take the exact same batch of brown rice and measure the arsenic levels before and after polishing it to white, you do get a significant drop in arsenic content.

It’s not what you eat, though. It’s what you absorb. The arsenic in brown rice appears to be less bioavailable than the arsenic in white rice. The texture of brown rice may cut down on the release of arsenic from the grain, or perhaps the bran in brown rice helps bind it up. Regardless, taking bioavailability into account, the difference in arsenic levels in white versus brown rice may be a third more, rather than 70 percent more, as you can see at 2:57 in my video. This estimate, however, was based on an in vitro gastrointestinal fluid system in which researchers strung together beakers and tubes to mimic our gut, with one flask containing stomach acid and another intestinal juices. What happened when it was tested in humans? Yes, “evidence suggests that brown rice may contain more arsenic than white rice,” but the researchers aimed to determine how much is actually absorbed by measuring the urine levels of arsenic in white-rice eaters compared with brown-rice eaters. For the arsenic to get from the rice into your bladder, it has to be absorbed through your gut into your bloodstream.

As you can see at 3:45 in my video, the urine of thousands of American test subjects who don’t eat rice at all still contains about 8 micrograms of toxic, carcinogenic arsenic a day. It’s in the air, it’s in the water, and there’s a little bit in nearly all foods. But, eat just one food—a cup or more of white rice a day—and your arsenic exposure shoots up by 65 percent to about 13 micrograms a day.

What about those who eat a cup or more of brown rice every day, which technically contains even more arsenic? Their exposure shoots up the same 65 percent. There is no difference between the urine arsenic levels of white-rice eaters compared with brown-rice eaters. However, this was not an interventional study in which they fed people the same amount of rice to see what happened, which would have been ideal. Instead, it was a population study, so maybe the reason the levels are the same is that white-rice eaters eat more rice than do brown-rice eaters. Could that be why they ended up with the same levels? We don’t know, but it should help to put the minds of brown-rice eaters to rest. But would it be better to eat no rice at all? That’s what I’ll explore in my next few blogs.

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 If you’re just joining in on this topic, check out these lead-up videos:

• Where Does the Arsenic in Chicken Come From?
• Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?
• The Effects of Too Much Arsenic in the Diet
• Cancer Risk from Arsenic in Rice and Seaweed

 

It seems like each of these videos just raises more questions, but don’t worry because I’ve got answers for you. See:

• Which Brands and Sources of Rice Have the Least Arsenic?
• How to Cook Rice to Lower Arsenic Levels
• Arsenic in Infant Rice Cereal
• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup
• How Risky Is the Arsenic in Rice?
• How Much Arsenic in Rice Is Too Much?
• Is White Rice a Yellow-Light or Red-Light Food?
• Do the Pros of Brown Rice Outweigh the Cons of Arsenic?
• Benefits of Turmeric for Arsenic Exposure

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

 

Si existe una cita con el destino, está escrita en el calendario del doctor Taison Bell.

Al mediodía del martes 15 de diciembre, Bell, especialista en cuidados intensivos del Sistema de Salud de la Universidad de Virginia será uno de los primeros en arremangarse para recibir la vacuna que lo protegerá del coronavirus.

Bell, de 37 años, se inscribió la semana pasada a través del correo electrónico del hospital para recibir la vacuna. “La historia de esta crisis es que cada semana se siente como un año. Esta es realmente la primera vez que hay una esperanza genuina de que podemos revertir esta situación”.

Por ahora, esa esperanza se limita a unos pocos elegidos. Bell atiende a algunos de los pacientes con COVID-19 más enfermos en el hospital UVA Health en Charlottesville, Virginia.

Bell es uno de los 12,000 trabajadores del hospital “que trabajan directo con estos pacientes”, que podrían ser elegibles para unas 3,000 primeras dosis de vacunas, dijo el doctor Costi Sifri, director de epidemiología del hospital.

“Estamos tratando de encontrar las categorías de mayor riesgo, aquellas que realmente pasan una cantidad significativa de tiempo cuidando a los pacientes”, dijo Sifri. “No se tiene en cuenta a todo el mundo”.

Incluso cuando la Administración de Alimentos y Medicamentos (FDA) participaba en intensas deliberaciones antes de la autorización del viernes de la vacuna contra COVID de Pfizer y BioNTech, y días antes de que se liberaran las 6.4 millones de dosis iniciales, los hospitales de todo el país ya estaban planeando cómo distribuir la primeras, y escasas, dosis.

Un comité asesor de los Centros para el Control y Prevención de Enfermedades (CDC) recomendó que la máxima prioridad sea para los hogares de adultos mayores de atención a largo plazo y para los trabajadores de atención médica de primera línea.

Pero se sabía que la primera tanda de vacunas no iba a cubrir toda la necesidad y que se iba a tener que hacer un proceso más selectivo, incluso entre los trabajadores críticos del hospital.

En general, se aconseja a los hospitales que cubran a los miembros de su fuerza laboral con mayor riesgo, pero las instituciones deben decidir exactamente quiénes serán, dijo Colin Milligan, vocero de la Asociación Estadounidense de Hospitales, en un correo electrónico.

“Está claro que los hospitales no recibirán lo suficiente en las primeras semanas para vacunar a todos los miembros de su personal, por lo que hubo que tomar decisiones”, escribió Milligan.

En Intermountain Healthcare, en Salt Lake City, Utah, las primeras inyecciones serán para los miembros del personal “con el mayor riesgo de contacto con pacientes COVID positivos o sus desechos”, dijo la doctora Kristin Dascomb, directora médica de prevención de infecciones y salud del personal. Dentro de ese grupo, los gerentes determinarán qué cuidadores son los primeros en la fila.

En la UW Medicine, en Seattle, Washington, que incluye el Harborview Medical Center, un plan temprano requería que el personal de alto riesgo fuera seleccionado al azar para recibir las primeras dosis, dijo la doctora Shireesha Dhanireddy, directora médica de la clínica de enfermedades infecciosas.

Pero el sistema hospitalario de la Universidad de Washington espera recibir dosis suficientes para vacunar a todas las personas en ese nivel de alto riesgo dentro de dos semanas, por lo que la selección aleatoria no ha sido necesaria por ahora.

“Permitimos que las mismas personas programen la cita”, dijo Dhanireddy, y alentamos al personal a vacunarse cerca del final de sus semanas laborales en caso de que tengan reacciones a la nueva vacuna.

Los resultados de los ensayos han demostrado que las inyecciones con frecuencia producen efectos secundarios que, aunque no debilitantes, podrían causar síntomas como fiebre, dolores musculares o fatiga que podrían mantener a alguien en casa por uno o dos días.

“Queremos asegurarnos de que no todo el mundo reciba la vacuna el mismo día para que, si hay algunos efectos secundarios, no acabemos quedando cortos de personal”, dijo Sifri, de UVA Health, y señaló que las directrices exigen que no más del 25% de cualquier unidad se vacune a la vez.

En UVA Health, una vez que se distribuyan las 3,000 dosis iniciales, el hospital planea confiar en lo que Sifri describió como “un código de honor muy estricto” para permitir que los miembros del personal decidan qué lugar ocupar en la fila. Se les ha pedido que consideren factores profesionales, como el tipo de trabajo que realizan, así como riesgos personales: la edad o afecciones subyacentes como la diabetes.

“Vamos a pedirles a los miembros del equipo, utilizando el código de honor, que determinen cuál es su riesgo de COVID y si necesitan tener una cita temprana para la vacuna o una fecha posterior”, explicó.

Se elaboró este plan después que el personal de atención médica rechazara rotundamente otras opciones. Por ejemplo, pocos favorecieron una propuesta para asignar dosis a través de una lotería, como el caótico sistema basado en la fecha de cumpleaños de la película “Contagion”, sobre una horrible pandemia.

Funcionarios del hospital también enfatizaron que están tratando de diseñar planes de distribución que garanticen que las vacunas se asignen de manera equitativa entre los trabajadores de salud, incluidos los grupos sociales, raciales y étnicos que han sido perjudicados de manera desproporcionada por COVID-19. Eso requiere pensar más allá de los médicos y enfermeras de primera línea.

Por ejemplo, en UVA Health, uno de los primeros grupos invitados a vacunarse será el de 17 trabajadores cuya tarea es limpiar cuartos en la unidad de patógenos especiales donde se tratan los casos graves de COVID.

“Reconocemos que todo el mundo está en riesgo de contraer COVID, todo el mundo merece una vacuna”, dijo Sifri.

En muchos casos, quedará claro quién debe ir primero. Por ejemplo, aunque Dhanireddy es doctora especialista en enfermedades infecciosas que consulta sobre casos de COVID, está feliz de esperar. “No me pondría en el primer grupo en absoluto”, dijo. “Creo que tenemos que proteger a nuestro personal que realmente está ahí con ellos la mayor parte del día, y esa no soy yo”.

Para algunos trabajadores de salud, no ser el primero en la fila para la vacunación está bien. Debido a que la vacuna inicialmente fue autorizada solo para uso de emergencia, los hospitales no requerirán que los empleados sean vacunados como parte de esta primera ronda. Entre el 70% y el 75% del personal de atención médica de UVA Health e Intermountain Health aceptaría una vacuna COVID, mostraron encuestas internas. El resto no está seguro o no está dispuesto.

“Hay algunos que aceptarán de inmediato y otros querrán observar y esperar”, dijo Dascomb.

Aún así, autoridades del hospital dicen que confían en que aquellos que quieran la vacuna no tengan que esperar mucho. Dosis suficientes para aproximadamente 21 millones del personal de atención médica deberían estar disponibles a principios de enero, según funcionarios de los CDC.

Bell, el médico de cuidados intensivos, dijo que está agradecido de estar entre los primeros en recibir la vacuna, especialmente después que sus padres, que viven en Boston, contrajeran COVID-19. Publicó sobre su próxima cita en Twitter y dijo que otros trabajadores de salud que se encuentran entre los primeros en la fila deberían hacer público el proceso.

“Serviremos como ejemplo de que esta es una vacuna segura y eficaz”, dijo. “La estamos dejando entrar en nuestros cuerpos. Deberías dejar que entre en el tuyo también”.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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This story also ran on NBC News. It can be republished for free.

If there’s such a thing as a date with destiny, it’s marked on Dr. Taison Bell’s calendar.

At noon Tuesday, Bell, a critical care physician, is scheduled to be one of the first health care workers at the University of Virginia Health System to roll up his sleeve for a shot to ward off the coronavirus.

“This is a long time coming,” said Bell, 37, who signed up via hospital email last week. “The story of this crisis is that each week feels like a year. This is really the first time that there’s genuine hope that we can turn the corner on this.”

For now, that hope is limited to a chosen few. Bell provides direct care to some of the sickest COVID-19 patients at the UVA Health hospital in Charlottesville, Virginia. But he is among some 12,000 “patient-facing” workers at his hospital who could be eligible for about 3,000 early doses of vaccine, said Dr. Costi Sifri, director of hospital epidemiology.

“We’re trying to come up with the highest-risk categories, those who really spend a significant amount of time taking care of patients,” Sifri said. “It doesn’t account for everybody.”

Even as the federal Food and Drug Administration engaged in intense deliberations ahead of Friday’s authorization of the Pfizer and BioNTech COVID vaccine, and days before the initial 6.4 million doses were to be released, hospitals across the country have been grappling with how to distribute the first scarce shots.

An advisory committee of the Centers for Disease Control and Prevention has recommended that top priority go to long-term care facilities and front-line health care workers, but the early allocation was always expected to fall far short of the need and require selective screening even among critical hospital workers.

Hospitals in general are advised to target the members of their workforce at highest risk, but the institutions are left on their own to decide exactly who that will be, Colin Milligan, a spokesperson for the American Hospital Association, said in an email.

“It is clear that the hospitals will not receive enough in the first weeks to vaccinate everyone on their staff, so decisions had to be made,” Milligan wrote.

At Intermountain Healthcare in Salt Lake City, the first shots will go to staff members “with the highest risk of contact with COVID-positive patients or their waste,” said Dr. Kristin Dascomb, medical director of infection prevention and employee health. Within that group, managers will determine which caregivers are first in line.

At UW Medicine in Seattle, which includes Harborview Medical Center, one early plan called for high-risk staff to be selected randomly to receive first doses, said Dr. Shireesha Dhanireddy, medical director of the infectious disease clinic. But the University of Washington hospital system expects to receive enough doses to vaccinate everyone in that high-risk tier within two weeks, so randomization isn’t necessary — for now.

“We are allowing people to schedule themselves,” Dhanireddy said, and encouraging staffers to be vaccinated near the end of their workweeks in case they have reactions to the new vaccine.

Trial results have shown the shots frequently produce side effects that, while not debilitating, could cause symptoms such as fever, muscle aches or fatigue that might keep someone home for a day or two.

“We want to make sure that not everybody has the vaccine on the same day so that if there are some side effects, we don’t end up being short-staffed,” said Sifri, of UVA Health, noting that guidelines call for no more than 25% of any unit to be vaccinated at once.

At UVA Health, once the initial 3,000 doses are distributed, the hospital plans to rely on what Sifri described as “a very strong honor code” to allow staff members to decide where they should be in line. They’ve been asked to consider professional factors, like the type of work they do, as well as personal risks, such as age or underlying conditions like diabetes.

“We’re going to ask team members, using the honor code, to determine what their risk is for COVID and to determine whether they need to have an early vaccine sign-up time or a later vaccine sign-up time,” he said.

That plan was chosen after health care staff members soundly rejected other options. For instance, few favored a proposal to allocate dosages via a lottery, like the chaotic birthday-based system depicted in the 2011 pandemic horror film “Contagion.” “That was the biggest loser,” he said.

Hospital officials also stressed they are trying to devise distribution plans that ensure vaccines are allocated equitably among health care workers, including the social, racial and ethnic groups that have been disproportionately harmed by COVID-19 infections. That requires thinking beyond front-line doctors and nurses.

At UVA Health, for example, one of the first groups invited to get shots will be 17 workers whose job is to clean rooms in the special pathogens unit where severe COVID cases are treated.

“We acknowledge that everybody is at risk for COVID, everybody is deserving of a vaccine,” Sifri said.

In many cases, it will be clear who should go first. For instance, although Dhanireddy is an infectious disease doctor who consults on COVID cases, she is happy to wait to be vaccinated. “I wouldn’t put myself in the first group at all,” she said. “I think that we need to protect our staff that are really right there with them most of the day — and that’s not me.”

But hospitals must remain vigilant about relying on workers to prioritize their own access, Dhanireddy cautioned. “Sometimes, self-selection works more for self-advocacy,” she said. “It’s great that some individuals say they would defer to others, but sometimes that’s not actually the case.”

For some health care workers, not being first in line for vaccination is fine. Because the vaccine initially has been authorized only for emergency use, hospitals won’t require employees to be inoculated as part of this first round. Between 70% and 75% of health care staff at UVA Health and Intermountain Health would accept a COVID vaccine, internal surveys showed. The rest are unsure — or unwilling.

“There are some that will be immediate acceptors and some who will want to watch and wait,” Dascomb said.

Still, hospital officials say they’re confident that those who want the vaccine won’t have to wait long. Enough doses for roughly 21 million health care personnel should be available by early January, according to CDC officials.

Bell, the critical care doctor, said he’s grateful to be among the first to receive the vaccine, especially after his parents, who live in Boston, both contracted COVID-19. He has posted about his upcoming appointment on Twitter and said he and other health care workers who are among the first in line should be public about the process.

“We’ll serve as an example that this is a safe and effective vaccine,” he said. “We’re letting it go into our bodies. You should let it go into yours, too.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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The vaccine was developed in record time, a big scientific accomplishment, even though the agency’s move follows an unusual level of pressure from President Trump’s White House, renewing concerns that politics drove the vaccine process even as the first doses are shipped.

The nonbinding recommendation is a final hurdle before an official emergency use authorization for the vaccine, which could be the first shot approved for use in the U.S. Distribution, however, will remain a challenge.

Arsenic levels were tested in 5,800 rice samples from 25 countries.The arsenic found in five servings of rice a week poses a hundred times the acceptable cancer risk. What did the rice industry have to say about that? When the story first broke in the media that U.S. rice had some of the highest arsenic levels in the world, the USA Rice Federation said, “Enough nonsense about arsenic already!” in the August 9, 2005, issue of USA Rice Daily, its daily newsletter. The study, in its mind, was “not only inaccurate in the highest degree, but also maliciously untrue.” One of the researchers responded, “By not addressing this problem [of arsenic] that has been ignored for decades, the U.S. cotton-belt rice industry is doing itself an injustice. “Had the problem been addressed in the past, given that it is well known that arsenic in paddy soils was a problem in the U.S….safe soils would have been identified and low grain arsenic rice varieties developed.” Instead, arsenic-resistant varieties have been developed that build up excessive levels of arsenic without dying themselves. I discuss arsenic levels in rice in my video Which Brands and Sources of Rice Have the Least Arsenic?.

Not all rice producers have been so dismissive, though. After a subsequent Consumer Reports exposé, one rice company detailed “how it is taking matters into its own hands.” Lundberg Farms started testing hundreds of samples of its rice to share the results with the FDA. “We’re committed to providing safe food,” said the CEO, “to really listening to our consumers, and dealing with this problem very openly….” Lundberg Farms isn’t just sharing its results with the FDA, but with everyone.

If you visit its website or go to 1:37 in my video, you can see it apparently followed through on its testing promise for its brown rice. Lundberg Farms use parts per million (ppm) instead of parts per billion (ppb) to make it look better than it is, but compared with the average U.S. brown rice level of 154 ppb, Lundberg does do better. In fact, at 80 ppb, its aromatic brown rice, presumably its brown basmati and brown jasmine, averages less than national white rice levels, as do, apparently, Lundberg’s red and black rices, at 90 ppb. In fact, none of its samples even reached the average U.S. brown rice level.

Consumer Reports found most other brands to be pretty comparable to the U.S. average arsenic levels in brown rice, as you can see at 2:15 in my video, including Uncle Ben’s and Walmart’s Great Value brand. Whole Foods, however, scored the worst with its 365 Everyday Value long grain brown rice, about a third higher than these others and exceeding the national average.

In the largest review to date, based on 5,800 rice samples from 25 countries, the highest total arsenic average came from the United States. U.S. studies averaged overall about double that of rice out of Asia, with the high levels in the United States blamed on “the heavy [historical] use of arsenic-based pesticides.” But arsenic levels were not the same across the United States. Yes, U.S. rice averages twice the arsenic of Asian rice and nearly all rice samples tested in upstate New York that were imported from India or Pakistan had arsenic levels lower than 95 percent of domestically produced rice. But, “[r]ice grown in the U.S. showed the widest overall range…and the largest number of outliers,” due primarily to where it was grown, as you can see at 3:01 in my video. There is significantly more arsenic in Texas and Arkansas rice than rice from California. California rice is comparable to rice produced around the rest of the world. These are presumably some of the data that led Consumer Reports to suggest brown basmati from California, India, or Pakistan might be among the safer rice choices.If the arsenic is from pesticides, would organic rice have less than conventionally grown rice? No, because arsenic pesticides were banned about 30 years ago. It’s just that 30,000 tons of arsenic chemicals had already been dumped onto cotton fields in the southern United States, “so it is understandable that arsenic residues still remain in the environment” even if you don’t add an ounce of new pesticides. That’s why the industry specifically selects for arsenic-resistant varieties of rice plants in the South. If only there were arsenic-resistant humans.

What about other brands of rice? That was the subject of Which Rice Has Less Arsenic: Black, Brown, Red, White, or Wild?.

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For even more background, see:

• Where Does the Arsenic in Chicken Come From?
• Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?
• The Effects of Too Much Arsenic in the Diet
• Cancer Risk from Arsenic in Rice and Seaweed

 You may also be interested in:

• How to Cook Rice to Lower Arsenic Levels
• Arsenic in Infant Rice Cereal
• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup
• How Risky Is the Arsenic in Rice?
• How Much Arsenic in Rice Is Too Much?
• Is White Rice a Yellow-Light or Red-Light Food?
• Do the Pros of Brown Rice Outweigh the Cons of Arsenic?
• Benefits of Turmeric for Arsenic Exposure

Kudos to Consumers Union, the wonderful organization that publishes Consumer Reports, for its pioneering work on this and so many other topics.

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

 

What You Should Know:

– LA-based Scanwell Health announced today that its at-home UTI test kits – featuring the first FDA-cleared urinalysis app that lets people test for a UTI without having to visit a lab or doctor – is now available on Amazon.com.

– The kits are priced at 3 for $15. Happy to send a graphic or answer any questions you have. Until now, the kits have been available only directly through the Scanwell Health web site.

– Scanwell enables clinical-grade testing, instant results, and professional guidance for comprehensive healthcare from the safety of your home. Once people have their Scanwell test results (which takes two minutes), they can receive a prescription by connecting directly to telehealth providers via their smartphone for treatment. The whole process can take place from the safety of their home and they do not have to visit a doctor’s office.

– The company is also working on tests for chronic kidney disease, malaria, and the virus that causes COVID-19.

What You Should Know:

– Lightspeed Venture Partners, the VC behind Nest and GrubHub, is leading a $10 million round for Freespira, an FDA-cleared digital therapeutic proven to significantly reduce or eliminate panic attacks and PTSD symptoms by training users to normalize respiratory irregularities.

– In 28 days, Freespira can reduce or eliminate panic
attacks and PTSD symptoms from home with just a tablet, sensor, and custom app.
There’s no medicine with possible side effects and no need to see a doctor or
therapist in person.

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Freespira, Inc.(formerly
Palo Alto Health Sciences, Inc.), a Kirkland, WA-based maker of the first
FDA-cleared digital therapeutic that significantly reduces or eliminates
symptoms of panic attacks, panic disorder and post-traumatic stress disorder
(PTSD) in only 28 days, announced it has completed a $10 million capital raise led
by Lightspeed Venture Partners. Joining
the financing round, the largest in the company’s history, were previous
investors Aphelion Capital, Medvest Capital, and Freespira Chairman,
Russell Siegelman.

Free from Panic Attacks & PTSD in 28 Days

Founded in 2013, Freespira® is the only FDA-cleared digital therapeutic proven
to significantly reduce or eliminate Panic Disorder and PTSD symptoms by
training users to normalize respiratory irregularities in just 28 days. This
4-week medication-free program can be done from the comfort of your home for 17
minutes, twice daily. Treatment is authorized and completed under the
supervision of a licensed healthcare provider and is clinically proven to
reduce or eliminate panic attacks and other symptoms of panic disorder. Freespira
uses a custom sensor to train patients to stabilize their respiration rate and
exhaled carbon dioxide levels, thereby reducing or eliminating panic attacks
and PTSD symptoms.

Recent Peer-Reviewed Studies

Numerous peer-reviewed studies have demonstrated the
clinical effectiveness and cost savings of the Freespira solution, including:

– A clinical trial conducted at the VA Palo Alto Health Care
System in Palo Alto, Calif. demonstrated the efficacy of Freespira for veterans
and non-veterans suffering from PTSD. Significant reductions in measures of
PTSD severity were achieved by 85% of subjects post-treatment, with half of
subjects reporting remission scores six months post-treatment. Patient
satisfaction was 84% at six months post-treatment, and mean patient adherence
to the treatment protocol was 77%. 

– A large multi-center trial conducted by David Tolin, PhD,
Director of the Anxiety Disorders Center at The Institute of Living, and Adjunct
Professor of Psychiatry at Yale University School of Medicine, found that
Freespira produced a clinically significant reduction in panic symptoms 12
months post-treatment in 82% of subjects, with 84% adherence and 88% patient
satisfaction.

– A study led by Alicia Kaplan, MD at the Allegheny Health
Network in Pittsburgh found that use of Freespira not only resulted in 91% of
patients reporting significant reduction in symptoms at 12-months but also
significant cost savings for the patients’ insurance provider, Highmark Blue
Cross Blue Shield. These included a 65% reduction in emergency department
costs; a 68% reduction in pharmacy costs; and a 35% reduction in total medical
costs for treatment of the study subjects. 

“We’re honored that Lightspeed, one of Silicon Valley’s premier venture firms, has joined our existing investors to help speed the commercialization of Freespira to benefit the millions of people who suffer from panic attacks and PTSD, including veterans, first responders, and increasingly, frontline healthcare workers,” said Dean Sawyer, Chief Executive Officer of Freespira. “Now that we have accumulated overwhelming evidence of the clinical and cost effectiveness of Freespira and achieved FDA clearance for its use treating both panic disorder and PTSD, we believe health plans and employers across the country will support the use of Freespira for their members and employees.”

Reviewers from the FDA have given their blessing to the Pfizer/BioNTech COVID-19 vaccine ahead of a key meeting tomorrow – but the regulator noted that there are still uncertainties about whether the shot can stop the disease from spreading.

The gist of a briefing document published from FDA reviewers ahead of an expert advisory board meeting is that the vaccine is good to go.

But there are still unanswered questions that can only be addressed once the shot is administered to the wider public.

Thursday’s vote of the Vaccines and Related Biological Products Advisory Committee is non-binding – but the FDA seldom takes a different viewpoint from its experts when it makes a final regulatory decision.

Pfizer and BioNTech are asking for an Emergency Use Authorization of their vaccine based on phase 3 data, which can be converted into a full licence at a later date once further trial information is published.

Phase 3 trials are designed to test whether vaccines are safe and effective and in this regard FDA reviewers said in the document that the Pfizer/BioNTech vaccine is a success, effective in around 95% of cases and with “favourable safety profile”.

The most common adverse reactions seen in a trial cohort of 38,000 patients were at injection sites (84.1%) followed by fatigue (62.9%) and headache (55.1%), and serious adverse reactions occurred in 0% to 4.6% of participants.

There were four cases of Bell’s palsy – a sudden muscle weakness – in the vaccine group and none in a placebo arm.

However the FDA noted there are currently insufficient data to make conclusions about the safety of the vaccine in subpopulations such as children less than 16 years of age, pregnant and lactating women, and those with compromised immune systems.

The biggest issue for the FDA staffers is one that will only be resolved with time – whether or not the shot can prevent people from infecting each other.

Reviewers said that “additional evaluations including data from clinical trials and from vaccine use post-authorisation will be needed to assess the effect of the vaccine in preventing virus shedding and transmission, in particular in individuals with asymptomatic infection”.

Reviewers noted there was no evidence of “vaccine-enhanced disease” but whether the vaccine could lead to a more resistant strain of the virus emerging is also unclear.

The regulator will be keeping a watching brief on this as more study data emerges over the course of the pandemic.

This risk “needs to be evaluated further in ongoing clinical trials and in observational studies that could be conducted following authorisation and/or licensure,” reviewers said.

Hospitals in the UK have already begun immunising high-risk patients after the country’s regulator became the first in the world to approve the Pfizer/BioNTech vaccine last week.

The post FDA reviewers back Pfizer/BioNTech COVID-19 vaccine ahead of panel appeared first on .

Sanofi and Regeneron’s Dupixent has had the US market for the treatment of chronic rhinosinusitis with nasal polyps (CRSwNP) mainly to itself since June 2019, but GlaxoSmithKline’s Nucala is now breathing down its neck.

The FDA has started its review of Nucala (mepolizumab) for the inflammatory condition, which results in the growth of nasal polyps in the nose that can obstruct airflow and cause mucus discharge, sometimes requiring surgical intervention.

In severe cases, polyps recur, so patients have to undergo multiple operations that according to GSK progressively become less effective and more risky.

The anti-IL-5 antibody is already approved by the FDA for severe asthma and two other conditions – hypereosinophilic syndrome (HES) and eosinophilic granulomatosis with polyangiitis – and is one of GSK’s top growth products with sales rising 28% to more than $930 million in the first nine months of this year.

If approved, it will be the first biologic challenger to Dupixent (dupilumab) – which has a different mechanism of action targeting IL-4 and IL-13 – and would also keep Nucala ahead of IL-5 class rivals like Teva’s Cinqair (reslizumab) and AstraZeneca’s Fasenra (benralizumab) that are challenging it in asthma.

The FDA’s review centres on the SYNAPSE trial, which involved 400 patients with a history of previous surgery who were in need of another procedure due to growing polyps and severe symptoms.

The drug improved both the size of nasal polyps at week 52 and in nasal obstruction during weeks 49-52, compared to placebo when added to standard of care, and extended the time to surgery by 57%.

Standard care for CRSwNP consists of corticosteroids in the nose, after surgery and systemic corticosteroids, but these often lack efficacy.

AZ reported positive results with Fasenra in CRSwNP in September, so may not be far behind with its own regulatory filings, and has also started to catch up with first-to-market Nucala in sales terms, growing 34% to $666 million in the first three quarters of 2020.

Regeneron meanwhile said last month that CRSwNP has been helping to drive increased sales of Dupixent, with a “strong uptick” in prescribing for this indication this year as well as a good performance in other uses like atopic dermatitis that drive it to more than $1.1 billion in global sales in the third quarter alone.

The post Sanofi on notice as FDA reviews GSK’s Nucala for nasal polyps appeared first on .

What You Should Know:

– Pear Therapeutics today announced that it has
successfully closed an $80 million Series D financing led by SoftBank Vision
Fund 2.

–  Pear is the
leader in prescription digital therapeutics and the first company to receive
FDA authorization for a prescription digital therapeutic (PDT) to treat
disease.

– Pear currently has three FDA authorized therapies, reSET, reSET-O and Somryst, for substance use disorder, opioid use disorder, and chronic insomnia, respectively.

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Pear Therapeutics,
Inc., (“Pear” or the “Company”) today announced that it has successfully
closed an $80 million Series D financing led by SoftBank Vision Fund 21 with
participation from existing investors including Temasek, 5AM Ventures,
Arboretum Ventures, JAZZ Venture Partners, Novartis, CrimsoNox, and EDBI, and
new investors, Forth Management, Pilot House, Sarissa Capital, Shanda Group,
and QUAD Investment Management.

What are PDTs?

PDTs are a new therapeutic class that uses software to treat
disease. Just like traditional medicines, prescription digital therapeutics are
prescribed by a physician and backed by clinical data that has been validated
by the FDA. As a new method of care, they offer patients a wide variety of
benefits, including: increased access to therapies, improved engagement and
adherence compared to face-to-face therapies.

Pear’s FDA Authorized Products

Pear’s products reSET®
and reSET-O® for the treatment
of substance use disorder and opioid use disorder, respectively, are the first
two PDTs to receive market authorization to treat disease from FDA. Pear
recently launched Somryst,
for the treatment of chronic insomnia, its third FDA-authorized PDT and the
third PDT to receive market authorization from FDA. Pear also recently launched
its end-to-end virtual care experience combining virtual doctor visit(s) via
telemedicine provider with PearConnect, the industry’s first patient service
center for PDTs.

The Company’s three FDA-authorized products address large
market opportunities with more than 20 million patients suffering from
substance and opioid use disorders and more than 30 million from chronic
insomnia, in the U.S. alone. These diseases are on the rise as the pandemic has
exacerbated the country’s mental health crises.

Expansion Plans

Pear plans to use the latest round of funding to accelerate
reimbursement coverage for its three commercial products, creating the first
market access pathway in the PDT industry. The Company collaborates with
innovators to build a broad and deep pipeline that has the potential to
redefine standard of care in a range of therapeutic areas, including specialty
psychiatry, specialty neurology, and a host of other non-CNS diseases. Pear has
built the first scalable platform infrastructure to discover, develop, and
deliver PDTs to patients.

“Pear is pleased to welcome our new investors and our new board members. SoftBank Investment Advisers represents an ideal partner to support Pear as we build the digital therapeutics industry,” said Corey McCann, M.D., Ph.D., President and CEO of Pear Therapeutics. “This oversubscribed round of funding will allow us to continue to invest in the launches of our three commercial products to accelerate revenue growth, which we intend to reinvest in our robust pipeline and platform.”

What
You Should Know:

–
Digital health startup Kleva Health launches FDA authorized saliva at-home
COVID-19 test kit and raises $1.5 million in seed funding.

–
Kleva Health’s At-Home COVID-19 Saliva Test Kit will be available for orders on
its website, klevahealth.com, and will retail for US$149. Test results will be
reported on klevahealth.com and available within 48 hours after the test has
arrived at the lab.

---

Kleva Health, a San Francisco, CA-based digital health company, today announced the availability of its FDA Authorized at-home COVID-19 testing kits to offer Americans the choice to self-administer COVID-19 tests. In addition to the launch of at-home COVID-19 test kits, the company is also announcing $1.8M in seed funding from VCs including IMO Ventures, Human Longevity, and Performance Impact Venture Fund and Zenni Optical.

---

At-Home COVID-19 Saliva Test Kit

Kleva Health’s At-Home COVID-19 Saliva Test Kit uniquely
includes a Kleva Health saliva DNA/RNA collection device. This enables Kleva
Health to rapidly scale up its supply, without being dependent on distributors.
But a saliva test also enables the company to offer the ability to test for a
wider assortment of diseases and viruses – not just COVID-19.

---

Availability

Kleva Health’s At-Home COVID-19 Saliva Test Kit will be
available for orders on its website, klevahealth.com, and will retail for US$149. Test results
will be reported on klevahealth.com
and available within 48 hours after the test has arrived at the lab.

---

Kleva Health Background

Kleva Health is founded by Harvard Business School
classmates and medical industry executives, David Yu, Bernie Siu and Kai Lim.
But the company started following a familiar story. Bernie Siu (Kleva Health’s
Chief Medical Officer and Doctor of Medicine from Stanford University’s School
of Medicine) had fallen ill from contract COVID-19 but discovered that getting
access to testing solutions ahead of his family reunion was easier said than
done. Not qualified for immediate testing at the time, Bernie believed that
there had to be a better solution. But he’s not alone. As many as 55.1% of
people do not know that at-home COVID-19 testing is an option for Americans,
according to the survey.

---

Responding to the New Normal

Fortunately, Kleva Health’s launch comes at an opportune
time. 55.4% of Americans are prepared for COVID-19 to be around longer than one
year. An additional 31.7% believe that COVID-19 will be around until a vaccine
is available. Not surprisingly, respondents overwhelmingly believe that
COVID-19 has made them realize the need for maintaining healthy habits,
including regular testing. As a digital health tracking company, Kleva Health
encourages healthy habits by offering Americans the convenience of at-home
tests, amid this new normal, and an online platform for keeping track of their
biomedical health.

 “With COVID-19, many of us have a heightened awareness for not only our personal well-being, but also for those family members and friends around us. As the holidays are fast approaching, Kleva Health’s FDA authorized saliva-based COVID-19 self-testing kit, offers Americans the convenience and assurance that they can spend time with loved ones with peace of mind,” said David Yu, CEO and co-founder of Kleva Health. “But this product and the Kleva platform is just the start for us. Supported by our investors including IMO Ventures and the Human Longevity Fund, we plan to scale and introduce additional products that will make biomedical testing as easy and common as brushing one’s teeth.”

---

Future Plans

In addition,
Kleva Health will introduce in January 2021 a patent-pending and FDA authorized
next-generation COVID-19 saliva rapid testing kit that enables Americans to
self-test for the virus at home and read the results in just minutes.

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A daily half-cup of cooked rice may carry a hundred times the acceptable cancer risk of arsenic. What about seaweed from the coast of Maine?

“At one point during the reign of King Cotton, farmers in the south central United States controlled boll weevils with arsenic-based pesticides, and residual arsenic still contaminates the soil.” Different plants have different reactions to arsenic exposure. Tomatoes, for example, don’t seem to accumulate much arsenic, but rice plants are really good at sucking it out of the ground—so much so that rice can be used for “arsenic phytoremediation,” meaning you can plant rice on contaminated land as a way to clear arsenic from the soil. Of course, you’re then supposed to throw the rice—and the arsenic—away. But in the South, where 80 percent of U.S. rice is grown, we instead feed it to people.

As you can see at 0:52 in my video Cancer Risk from Arsenic in Rice and Seaweed, national surveys have shown that most arsenic exposure has been measured coming from the meat in our diet, rather than from grains, with most from fish and other seafood. Well, given that seafood is contributing 90 percent of our arsenic exposure from food, why are we even talking about the 4 percent from rice?

The arsenic compounds in seafood are mainly organic—used here as a chemistry term having nothing to do with pesticides. Because of the way our body can deal with organic arsenic compounds, “they have historically been viewed as harmless.” Recently, there have been some questions about that assumption, but there’s no question about the toxicity of inorganic arsenic, which you get more of from rice.

As you can see at 1:43 in my video, rice contains more of the toxic inorganic arsenic than does seafood, with one exception: Hijiki, an edible seaweed, is a hundred times more contaminated than rice, leading some researchers to refer to it as the “so-called edible hijiki seaweed.” Governments have started to agree. In 2001, the Canadian government advised the public not to eat hijiki, followed by the United Kingdom, the European Commission, Australia, and New Zealand. The Hong Kong Centre for Food Safety advised the public not to eat hijiki and banned imports and sales of it. Japan, where there is actually a hijiki industry, just advised moderation.

What about seaweed from the coast of Maine—domestic, commercially harvested seaweed from New England? Thankfully, only one type, a type of kelp, had significant levels of arsenic. But, it would take more than a teaspoon to exceed the provisional daily limit for arsenic, and, at that point, you’d be exceeding the upper daily limit for iodine by about 3,000 percent, which is ten times more than reported in a life-threatening case report attributed to a kelp supplement.

I recommend avoiding hijiki due to its excess arsenic content and avoiding kelp due to its excess iodine content, but all other seaweeds should be fine, as long as you don’t eat them with too much rice.

In the report mentioned earlier where we learned that rice has more of the toxic inorganic arsenic than fish, we can see that there are 88.7 micrograms of inorganic arsenic per kilogram of raw white rice. What does that mean? That’s only 88.7 parts per billion, which is like 88.7 drops of arsenic in an Olympic-size swimming pool of rice. How much cancer risk are we talking about? To put it into context, the “usual level of acceptable risk for carcinogens” is one extra cancer case per million. That’s how we typically regulate cancer-causing substances. If a chemical company wants to release a new chemical, we want them to show that it doesn’t cause more than one in a million excess cancer cases.

The problem with arsenic in rice is that the excess cancer risk associated with eating just about a half cup of cooked rice a day could be closer to one in ten thousand, not one in a million, as you can see at 4:07 in my video. That’s a hundred times the acceptable cancer risk. The FDA has calculated that one serving a day of the most common rice, long grain white, would cause not 1 in a million extra cancer cases, but 136 in a million.

And that’s just the cancer effects of arsenic. What about all the non-cancer effects? The FDA acknowledges that, in addition to cancer, the toxic arsenic found in rice “has been associated with many non-cancer effects, including ischemic heart disease, diabetes, skin lesions, renal [kidney] disease, hypertension, and stroke.” Why, then, did the FDA only calculate the cancer risks of arsenic? “Assessing all the risks associated with inorganic arsenic would take considerable time and resources and would delay taking any needed action to protect public health” from the risks of rice.

“Although physicians can help patients reduce their dietary arsenic exposure, regulatory agencies, food producers, and legislative bodies have the most important roles” in terms of public health-scale changes. “Arsenic content in U.S.-grown rice has been relatively constant throughout the last 30 years,” which is a bad thing.

“Where grain arsenic concentration is elevated due to ongoing contamination, the ideal scenario is to stop the contamination at the source.” Some toxic arsenic in foods is from natural contamination of the land, but soil contamination has also come from the dumping of arsenic-containing pesticides, as well as the use of arsenic-based drugs in poultry production and then the spreading of arsenic-laced chicken manure on the land. Regardless of why south central U.S. rice paddies are so contaminated, we shouldn’t be growing rice in arsenic-contaminated soil.

What does the rice industry have to say for itself? Well, it started a website called ArsenicFacts. Its main argument appears to be that arsenic is everywhere, we’re all exposed to it every day, and it’s in most foods. But shouldn’t we try to cut down on the most concentrated sources? Isn’t that like saying look, diesel exhaust is everywhere, so why not suck on a tailpipe? The industry website quotes a nutrition professor saying, “All foods contain arsenic. So, if you eliminate arsenic from your diet, you will decrease your risk…and you’ll die of starvation.” That’s like Philip Morris saying that the only way to completely avoid secondhand smoke is to never breathe—but then you’ll asphyxiate, so you might as well just start smoking yourself. If you can’t avoid it, you might as well consume the most toxic source you can find?!

That’s the same tack the poultry industry took. Arsenic and chicken? “No need to worry” because there’s a little arsenic everywhere. That’s why it’s okay the industry fed chickens arsenic-based drugs for 70 years. If you can’t beat ’em, join ’em.

How can the rice industry get away with selling a product containing a hundred times the acceptable cancer risk? I cover that and so much more in my other videos on arsenic and rice, which also include concrete recommendations on how to mediate your risk.

---

Check out:

• Which Rice Has Less Arsenic: Black, Brown, Red, White, or Wild?
• Which Brands and Sources of Rice Have the Least Arsenic?
• How to Cook Rice to Lower Arsenic Levels
• Arsenic in Infant Rice Cereal
• Arsenic in Rice Milk, Rice Krispies, and Brown Rice Syrup
• How Risky Is the Arsenic in Rice?
• How Much Arsenic in Rice Is Too Much?
• Is White Rice a Yellow-Light or Red-Light Food?
• Do the Pros of Brown Rice Outweigh the Cons of Arsenic?
• Benefits of Turmeric for Arsenic Exposure

Pesticides were not the only source of arsenic. Poultry poop, too, if you can believe it! I cover that story in Where Does the Arsenic in Chicken Come From? and Where Does the Arsenic in Rice, Mushrooms, and Wine Come From?.

Chronic low-dose arsenic exposure is associated with more than just cancer. See The Effects of Too Much Arsenic in the Diet.

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

Americans have made no secret of their skepticism of COVID-19 vaccines this year, with fears of political interference and a “warp speed” timeline blunting confidence in the shots. As recently as September, nearly half of U.S. adults said they didn’t intend to be inoculated.

But with two promising vaccines primed for release, likely within weeks, experts in ethics and immunization behavior say they expect attitudes to shift quickly from widespread hesitancy to urgent, even heated demand.

“People talk about the anti-vaccine people being able to kind of squelch uptake. I don’t see that happening,” Dr. Paul Offit, a vaccinologist with Children’s Hospital of Philadelphia, told viewers of a recent JAMA Network webinar. “This, to me, is more like the Beanie Baby phenomenon. The attractiveness of a limited edition.”

Reports that vaccines produced by drugmakers Pfizer and BioNTech and Moderna appear to be safe and effective, along with the deliberate emphasis on science-based guidance from the incoming Biden administration, are likely to reverse uncertainty in a big way, said Arthur Caplan, director of the division of medical ethics at New York University School of Medicine.

“I think that’s going to flip the trust issue,” he said.

The shift is already apparent. A new poll by the Pew Research Center found that by the end of November 60% of Americans said they would get a vaccine for the coronavirus. This month, even as a federal advisory group met to hash out guidelines for vaccine distribution, a long list of advocacy groups — from those representing home-based health workers and community health centers to patients with kidney disease — were lobbying state and federal officials in hopes their constituents would be prioritized for the first scarce doses.

“As we get closer to the vaccine being a reality, there’s a lot of jockeying, to be sure,” said Katie Smith Sloan, chief executive of LeadingAge, a nonprofit organization pushing for staff and patients at long-term care centers to be included in the highest-priority category.

Certainly, some consumers remain wary, said Rupali Limaye, a social and behavioral health scientist at the Johns Hopkins Bloomberg School of Public Health. Fears that drugmakers and regulators might cut corners to speed a vaccine linger, even as details of the trials become public and the review process is made more transparent. Some health care workers, who are at the front of the line for the shots, are not eager to go first.

“There will be people who will say, ‘I will wait a little bit more for safety data,” Limaye said.

But those doubts likely will recede once the vaccines are approved for use and begin to circulate broadly, said Offit, who sits on the FDA advisory panel set to review the requests for emergency authorization Pfizer and Moderna have submitted.

He predicted demand for the COVID vaccines could rival the clamor that occurred in 2004, when production problems caused a severe shortage of flu shots just as influenza season began. That led to long lines, rationed doses and ethical debates over distribution.

“That was a highly desired vaccine,” Offit said. “I think in many ways that might happen here.”

Initially, vaccine supplies will be tight, with federal officials planning to ship 6.4 million doses within 24 hours of FDA authorization and up to 40 million doses by the end of the year. The CDC panel recommended that the first shots go to the 21 million health care workers in the U.S. and 3 million nursing home staff and residents, before being rolled out to other groups based on a hierarchy of risk factors.

Even before any vaccine is available, some people are trying to boost their chances of access, said Dr. Allison Kempe, a professor of pediatrics at the University of Colorado School of Medicine and expert in vaccine dissemination. “People have called me and said, ‘How can I get the vaccine?’” she said. “I think that not everyone will be happy to wait, that’s for sure. I don’t think there will be rioting in the streets, but there may be pressure brought to bear.”

That likely will include emotional debates over how, when and to whom next doses should be distributed, said Caplan. Under the CDC recommendations, vulnerable groups next in line include 87 million workers whose jobs are deemed “essential” — a broad and ill-defined category — as well as 53 million adults age 65 and older.

“We’re going to have some fights about high-risk groups,” said Caplan of NYU.

The conversations will be complicated. Should prisoners, who have little control over their COVID exposure, get vaccine priority? How about professional sports teams, whose performance could bolster society’s overall morale? And what about residents of facilities providing care for people with intellectual and developmental disabilities, who are three times more likely to die from COVID-19 than the general population?

Control over vaccination allocation rests with the states, so that’s where the biggest conflicts will occur, Caplan said. “It’s a short fight, I hope, in the sense in which it gets done in a few months, but I think it will be pretty vocal.”

Once vaccine supplies become more plentiful, perhaps by May or June, another consideration is sure to boost demand: requirements for proof of COVID vaccination for work and travel.

“It’s inevitable that you’re going to see immunity passports or that you’re required to show a certificate on the train, airplane, bus or subway,” Caplan predicted. “Probably also to enter certain hospitals, probably to enter certain restaurants and government facilities.”

But with a grueling winter surge ahead, and new predictions that COVID-19 will fell as many as 450,000 Americans by February, the tragic reality of the disease will no doubt fuel ample demand for vaccination.

“People now know someone who has gotten COVID, who has been hospitalized or has unfortunately died,” Limaye said.

“We’re all seeing this now,” said Kempe. “Even deniers are beginning to see what this illness can do.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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Biosimilars are developed to be highly similar versions of approved biologics in terms of safety, purity, and potency. Biosimilars are expected to be a cost-effective alternative to the high-priced branded biologics, offering significant and much-needed cost savings to both payers and the patients. Hence, the providers are more likely to adopt biosimilars as a “reference product to biologics” possessing similar therapeutic properties. During the month of November, Formycon and Bioeq reported the first patient dosing in the P-III study of FYB202 while Prestige collaborated with Teva to commercialize Tuznue. Our team at PharmaShots has summarized 21 key events of the biosimilar space of Nov 2020.

Celltrion Presented Results of CT-P17 (biosimilar, adalimumab) in P-III Study for RA at ACR 2020

Published: Nov 03, 2020

Product: CT-P17 (biosimilar, adalimumab)

• The P-III study involves assessing CT-P17 (40mg, q2w) vs reference adalimumab for up to 24wks. in 648 patients with active moderate-to-severe RA despite MTX treatment
• Results demonstrated that CT-P17 has equivalent efficacy to reference adalimumab i.e. ACR20 is 82.7% for both, 2EPs include ACR20/50/70 response rates, mean DAS28, CDAI & SDAI & EULAR (CRP) response, Ctrough of adalimumab is higher for CT-P17 & lower in the ADA positive subgroup than the ADA negative subgroup in both treatment groups, the safety profile is comparable
• Additionally, comparable PK and safety data is presented for CT-P17 in comparison with EU-approved & US-licensed adalimumab in 312 healthy subjects. Celltrion also presented PK and safety data for two delivery methods for CT-P17, the auto-injector (AI) and pre-filled syringe (PFS)

Formycon Reported BLA Resubmission Strategy for FYB201 (biosimilar, ranibizumab)

Published: Nov 06, 2020

Product: FYB201 (biosimilar, ranibizumab)

• Formycon & Bioeq reported the BLA resubmission strategy for FYB201 (biosimilar referencing Lucentis) has been adjusted
• With the revised submission strategy, the companies expect a simplification of the approval procedure. The modified submission dossier is anticipated to be filed with the US FDA in H1’21
• The adjustment of the regulatory strategy while optimizing the commercial supply chain is not expected to have any impact on the timing of the anticipated launch of FYB201 in the US & EU

Formycon and Bioeq Reported First Patient Dosing in P-III Study of FYB202 (biosimilar, ustekinumab)

Published: Nov 09, 2020

Product: FYB202 (biosimilar, ustekinumab)

• The focus of the P-III study is to demonstrate the comparability of FYB202 & the reference product Stelara in terms of efficacy, safety & immunogenicity in patients with moderate to severe psoriasis vulgaris
• FYB202 is being developed as part of JV b/w Aristo Pharma & Formycon along with Bioeq. Bioeq is responsible for the clinical studies which were developed in close cooperation with the US FDA & the EMA
• The ustekinumab is mAb targeting the cytokines IL-12 & IL-23. Stelara is used to treat various severe inflammatory conditions such as mod. to sev. psoriasis, CD & for UC

Alvotech and Cipla Collaborated to Ensure Access to Biosimilars in South Africa

Published: Nov 09, 2020

Product: Biosimilar

• Alvotech and Cipla entered an exclusive partnership to provide patients with better access to high quality and cost-effective biosimilar medicines in South Africa
• Alvotech will be responsible for the development and supply of the products and Cipla will be responsible for the registration and commercialization
• The biosimilar portfolio will include five biosimilars- two for oncology and three for treating auto-immune diseases

Genentech Filed Complaint Against Centus Over Proposed Bevacizumab Biosimilar

Published: Nov 13, 2020

Product: Proposed Bevacizumab Biosimilar

• Genentech filed a complaint in the Eastern District of Texas alleging that the proposed biosimilar to Avastin (bevacizumab) product infringes 10 US patents
• Genentech alleges that Centus and partners failed to disclose sufficient information about the proposed biosimilar to enable Genentech to do a sufficient analysis of potential patent infringements
• Centus has a BLA under review with the FDA for the bevacizumab biosimilar candidate FKB238, and the company has filed a notice of intent to commercialize the agent

Prestige Signed an Exclusive Agreement with Teva to Commercialize Tuznue (biosimilar, trastuzumab) in Israel

Published: Nov 11, 2020

Product: Tuznue (biosimilar, trastuzumab)

• Teva to get an exclusive right to commercialize Tuznu in Israel, leveraging its marketing capabilities and experience in bringing pharmaceutical products to market and will be responsible for local registration, sales, and marketing in Israel
• Prestige will assume responsibility for product registration with the EMA and commercial supply of Tuznue from its manufacturing facilities in Osong, Korea
• Tuznue is biosimilar referencing Roche’s Herceptin (trastuzumab), used to treat HER2-overexpressing BC & m-gastric adenocarcinoma. Additionally, the EMA has accepted an MAA for Tuznue based on the global clinical trial results

Samsung Bioepis Initiated P-I Study of SB16 Proposed Biosimilar to Prolia (denosumab)

Published: Nov 11, 2020

Product: SB16 proposed biosimilar to Prolia

• The P-I study assesses the PK/PD, safety, tolerability of SB16 (denosumab) vs Prolia in 168 healthy male volunteers for osteoporosis. The study will be 3 arms study that involves dosing with SB16 either the EU or US-sourced Prolia
• The proposed biosimilar references Amgen’s Prolia which was approved in 2010 for osteoporosis with a high risk of fracture
• With the initiation, Samsung Bioepis continues to advance its biosimilar portfolio covering immunology, oncology, ophthalmology, and hematology

Henlius Reported First Patients Dosing in P-I Study of HLX14 (denosumab, biosimilar)

Published: Nov 11, 2020

Product: HLX14 (denosumab, biosimilar)

• The first patient has been dosed in a P-I study of HLX14, conducted in 2 parts, Part 1 is a pilot study assessing PK/PD, safety, tolerability & immunogenicity of HLX14 vs EU-sourced denosumab (SC) in healthy male volunteers
• Part 2 is a four-arm study assessing the bioequivalence of HLX14 vs US-, EU-, CN-sourced denosumab. The study also evaluates PD, safety, tolerability, and immunogenicity between HLX14 and the reference drug
• Results from the P-I study will provide reference for the dosing scheme in the clinical studies of HLX14

Xbrane Reported Patient Enrollment Completion in P-lll XPLORE Study of Xlucane (biosimilar, ranibizumab)

Published: Nov 11, 2020

Product: Xlucane (biosimilar, ranibizumab)

• Xbrane reported that the last patient has been enrolled into the P-III XPLORE study assessing Xlucane vs Lucentis in 580 patients with wet AMD
• The company will conduct an interim read-out from the XPLORE study when the last patient has reached 6mos. of their treatment schedule. Top-line data is expected to be communicated mid-2021 and filing of the MAA/BLA to EMA and the US FDA anticipated to take place imminently
• Filing of MAA/BLA is expected to take place mid-2021. With an expected 12mos. regulatory process upon filing, MAA is expected in the EU and the US mid-2022 allowing for the launch of Xlucane

Henlius Reported the NMPA’s Acceptance of HLX15 (biosimilar, daratumumab) to Treat Multiple Myeloma

Published: Nov 16, 2020

Product: HLX15 (biosimilar, daratumumab)

• The NMPA has accepted HLX15’s IND to be used in the treatment of multiple myeloma. HLX15 is Henlius’ second self-developed product around blood tumor treatment
• The company evaluated the biosimilar in a head to head clinical studies demonstrating that HLX15 is highly similar to its reference daratumumab while the safety profiles are also similar
• The company has developed the HLX15 in accordance with the technical guidelines of development and evaluation of biosimilar drugs and EMA guideline on similar biological medicinal products

Samsung Bioepis Presented Results of SB11 Proposed Biosimilar to Lucentis in P-III Study at the AAO 2020 Virtual

Published: Nov 16, 2020

Product: SB11 proposed biosimilar to Lucentis

• The P-III study involves assessing SB11 vs reference ranibizumab in monthly injections (0.5 mg) in 705 patients in a ratio (1:1) with nAMD while only 634 patients continued to receive treatment up to 48wks.
• One-year results from the P-III study demonstrated equivalence between SB11 and reference ranibizumab in patients with nAMD
• The study met its 1EPs i.e. changes from baseline in BCVA @8wks. and CST @4wks. The EMA has accepted for review the MAA of SB11 in Oct’2020

Samsung Bioepis and Biogen Reported the FDA’s Acceptance of BLA for SB11 Proposed Biosimilar to Lucentis

Published: Nov 18, 2020

Product: SB11 proposed biosimilar to Lucentis

• The US FDA has accepted for review the BLA of SB11, a proposed biosimilar referencing Lucentis (ranibizumab)
• The EMA has accepted for review the MAA of SB11 in Oct’2020. If approved, SB11 will add to the biosimilars portfolio developed under the collaboration of Samsung Bioepis and Biogen including Benepali, Imraldi & Flixabi
• In Nov’2019, Samsung Bioepis entered into a commercialization agreement with Biogen for 2 ophthalmology biosimilar candidates, SB11 (ranibizumab) & SB15 (aflibercept) in the US, Canada, Europe, Japan & Australia. Ranibizumab is an anti-VEGF therapy for retinal vascular disorders

The US FDA Draft New Guidelines for Biosimilarity and Interchangeability

Published: Nov 19, 2020

Product: Biosimilar

Shots:

• The FDA has released a draft guidance for industry entitled “Biosimilarity and Interchangeability: Additional Draft Q&As on Biosimilar Development and the BPCI Act”
• The draft guidance is intended to inform prospective applicants and facilitate the development of proposed biosimilars and proposed interchangeable products, as well as describe FDA’s interpretation of statutory requirements added by the BPCI Act
• The draft guidance is to be published in the Federal Register on Nov 20, 2020

Alvotech Reported the US FDA and EMA’s Acceptance of AVT02 Proposed Biosimilar to Humira (adalimumab)

Product: Nov 20, 2020

Product: AVT02, a proposed biosimilar to Humira

• The US FDA has accepted the BLA of AVT02 for review and is expected to decide on the filing in Sept’2021 while the EMA has accepted for review an MAA for AVT02 with an EMA decision anticipated in the Q4’21
• The filings were based on AVT02-GL-101 & AVT02-GL-301 studies demonstrating a high degree of similarity b/w AVT02 and the reference products. AVT02-GL-101 study met its 1EPs of PK similarity while the later study confirmed the efficacy and safety of AVT02 in patients with mod. to sev. chronic psoriasis
• AVT02 is a proposed biosimilar to the reference product Humira (adalimumab) with high concentration (100mg/mL) dosage forms

Henlius Presented Results of HLX04 (biosimilar, bevacizumab) in P-III Study at ESMO Asia 2020

Published: Nov 20, 2020

Product: HLX04 (biosimilar, bevacizumab)

• The P-III HLX04-mCRC03 study involves assessing the efficacy, safety and immunogenicity of HLX04 vs reference bevacizumab (7.5 mg/kg, q3w or 5 mg/kg, q2w) + CT (Xelox or mFOLFOX6) as a 1L treatment in patients in the ratio of (1:1) with mCRC
• Result: PFSR36wk (46.4% vs 50.7%); no significant difference b/w the treatment groups in 2EPs including OS, PFS, ORR, TTR and DoR; safety and immunogenicity profiles were similar b/w HLX04 and the reference
• The NMPA has accepted the NDA for HLX04. Additionally, Henlius has submitted a patent for a new formulation of HLX04 with potential better safety and stability, designed for ophthalmic use

Samsung Biologics and AstraZeneca Dissolved Rituximab Alliance

Published: Nov 20, 2020

Product: SAIT101 (biosimilar, rituximab)

• Samsung Biologics and AstraZeneca had decided to suspend long-running research and development activities by a jointly owned subsidiary, Archigen Biotech, which was solely engaged in development of SAIT101 (biosimilar, rituximab)
• Samsung halted the P-III study of SAIT101 in Oct’2012 and resumed it in 2014 via Archigen. The P-III study similar therapeutic effect to Rituxan in 315 FL patients with ORR (66.3% vs 70.6%)
• The companies decided to stop commercializing SAIT101 and take step for liquidation of Archigen as the product lacks commercial viability

The US FDA Approved Pfizer’s Oncology Supportive Care Biosimilar Nyvepria (biosimilar, pegfilgrastim)

Product: Nov 20, 2020

Product: Nyvepria (biosimilar, pegfilgrastim)

• The EC has approved Nyvepria, a biosimilar referencing Neulasta to reduce the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic CT for malignancy
• The EC approval is based data demonstrating a high degree of similarity of Nyvepria to its reference product
• Pfizer plans to make Nyvepria available to patients in multiple EU countries starting in Q1’21. The EC’s approval follows the US FDA’s approval granted in Jun’2020

Innovent Reported Results of Tyvyt + Byvasda (biosimilar, bevacizumab) in P-lll ORIENT-32 Study as 1L Treatment for HCC

Product: Nov 23, 2020

Product: Byvasda (biosimilar, bevacizumab)

• The P-lll ORIENT-32 study involves assessing of Tyvyt (sintilimab) + Byvasda vs sorafenib as a 1L treatment in 571 patients in a ratio (2:1) with advanced HCC and the result were released in an oral presentation at the ESMO Asia Virtual Congress 2020
• Result: Reduction in risk of all-cause mortality (43.1%); the median OS (not reached vs 10.4 mos.); reduction in risk of progression (43.5%); m-PFS (4.6 vs 2.8 mos.), results was presented at ESMO 2020
• The improved OS and PFS benefits of the dual regimen were generally consistent across all subgroups and showed an acceptable safety profile with no new safety signals

Innovent’s Sulinno (biosimilar, adalimumab) Received NMPA’s Approval for Polyarticular Juvenile Idiopathic Arthritis

Published: Nov 23, 2020

Product: Sulinno (biosimilar, adalimumab)

• The NMPA has approved Sulinno for the treatment of pJIA which is the fourth approved indication of the therapy in China. Earlier, Sulinno was approved for RA, PS, and psoriasis
• The launch of Sulinno has provided more Chinese patients with high-quality and relatively affordable adalimumab injection, bringing hope and opportunities to more patients
• Sulinno is a human anti-TNF-α mAb referencing Humira. The clinical results were published at the Inaugural Issue of The Lancet Rheumatology in 2019

Alvotech and Alvotech & CCHT Signed an Exclusive Commercialization Agreement with Yangtze River for Eight Biosimilars in China

Product: Nov 25, 2020

Product: Biosimilar

• The companies collaborate with the Yangtze to commercialize eight biosimilars in China. The initial pipeline contains biosimilar candidates for the treatment of autoimmunity, ophthalmology, and oncology
• Alvotech and Alvotech & CCHT will be jointly responsible for the development, registration, and supply of biosimilars in China while Yangtze River Pharmaceutical will exclusively commercialize the biosimilars
• The manufacturing of biosimilars will be made in a new state-of-the-art biopharmaceutical facility, currently being built in Changchun, China, through the Alvotech & CCHT. The first phase of the facility is expected to be completed in 2021

Bio-Thera Reported MAA Submission to EMA for BAT1706 a Proposed Biosimilar to Avastin

Product: Nov 26, 2020

Product: BAT1706 (a proposed biosimilar to Avastin)

• The company has submitted an MAAA for BAT1706 to EMA. Bio-Thera seeks a commercial license for all approved indications of bevacizumab in the EU Member States, Iceland, Norway, and Liechtenstein
• The submission of the MAA for BAT1706 marks it as the first ex-China MAA/ BLA submission. The BLA of the biosimilar for metastatic carcinoma of the colon or rectum and NSCLC is under NMPA’s review
• The company plans to submit a BLA for BAT1706 to the US FDA by the end of 2020. Bevacizumab is a mAb that targets VEGF thus reduces neovascularization, thereby inhibiting tumor growth

Related Post: Insights+ Key Biosimilars Events of October 2020

The post Insights+ Key Biosimilars Events of November 2020 first appeared on PharmaShots.

With multiple COVID-19 vaccines rapidly heading toward approval, optometrists and dentists are pushing for the authority to immunize patients during routine eye exams and dental cleanings.

Across the country, these medical professionals say their help will be needed to distribute the vaccines to millions of Americans — and they already have the know-how.

“When you look at what dentists do, and how many injections they give day in and day out, I think they’re more than qualified,” said Jim Wood, a California state assembly member and dentist. “It’s kind of a no-brainer.”

In California, the professional organizations representing dentists and optometrists are in talks with state officials to expand their job descriptions to include administering vaccines. Oregon has already begun training and certifying dentists to give vaccines. And at least half the states have considered allowing dentists to administer COVID vaccines once they’re available, according to the American Association of Dental Boards.

That list is likely to grow, because the U.S. Centers for Medicare & Medicaid Services recommended in October that states consider expanding their list of vaccine providers.

The dentists and optometrists seeking permission to vaccinate patients against COVID-19 and other diseases argue that their help will take some of the pressure off hospitals and doctors’ offices. It could also bring some extra money into their practices.

“Everyone in our specialized health care system should also play a preventive role,” said Dr. William Sage, a professor of law and medicine at the University of Texas-Austin. “Pandemic or not, being alert to preventive health in any setting is a good thing.”

In November, Pfizer, Moderna and AstraZeneca announced that their COVID vaccine candidates delivered promising results in clinical trials, and that millions of doses could be ready before the end of the year. Pfizer’s has to be stored at ultracold temperatures, while Moderna’s and AstraZeneca’s can be kept at standard refrigerator temperatures.

This wouldn’t be the first time health professionals other than doctors administered vaccines during a pandemic. Nursing students, EMTs and midwives in a handful of states were granted temporary and limited authority to administer flu vaccines during the H1N1 swine flu pandemic of 2009-10. Dentists in Massachusetts, Illinois, New York and Minnesota also were temporarily deputized as vaccinators.

Since then, Minnesota and Illinois have adopted laws to allow dentists to give flu shots to adults. And last year, Oregon became the first state to allow dentists to give any vaccine to any patient, whether a child or an adult.

So far, more than 200 dentists and dental students in Oregon have completed the training course offered by the Oregon Health & Science University’s School of Dentistry, with 60 others expected to finish by the end of December, said Mary Pat Califano, an instructor who helped develop the hands-on part of the training.

Students spend around 10 hours in online classes. They then undergo hands-on training during which they practice injections on a shoulder pad before practicing injecting a partner with saline. They’re taught how to counsel patients about vaccines and avoid injuring patients’ shoulders when giving the shots.

Once dentists pass an exam, they can register with the Oregon Health Authority and begin getting their staff trained to handle vaccines and procuring a fridge to store them.

The goal, Califano said, is not to replace family doctors or primary care physicians, but to supplement them. The federal Agency for Health Research and Quality found that, in 2017, 31.1 million Americans saw a dentist but not a physician.

“We just need as many people as possible to give flu shots and COVID-19 vaccines when they’re available,” Califano said. “If it happens that they’re in a dental office, and that provider is educated and capable of giving a vaccine, why not?”

In California, the state dental association is exploring options for gaining vaccine authority, which would likely require the legislature to step in. This year, California passed a law allowing pharmacists to administer COVID vaccines approved by the U.S. Food and Drug Administration.

Wood, who carried that measure, hasn’t yet committed to sponsoring a bill that would let dentists give vaccines, but says he supports the idea.

“We give injections in the mouth all day long, and these are very precise kinds of injections,” Wood said. “I think the learning curve for a dentist would be small.”

Dr. Bill Schaffner, a professor of preventive medicine and infectious disease at Vanderbilt University, said these proposals for expanding the vaccine workforce are promising. Flu vaccines, which are relatively low-risk and simple to administer, would be the perfect candidate to stock in dental and optometric fridges to start.

But Schaffner doesn’t believe dentists and optometrists will play a major role in the COVID immunization effort. It would take too long to pass legislation to expand the scope of practice for every professional who wants it in every state, he said. And since some COVID vaccines have specific shipping and subzero storing requirements, they will probably be distributed only to specially trained personnel at a small number of locations, he said.

There’s also the question of payment. It’s hard — but not impossible — to make a profit administering vaccines, Schaffner said.

Providers have to decide each season how many doses to buy, and any that go bad or remain in the fridge at the end of their shelf life equal monetary losses.

“Unless you’re very assiduous about moving the vaccine from the fridge into arms, you’re not going to make money,” Schaffner said. “People who do that can augment their income, but nobody is going to drive a Porsche because of vaccines.”

Jeff McCombs, an associate professor of health economics at the University of Southern California School of Pharmacy, agreed it might not make business sense for most dentists to start vaccinating. He said it would be hard to keep a well-stocked vaccine fridge with enough variety to meet patients’ needs without wasting doses. Generally, adults who choose not to get vaccinated do so because they’re uneducated about vaccines or afraid, he said, not because they can’t access them.

“I don’t think it’s going to harm people,” McCombs said. “I just don’t think they’ll make any money at it.”

While the California Department of Public Health said the state’s current vaccine infrastructure is sufficient for flu shots and routine immunizations, it is “carefully considering the need to include additional types of immunizers” to get Californians vaccinated against COVID-19, according to a statement from the department.

The California Optometric Association said it is in talks with Gov. Gavin Newsom’s vaccine task force about how to get optometrists into the mix, and is exploring legislative options as well.

“We can serve the dual role of assisting with vision needs and protecting from COVID,” said David Ardaya, an optometrist in Whittier who chairs an association committee that is looking into the issue. “Our whole hope is to assist our nation in regaining its health and in returning to a sense of normal.”

But three years after AB-443 was signed, the regulations implementing it have yet to be finalized.

That didn’t stop Frank Giardina, an optometrist in Nipomo, from going through a certification program anyway.

The 20-hour course, which includes online lectures, hands-on lessons and an exam, is the same course pharmacists take when learning how to give all vaccines to people of all ages.

Giardina pointed to the shingles, or herpes zoster, virus as an example of why optometrists are well suited to give vaccines. The virus can infect the eyes, and even though he’s allowed to treat shingles, he can’t give a vaccine to prevent it.

For now, he’s holding out hope he will get permission to administer vaccines, including for COVID-19. He envisions a world in which a patient comes in for contact lenses and he can offer them a flu or COVID vaccine while they’re there.

“We’re another member of the health care team. It’s a waste of manpower not to,” Giardina said. “If you’re trying to vaccinate all these people, especially in rural areas, you need whoever you can find.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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This is the question that a recent paper by Chorniy et al. (2020) attempt to answer. This issue is clearly very relevant as the UK recently has approved the Pfizer/BioNTech vaccine for COVID-19 before the US. Unlike most studies that attempt to examine the relationship between FDA review time and R&D investment dollars, the authors aim to measure the relationship between FDA review time and number of drugs in the pipeline. Whether the former is probably a better measure of R&D investment efforts, the latter is what society cares more about.

The dependent variable is the number of drugs in the pipeline for indication category, and the key independent variable is the natural log of the FDA review time for drug category C. The drug pipeline data comes from AdisInsight and the review time comes from the [email protected] database. The regression also controls for whether the drug is receives priority or orphan status (also from Drugs @FDA), the development cost and the market size. The development cost is endogenous so the authors use the number of pages in an NDA submission, the number of Phase III clinical trials and the Phase III trial sample size. The vector of market characteristics include disease mortality and morbidity (from World Health Organization data by disease), all‐payer drug expenditures (from the Medicare Expenditure Panel Survey, MEPS); number of drugs on the market (also from MEPS); and drug prices (from Express Scripts/Medco and Redbook).

The authors find that:

The average FDA review time for drugs approved after 1999 is 466 days, or about 1.3 years, but …it takes anywhere from 46 days (Eloxatin) to 1827 days (Prialt) for a drug to complete the review process that gives a drug a green light to be marketed. Post‐PDUFA, many NDAs were eligible for a special review status. About a half of the drugs in our sample received a priority review status, and about 20% were classified as orphan, on average by disease category.

Using the regression specification described above, they also find that longer review time decreases the number of drugs in the pipeline.

A doubling of the review length is associated with approximately six fewer drugs in the development pipeline in that disease category. This implies that a one‐sixth increase in review length is associated with approximately one fewer drug in development; with a mean review length of 466 days, this implies that each 78 extra days of review are associated with one fewer drug in development.

One challenge of this study is that pipeline decisions are made years in advance. Thus, longer review time may also impact the decision for early phase drug development, but the data the authors use is from a fairly limited time period 1999 to 2005. Given that the drug development timeline is typically more than 10 years, this study estimates the impact over a relatively short time period. The study also ignores the regulatory process in other countries as well, and their impact on drug development. While the US is the largest pharmaceutical markets, the regulatory environment in other countries–particularly in Europe–may affect investment decisions.

Nevertheless, it is clearly logical that additional regulatory burden and delays in time to market clearly do affect this study does contribute to pharmaceutical firms investment decisions. Budish et al. (2015) find that firms often invest in oncology indications for late stage disease because the time for trials to read out is much shorter. To expedite the FDA review process, in 1992 Congress passed the Prescription Drug User Fee Act (PDUFA) which allowed the FDA to charge fees to pharmaceutical firms to expedite the review process. These payments fund just under half of all drug reviews.

This study does add to the literature on how pharmaceutical firm R&D respond to incentives. For instance, Acemoglu and Linn (2004) found that a potential market size affects affects the number of new drugs that get to market. Other studies have found that higher profits boost pharmaceutical firm R&D investments, for instance the advent of Medicare Part D (Blume-Kohout and Sood 2013) and changes in patent law (Williams 2017). Perhaps the best-known paper–Dubois et al. 2015–found an elasticity of innovation with respect to market size of 0.23, suggesting that $2.5 billion of revenue is required to bring a new drug to market based on drug development costs. The paper by Chorniy et al. (2020), despite some limitations, helps add to this literature.

Source:

• Chorniy A, Bailey J, Civan A, Maloney M. Regulatory review time and pharmaceutical research and development. Health Economics. 2020 Oct 19.

US biotech TG Therapeutics has begun a rolling filing with the FDA for its combination therapy for chronic lymphocytic leukaemia, in a challenge to Roche.

The New York-based firm has requested approval for the combination of its anti-CD20 antibody ublituximab and umralisib, an oral drug that inhibits PI3K-delta and CK-1 epsilon.

The FDA had already granted fast-track designation to the combination therapy, allowing extra help during the development process on the basis of earlier clinical data.

Aside from Gazyvaro, CLL can be treated with older chemotherapy agents, or Roche/AbbVie’s Venclexta (venetoclax) in some instances, and the fast track tag showed the FDA considered the combination to be a promising new contender on the market.

TG said it expects the data submission to be complete in the first half of 2021.

The rolling filing is designed to speed up the review process for results from the UNITY-CLL trial, a phase 3 study comparing the combination therapy also known as U2, with an active control arm of Roche’s Gazyvaro (ofatumumab) plus chlorambucil.

The trial randomised patients into four treatment arms: ublituximab single agent, umbralisib single agent, ublituximab plus umbralisib, and an active control arm of obinutuzumab plus chlorambucil.

A prespecified analysis was conducted to assess the contribution of ublituximab and umbralisib in the U2 combination arm and allowed for the termination of the single agent arms.

The trial continued enrolment in a 1:1 ratio into the two combination arms: the investigational arm of U2 and the control arm of obinutuzumab plus chlorambucil.

Around 420 people were recruited to the two combination arms and around 60% were untreated and 40% were relapsed or refractory.

Primary endpoint was superior progression-free survival in the control arm and topline results were announced in May, showing a statistically significant improvement in PFS in the combination therapy arm.

The company had hoped to file data from an interim analysis of the trial in late 2018, but suffered a setback when an independent assessment board said the data was not mature enough for an analysis of the overall response rate.

The FDA is also due to make a decision next year on umbralisib as a single agent for marginal zone lymphoma and follicular lymphoma.

The post TG Therapeutics challenges Roche with FDA filing for CLL drug appeared first on .

CVS Health Corporation names Neela Montgomery Executive Vice President and President of CVS Pharmacy/Retail, effective November 30, 2020. Montgomery will oversee the company’s 10,000 pharmacies across the United States. Montgomery, currently a Board Partner at venture capital firm Greycroft, most recently served as chief executive officer of furniture retailer Crate & Barrel and has nearly 20 years of global retail experience.

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The Cleveland Clinic and Amwell joint venture appoint Egbert van Acht as Executive Vice Chairman to the Board of Directors and Frank McGillin as CEO. Formed one year ago as a first-of-its-kind company to provide broad access to comprehensive, high-acuity care via telehealth, the company has made great progress scaling digital care through its MyConsult® offering. With an initial focus on clinical second opinions, the organization also offers health information and diagnosis on more than 2,000 different types of conditions including cancer, cardiac, and neuroscience issues.

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Healthcare industry veteran Dana Gelb Safran, Sc.D. has joined Well Health Inc. as Senior Vice President, Value-Based Care, and Population Health. In her new role, Dr. Safran will expand WELL’s uses to improve healthcare quality, outcomes, and affordability through partnerships with payers and Accountable Care Organization (ACO) providers.

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Talkdesk®, Inc., the cloud contact center for innovative enterprises appoints Cory Haynes to lead Talkdesk’s strategy for the financial service industry and Greg Miller to lead the strategy for healthcare and life sciences. Haynes and Miller are key members of the Talkdesk industries team led by Andrew Flynn, senior vice president of industries strategy for Talkdesk.

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Imprivata appoints Mark McArdle to Senior Vice President of Products and Design. Mr. McArdle has more than two decades of experience in software development, Software-as-a-Service (Saas), in Cybersecurity, and advanced products for the enterprise, SMB, and consumer markets.

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Eden Health names Jack Stoddard as executive chairman of its board of directors. Formerly serving in COO roles for Accolade and Haven, Stoddard brings two decades of healthcare innovation and operating experience to the board position, providing leadership, wisdom, and counsel during a time of monumental growth and adoption for the company. 

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Augmedix names Saurav Chatterjee Chief Technology Officer. Prior to joining Augmedix, he most recently served as Vice President of Engineering at Lumiata, Inc., where he led the engineering team that built a leading AI platform, focusing specifically on transforming, cleaning, enriching, featurizing, and visualizing healthcare data, and on building, deploying and operationalizing machine learning and deep-learning models at scale.

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Tridiuum, the nation’s premier provider of digital behavioral health solutions names Philip Vecchiolli has joined the company as Chief Growth and Strategy Officer. Vecchiolli, who brings over 30 years of experience to the new role, has a successful track record of leading business development for large and mid-size healthcare companies.

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Connect America appoints Janet Dillione as its new chief executive officer (CEO). Prior to joining Connect America, Dillione worked in the healthcare information services industry as CEO of Bernoulli Enterprise, Inc., GM of Nuance Healthcare, and CEO of Siemens Healthcare IT.

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Health Catalyst, Inc. announces that current Chief Financial Officer Patrick Nelli has been named President, effective January 1, 2021. Following Nelli’s promotion to the President role, Health Catalyst has named Bryan Hunt, current Senior Vice President of Financial Planning & Analysis, Chief Financial Officer, also effective January 1, 2021.

Two additional promotions, also effective January 1, 2021, include Jason Alger, Senior Vice President of Finance, to Chief Accounting Officer, and Adam Brown, Senior Vice President of Investor Relations, to Senior Vice President of Investor Relations and Financial Planning & Analysis. 

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Apervita hires health IT veteran Rick Howard as Chief Product Officer. In his role, Rick will oversee product vision, innovation, design, and delivery of Apervita’s digital platform, which enables digital quality measurement, clinical intelligence, as well as value-based contract monitoring and performance measurement.

Conversion Labs, Inc. appoints Roberto Simon to its board of directors and as the chair of its audit committee. Following his appointment, the board now has eight members, with six serving as independent directors. Mr. Simon currently serves as CFO of WEX (NYSE: WEX), a $6+ billion fintech services provider.

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PRA Health Sciences, Inc. appoints senior FDA official Isaac Rodriguez-Chavez, Ph.D., MHS, MS, as Senior Vice President, Scientific and Clinical Affairs. He will lead the company’s Global Center of Excellence for Decentralized Clinical Trial (DCT) Strategy. Dr. Rodriguez-Chavez’s responsibilities will involve the continued growth and development of PRA’s industry-leading decentralized clinical trial strategy, regulatory framework creation, and clinical trial modernization.

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Proprio appoints three global thought leaders to its Medical Advisory Board. Dr. Sigurd Berven, Orthopedic Surgeon and Professor at the University of California, San Francisco, Dr. Charles Fisher, Professor and Head of the Combined Neurosurgical & Orthopedic Spine Program at Vancouver General Hospital and the University of British Columbia, and Dr. Ziya Gokaslan, Professor and Chair of the Department of Neurosurgery at Brown University and Neurosurgeon-in-Chief at Rhode Island Hospital and The Miriam Hospital will apply their globally respected surgical and research expertise to the development of the Proprio navigation platform.

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Kaiser Permanente names Andrew Bindman, MD Executive Vice President and Chief Medical Officer.  In this role, Dr. Bindman will collaborate with clinical and operational leaders throughout the enterprise to help lead the organization’s efforts to continue improving the high-quality care provided to members and patients throughout Kaiser Permanente. Dr. Bindman will report directly to Kaiser Permanente chairman and CEO Greg A. Adams.

Greenway names Dr. Michael Blackman Chief Medical Officer at Greenway. Dr. Blackman will further support the company’s ambulatory care customers, ensuring providers are equipped with the solutions and services they need to improve patient outcomes and succeed in value-based care.

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Suki expands its leadership team with six key hires to support the company’s rapid commercial growth. Tracy Rentz, formerly Vice President of Implementation at Evolent Health, joins Suki as the Vice President of Customer Success and Operations to lead all customer operations, with a particular focus around deploying new Suki customers. Brian Duffy brings over 20 years of sales experience to Suki, joining the team as Director of Sales-East, after having most recently served as Regional Director at Qventus, Inc. Brent Jarkowski will also join Suki’s sales team this November as the Director of Sales-West, bringing over 15 years of experience in strategic relationship management. Brent joins Suki after serving as Senior Client Development Director at Kyyrus. Together, Brian and Brent will head the company’s efforts in building new partnerships across the country. And Josh Margulies, who previously served as the Director of Integrated Brand Marketing for the Jacksonville Jaguars, will serve as Suki’s new Senior Director of Field Marketing.

What You Should Know:

– AliveCor announced they received FDA clearance of new
algorithms for use with their personal EKG devices, KardiaMobile and
KardiaMobile 6L. These additional determinations will be available via a
software upgrade for the Kardia devices in 2021.

– The additional FDA-cleared algorithms double the number
of heart rhythm disturbances that AliveCor’s Kardia devices can detect,
broadening the number of patients who are able to use their remote monitoring
devices.

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AliveCor, an AI-based
personal ECG technology and provider of enterprise cardiology solutions, today
announced that the US FDA had given clearance to the company’s next generation
of interpretive ECG algorithms. AliveCor’s KardiaMobile and KardiaMobile 6L
devices, along with the Kardia app, allow users to take a 30-second ECG and
receive instant determinations of multiple cardiac conditions.

Why It Matters

This new FDA clearance positions AliveCor to deliver
AI-based remote cardiological services for the vast majority of cases when
cardiac patients are not in front of their doctor. AliveCor’s goal is to help
cardiologists efficiently provide the best possible 24/7 service to their
patients.

New Generation of AI-Powered Remote Cardiology

This new FDA 510(K) clearance provides detail and fidelity
unlike any previously seen in personal ECG devices including:

– A “Sinus Rhythm with Premature Ventricular
Contractions (PVCs)” determination if two or more ventricular ectopic
beats are detected. PVCs are a common occurrence where extra heartbeats
originate in the bottom chamber of the heart and occur sooner than the next
expected regular heartbeat. After the PVC beat, a pause usually occurs, which
causes the next normal heartbeat to be more forceful. When one feels the heart
“skip a beat,” it is this more forceful beat that is felt.

– A “Sinus Rhythm with Supraventricular Ectopy
(SVE)” determination if narrow-complex ectopy, such as premature atrial
contractions (PACs), are detected. PACs are similar to PVCs, but these beats
originate in the top chamber of the heart, however not in the heart’s natural
pacemaker, the Sinus Node.

– A “Sinus Rhythm with Wide QRS,” determination
for QRS intervals of 120ms or longer. 
Wide QRS indicates that the activation of the bottom chamber of the
heart is taking longer than expected. This could indicate a bundle branch block
in which there is a delay in the passage of heart’s electrical signals along
the bottom of the heart.

– A reduced number of “Unclassified” readings,
thereby giving users more reliable insight into their heart rhythms.

– Improved sensitivity and specificity on the company’s
“Normal” and “Atrial Fibrillation” algorithms, giving users
fewer false positives, fewer false negatives, and even greater confidence in
Kardia determinations.

– New visualizations, including average beat, PVC
identification, and a tachogram.

“Kardia AI V2 is the most sophisticated AI ever brought to personal ECG,” said AliveCor CEO Priya Abani. “This suite of algorithms and visualizations will provide the platform for delivery of new consumer and professional service offerings beyond AFib, by allowing a much wider range of cardiac conditions to be determined on a personal ECG device.”

Availability

Today, KardiaMobile and KardiaMobile 6L are the most
clinically validated personal ECG devices in the world, and provide instant
detection of Normal Sinus Rhythm, Atrial Fibrillation, Bradycardia, and
Tachycardia. The new determinations and services will be available in 2021.

Regeneron’s antibody cocktail is the latest COVID-19 drug to receive Emergency Use Authorization in the US, becoming the first therapy of this kind to become available.

The cocktail of casirivimab and imdevimab is still being investigated in trials but the FDA has enough data to grant a temporary licence while the pandemic continues.

Formerly known as REGN-COV2 or REGEN-COV2, the cocktail can be used for mild to moderate COVID-19 in adults and children at least 12 years of age and weighing at least 40 kg.

Doctors now have another option to choose from to combat the disease, following EUAs for drugs such as Eli Lilly’s baricitinib and Gilead’s Veklury (remdesivir).

To be eligible for Regeneron’s combination, patients must have received positive results of direct SARS-CoV-2 viral testing and be considered at high risk for progressing to severe COVID-19 and/or hospitalisation.

Clinical evidence from Regeneron’s outpatient trial suggests that monoclonal antibodies such as casirivimab and imdevimab have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load.

As part of the Operation Warp Speed project to rapidly develop therapies and vaccines against the disease, the US government and Regeneron a supply agreement for the cocktail, which was famously used to treat president Donald Trump after he became infected.

Regeneron will coordinate with state authorities to allocate the cocktail on a weekly basis, based on prevalence of the disease in each state.

The first 300 doses will be provided at no cost to patients, although hospitals and clinics may add their own fees.

AmerisourceBergen will be the first national distributor to begin delivering the therapy.

Earlier this month, Regeneron had to modify a trial protocol for the therapy after independent safety experts said it should not be given to high-risk patients after an undisclosed safety issue emerged in testing.

In late October, Eli Lilly said it wouldn’t resume a trial of its rival antibody therapy in hospitalised patients after National Institutes of Health researchers said it wouldn’t help.

AstraZeneca is developing a long-acting antibody therapy combination in the US and other countries to prevent infection happening and as a therapy for those already infected.

Regeneron’s antibodies were designed to combat SARS-CoV-02 using the company’s proprietary genetically modified mice, which have been engineered to have a human immune system.

The antibodies bind non-competitively to the virus’s spike protein, which diminishes the ability of mutant viruses to escape treatment and protects against spike variants that have arisen in the human population.

The post Regeneron’s COVID-19 antibody cocktail gets US emergency licence appeared first on .

Doctors in the US are to get another option to treat COVID-19 after the FDA granted an Emergency Use Authorization (EUA) for Eli Lilly’s baricitinib.

Baricitinib is the active ingredient in Lilly’s rheumatoid arthritis drug Olumiant and is the latest example of a drug being repurposed to combat COVID-19.

Most of the drugs attempt to tackle the inflammation that can cause complications in patients with severe disease.

While not all the attempts to get existing drugs to work in COVID-19 have been successful, the FDA said there is enough evidence to justify the emergency use for baricitinib in adults and children aged over two who are hospitalised with the disease and require oxygen therapy or invasive mechanical ventilation.

This temporary authorisation, which only applies during the COVID-19 crisis, is based on data from the Adaptive COVID-19 Treatment Trial (ACTT-2) conducted by the National Institute of Allergy and Infectious Diseases, part of the government funded National Institutes of Health.

Patients treated with baricitinib in combination with Gilead’s remdesivir had a significant reduction in median time to recovery from eight to seven days (12.5% improvement) compared to remdesivir.

The FDA’s decision came as the World Health Organization has advised against using Gilead’s Veklury (remdesivir) to treat hospitalised patients with COVID-19, no matter how severe their illness, after saying new evidence no longer supports its use.

The update is in contrast to the FDA, which has approved remdesivir to treat patients hospitalised with COVID-19 last month, after it was granted and Emergency Use Authorization earlier in the year.

But in the new guidance published in the BMJ, the WHO’s experts recommend against using remdesivir in non-severe, severe, or critical patients, citing weak evidence.

However the panel said there is strong evidence in favour of using corticosteroids in patients with severe or critical disease.

The latest guidance was produced using a systematic review of latest evidence.

According to the panel of experts who drew up the guideline, the evidence suggests that remdesivir has no important effect on mortality, need for mechanical intervention, time to clinical improvement and other outcomes.

The guidance was triggered by findings of the WHO’s SOLIDARITY trial last month, which reported results treating hospitalised COVID-19 patients with remdesivir, the steroid hydroxychloroquine, and the antiviral combination lopinavir-ritonavir.

Findings of SOLIDARITY diverged from evidence gathered in the US – last month data from the National Institutes of Health-backed ACTT-1 trial study showed Veklury resulted in five days’ faster recovery in patients hospitalised with COVID-19.

There was a non-statistically significant trend towards a reduction in mortality, with the effect being more pronounced in patients who were on low-flow oxygen at baseline.

In a statement, Gilead noted that Veklury has been included in guidelines from the NIH and Infectious Diseases Society of America, as well as in national guidelines in Japan, the UK, and Germany.

Gilead added: “These recommendations are based on the robust evidence from multiple randomised, controlled studies published in peer-reviewed journals that demonstrate the clinical benefits of Veklury, such as significantly faster recovery, which can free up limited hospital resources.

“We are disappointed the WHO guidelines appear to ignore this evidence at a time when cases are dramatically increasing around the world and doctors are relying on Veklury as the first and only approved antiviral treatment for patients with COVID-19 in approximately 50 countries.”

 

The post Lilly’s baricitinib granted US emergency use in COVID-19 appeared first on .

The San Francisco-based company aims to complete at least one Phase III study by 2025, and it is weighing an IPO next year to fund more.

The US FDA has approved 4 NDAs and 1 BLA in Oct 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 90 novel products so far in 2020, including 5 in Oct 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 5 new drugs approved by the US FDA in Oct 2020.

Regeneron’s Inmazeb (atoltivimab, maftivimab, and odesivimab) Received the US FDA’s Approval as the First Treatment for Ebola

Published: Oct 15, 2020 | Tags: Approval, Atoltivimab, Ebolavirus, Firs,t Inmazeb, Maftivimab, Odesivimab, Receives, Regeneron, Treatment, US FDA

• The approval is based on the PALM trial assessing Inmazeb vs Zmapp and remdesivir in 681 adult and pediatric patients including newborns of mothers who have tested positive for the infection. The study demonstrated 1EPs of mortality @28days (33.5% vs 51.3%) and 2EPs of reduction in days until the virus was undetectable in the bloodstream
• As per the agreement signed in Jul’2020, Regeneron will deliver a number of Inmazeb treatment doses for 6yrs. to the BARDA
• Inmazeb is a triple antibody cocktail consisting of 3 mAbs (atoltivimab, maftivimab & odesivimab, 50 mg each /kg) that bind to different, non-overlapping epitopes on Zaire ebolavirus glycoprotein

Nevakar’s Ephedrine Sulfate Injection Received the US FDA’s Approval as Ready-To-Use Vials

Published: Oct 22, 2020 | Tags: Nevakar, Ephedrine Sulfate, Injection, Receives, US FDA, Approval, Ready to Use, Vials

• Nevakar received US FDA’s approval to market Ephedrine Sulfate Injection (50mg/10 ml) in a ready to use vial presentation and it is the 1^st approval under collaboration bw Nevakar and Endo for the development of sterile injectable product in the US and Canada
• Nevakar to develop and obtain FDA approval for these products and Par Pharmaceuticals Sterile Products division will launch and distribute the products
• The company is focused on developing and commercializing innovative products to address unmet medical needs, thereby improving patient care and quality of life

Gilead’s Veklury (remdesivir) Received the US FDA’s Approval as the First Treatment for COVID-19

Published: Oct 23, 2020 | Tags: COVID-19, Gilead, Receives, Remdesivir, US FDA, Approval, Veklury

• The approval is based on three studies including P-lll ACTT-1 study assessing the efficacy and safety of a 10-day treatment course of Veklury vs PBO in 1063 hospitalized patients with confirmed SARS-CoV-2 infection and mild, moderate or severe COVID-19 receiving the treatment with SOC. The other two studies include two P-II OLE studies (SIMPLE-Severe trial & SIMPLE-Moderate trial)
• ACTT-1 trial results: improvement in time to recovery in overall study population & in patients who required oxygen (10 vs 15days & 11 vs 18days); reduction in disease progression in patients needing oxygen, reduction in new mechanical ventilation or ECMO (13% vs 23%)
• Additionally, FDA also issued a new EUA for the use of Veklury to treat hospitalized pediatric patients aged <12yrs. weighing at least 3.5 kg or hospitalized pediatric patients weighing 3.5 kg to <40 kg with suspected or laboratory confirmed COVID-19 for whom use of an IV agent is clinically appropriate. Veklury is now the 1^st and only approved COVID-19 treatment in the US

Kala Pharmaceuticals’ Eysuvis (loteprednol etabonate ophthalmic suspension) Received US FDA’s Approval for Dry Eye Disease

Published: Oct 27, 2020 | Tags: Kala Pharmaceuticals, Eysuvis, Loteprednol, Etabonate, Ophthalmic, Suspension, US FDA, Dry Eye Disease

• The approval is based on results from four clinical trials, including three P-III trials and one P-II trial, that demonstrated significant improvements in both the signs and symptoms of dry eye disease
• The approval has made Eysuvis, the 1^st ocular corticosteroid for the treatment of dry eye disease and the 1^st drug approved specifically for the short-term (up to 2 wks.) treatment of the signs and symptoms of dry eye disease
• Eysuvis utilizes Kala’s Ampplify mucus-penetrating particle (MPP) drug delivery technology to enhance penetration of loteprednol etabonate into target tissue on the ocular surface. The company plans to launch Eysuvis in the US by the end of the year 2020

Chiesi’s Bronchitol (mannitol) Inhalation Powder Received US FDA’s Approval for Cystic Fibrosis

Published: Oct 30, 2020 | Tags: Chiesi, Bronchitol, Mannitol, Inhalation, Powder, Receives, US FDA, Approval, Cystic Fibrosis, Pulmonary

• In the three large-scale global clinical trials assessing Bronchitol in 761 patients, sustained improvement in FEV1 (Forced Expiratory Volume) with Bronchitol use vs. control group was observed
• Bronchitol is currently approved and marketed in Australia, Italy, Germany, Russia, and several other countries. Additionally, the company anticipates launching Bronchitol in the US in Mar 2021
• Bronchitol (mannitol) inhalation powder is a sugar alcohol and also the 1^st and only inhaled dry powder indicated as add-on maintenance therapy to improve pulmonary function in CF patients aged 18 yrs. of age and older

Related Post: Insights+: The US FDA New Drug Approvals in September 2020

The post Insights+: The US FDA New Drug Approvals in October 2020 first appeared on PharmaShots.

The FDA has agreed to expedite development of two rare disease drugs from Sanofi.

The most significant of the two announcements is the Priority Review for avalglucosidase alfa, a potential new therapy for Pompe disease.

The review period will be shortened from the standard ten months to six months or less, and the French pharma said the FDA will make the regulatory decision before May 18th.

These faster reviews are reserved for drugs that could be a significant improvement in safety or efficacy over standard care.

Avalglucosidase alfa is an investigational enzyme replacement therapy designed to improve the delivery of acid alpha-glucosidase (GAA) enzyme to muscle cells.

The FDA will review data from the phase 3 COMET trial in patients with late-onset Pompe disease.

Also in the file will be data from the phase 2 mini-COMET trial testing safety and efficacy in patients with infantile-onset Pompe disease previously treated with standard enzyme replacement therapy, alglucosidase alfa.

European regulators began their review of the data last month.

Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme GAA, which results in build-up of complex sugars (glycogen) in muscle cells throughout the body.

The accumulation of glycogen leads to irreversible damage to the muscles, including respiratory muscles and the diaphragm muscle supporting lung function, and other skeletal muscles that affect mobility.

In a separate announcement, Sanofi said the FDA granted Fast Track Designation for its oral investigational Bruton’s tyrosine kinase inhibitor rilzabrutinib, which is in development for immune thrombocytopenia.

Fast Track designation gives developers more support from the FDA for potentially important new drug for serious conditions and can lead to a Priority Review if clinical trial data looks promising enough.

Fast-tracked drugs can also be granted a tentative Accelerated Approval based on earlier data, with a full approval granted on the basis of information from a larger trial.

Sanofi has already begun a phase 3 trial of rilzabrutinib in the disease caused by destruction and impairment of platelet production by the immune system following supportive phase 1/2 results.

Rilzabrutinib is being investigated in a phase 3 trial for pemphigus, an immune mediated disease characterized by blisters in mucous membranes and skin. Sanofi has also begun a phase 2 study in the autoimmune condition IgG4 disease.

The post FDA to expedite development of two Sanofi rare disease drugs appeared first on .

The Food and Drug Administration (FDA) on Tuesday issued an emergency use authorization for the first COVID-19 diagnostic at-home test.

The move will not have an immediate impact on in-home care providers, but it could give them one more tool in their pandemic toolbox in weeks to come.

The FDA specifically signed off on the Lucira COVID-19 “All-In-One Test Kit,” a molecular test that provides results in 30 minutes or less. Currently, the test is authorized for use by prescription only.

In an announcement on the authorization, U.S. Health and Human Services (HHS) officials described the news as another major advancement for the country’s COVID-19 testing ecosystem. The newfound availability of the Lucira test “represents a leap ahead” in terms of bringing the convenience of home testing to Americans, they noted.

“Making it possible for Americans to do their own rapid COVID-19 self-test at home by prescription is the latest addition to our constantly expanding arsenal of COVID-19 testing options,” HHS Secretary Alex Azar said.

Moving forward, the Lucira All-In-One Test Kit test will be authorized for home use with self-collected nasal swab samples in individuals age 14 and older who are suspected of COVID-19 by their health care provider.

Although the idea is for individuals to test themselves, home health and home care professionals will likely play a huge role in supporting their high-risk patient populations during the process. If agencies can secure the newly authorized Lucira test themselves, it could also serve as a safer and more accurate method for screening in-home workers.

Due to accessibility and cost challenges, many agencies are using digital screening tools to best identify COVID-positive workers.

“Today’s authorization for a complete at-home test is a significant step toward FDA’s nationwide response to COVID-19,” Dr. Jeff Shuren, director of the FDA’s Center for Devices and Radiological Health, said. “A test that can be fully administered entirely outside of a lab or health care setting has always been a major priority for the FDA to address the pandemic.”

The Lucira at-home test works by swirling the self-collected sample swab in a vial that is then placed in the test unit. In 30 minutes or less, the results can be read directly from the test unit’s light-up display, which shows whether a person is positive or negative for the coronavirus.

Lucira said in a news release that the test will be available on a limited basis while it builds up further manufacturing capabilities.

“We look forward to proactively working with test developers to support the availability of more at-home test options,” Shuren continued.

Aging services providers across the continuum of care continue to face COVID-19 hardships.

In a new survey from Washington, D.C.-based trade group LeadingAge, for example, over half of respondents said they currently have diagnosed or suspected COVID-19 cases among residents or clients. Nearly all of them — 91% — said they have current diagnosed or suspected cases among staff.

The survey included 193 respondents from nursing homes, senior living communities, home health agencies and other senior care organizations. 

While the FDA’s emergency use authorization for the Lucira at-home test is encouraging, some experts have called for caution, pointing out that at-home tests aren’t always as accurate as those carried out in a facility or office.

“Some of these tests have never been checked for reliability or accuracy,” Liz Richardson, who studies the regulation of medical products for the Pew Charitable Trusts, told the Los Angeles Times. “Even very good tests sometimes get it wrong.”

The Lucira All-In-One Test Kit test costs about $50, according to USA Today.

The post FDA Signs Off on First At-Home Coronavirus Test appeared first on Home Health Care News.

The company is the second this month to report promising results for a Covid-19 vaccine even as the virus rages across the U.S.

What You Should Know:

– White house coronavirus task force doubles down on rapid testing strategy to fight the coronavirus as some states say they don’t have the supplies to comply with the federal government’s advice.

– This article was originally published by the Center for Public Integrity, a nonprofit investigative news organization based in Washington, D.C.

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The White House coronavirus task force is doubling down on part of its strategy for halting the spread of the virus: widespread use of rapid tests.

As COVID-19 cases and hospitalizations reached record highs, the task force last week issued advice to a number of governors: Begin using rapid tests on all young people, even those without COVID-19 symptoms, in counties with exploding numbers of cases.

The guidance came as state officials and some scientists express doubt about rapid testing strategies. The rapid tests, known as antigen tests, give results faster but are more likely to return false negatives than lab-based polymerase chain reaction (PCR) tests, and the Food and Drug Administration has not yet approved the rapid tests for use in patients who show no symptoms.

But the nation’s testing czar, Adm. Brett Giroir, who also sits on the task force, gave a robust defense of the strategy in emailed responses to questions from the Center for Public Integrity.

“The testing of asymptomatic individuals with rapid antigen tests is vital,” Giroir wrote. “The data are really to a point that those who argue against asymptomatic testing … are more influenced by politics, financial self-interest of their industry, or lack of knowledge, than they are by the evidence of how to support control of the pandemic.”

The task force previously urged states to use the rapid tests to screen certain groups, such as teachers or health-care workers. The federal government is distributing to states 150 million antigen tests made by Abbott Laboratories.

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In his email Giroir pushed back on comments from another member of the task force, Dr. Scott Atlas, who told The New York Times last month that testing asymptomatic people would amount to “destroying the workforce.” Atlas, a favorite adviser of President Donald Trump, holds views repudiated by many scientists studying the pandemic and has been the source of rifts within the task force.

“No credible public health expert would suggest that it is good practice to allow an infectious person — whether symptomatic or asymptomatic — into the community or workforce,” Giroir wrote. “The best way to keep America working and Americans in school and employed is to control the spread of the virus.”

But it’s not clear that states can keep up with the federal advice to deploy rapid tests more broadly.

Of the 16 states that responded to questions from Public Integrity, 11 said they were using rapid tests to screen special populations, such as nursing-home residents or health-care workers, but only four indicated plans to use them on broader populations. Several said they didn’t have enough tests to screen the general public. 

“We do not have enough supplies to use for general population testing,” said Taylor Gage, spokesman for Nebraska Gov. Pete Ricketts. “The state is using all the tests — we do not have a reserve or backlog on hand.”

Manpower is another dilemma. Some states don’t have enough school nurses to deploy antigen test screening in schools, let alone the general population, said Marcus Plescia, chief medical officer at the Association of State and Territorial Health Officials. 

“Nobody’s against that but some of it is just pure logistics. If you want us to do that, where are the tests?” Plescia said. “It’s just there are sort of on-the-ground challenges to rolling some of the stuff out with the speed that the administration would like.”

Another hurdle to testing 18-40-year-olds: the millennials themselves. Young people so far have not responded well to efforts such as contact tracing, said Lori Tremmel Freeman, CEO of the National Association of County and City Health Officials.

“Getting them to test frequently, if this is the population we’re targeting, will require more than just putting the tests out there,” she said. “It requires, really, a campaign to change public sentiment around the disease.”Preview mode is not supported for related articles. Please change to Edit mode by clicking the pencil icon in the toolbar above.

That’s something the White House has not done. The president lately has been mostly silent about coronavirus testing, after weeks of falsely blaming high case counts on increased testing. And the task force’s recent endorsements of antigen testing are contained in reports to governors that the White House does not make public.

“We’re hearing some of the right words, the right public health tactics, the right strategies emerging from the White House task force behind the scenes, but we really have to turn that internal to an external, public-facing messaging campaign,” Tremmel Freeman said.  “Nobody really knows what the strategy is or why it’s important.”

In addition to concerns about “how,” some states are hung up on whether they should use rapid tests on asymptomatic people when the FDA has not approved them for that use. 

Several states still have official health guidance that contradicts the White House view of rapid tests — Virginia policy, for example, says PCR testing should be used whenever possible. At least three states have discouraged the antigen tests’ use in nursing homes. The federal government has shipped more than 13 million rapid tests to nursing homes, but a Kaiser Health News investigation found that roughly 38% of the nation’s nursing homes have yet to use them.

“There’s quite a bit of uncertainty and things we’re sorting out that have caused most of us to move forward with some caution,” Plescia said. The health officials in his organization “just have some anxieties around the accuracy of the test.”

But several scientists who spoke to Public Integrity said the administration’s push to test more asymptomatic young people using antigen tests is a good idea.

“We have to do more to break these chains of transmission,” said Gigi Gronvall, an immunologist at the Johns Hopkins Center for Health Security. “You could be saving somebody by testing them and getting them to isolate.”

Some cautioned that jurisdictions who deploy the tests more broadly need to have clear plans to ensure positive people isolate — which may be a challenge for those who need to show up to jobs or risk losing them.

“Advocating for [antigen tests’] use really broadly without a plan for what to do with the results is going to create problems,” said Susan Butler-Wu, an associate professor of clinical pathology at the University of Southern California’s medical school. “You have to have a plan for what to do when it’s positive, and you have to have a lot of education around what to do if it’s negative.”

Giroir said states must figure out how exactly to test wide swaths of their populations, though he said weekly testing at universities has shown the best results. 

“States and counties need to employ strategies specific to their populations including education and resources,” he wrote.

---

KHN Editor-in-Chief Elisabeth Rosenthal discussed how to manage unexpected health care costs with CBSN on Wednesday.

• Click here to watch Rosenthal on CBSN

KHN chief Washington correspondent Julie Rovner discussed the Affordable Care Act case before the Supreme Court with WBEZ’s “Reset” and WDET’s “Detroit Today” on Tuesday and with WHYY’s “Radio Times” on Wednesday.

• Click here to hear Rovner on WBEZ
• Click here to hear Rovner on WDET
• Click here to hear Rovner on WHYY
• Read Rovner’s “What to Know as ACA Heads to Supreme Court — Again“

KHN partnerships editor and senior correspondent Mary Agnes Carey discussed the ACA Supreme Court case on Newsy’s “Morning Rush” on Tuesday and on Connecticut Public Radio’s “Where We Live” on Nov. 6.

• Click here to watch Carey on Newsy
• Click here to hear Carey on Connecticut Public Radio

On Thursday, KHN correspondent Rachana Pradhan discussed with Newsy the challenges President-elect Joe Biden faces in trying to seat Food and Drug Administration leadership quickly to deal with the pandemic.

• Click here to watch Pradhan on Newsy
• Read Pradhan’s “As Nation Awaits Vaccine, Biden Is Under Pressure to Name New FDA Chief ASAP“

KHN senior correspondent Sarah Jane Tribble discussed KHN’s “Where It Hurts” podcast with Kansas Public Radio’s “KPR Presents” on Nov. 1.

• Click here to hear Tribble on Kansas Public Radio
• Tribble hosts “No Mercy,” the first season of “Where It Hurts.” Click here to listen to Chapter 1.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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What You Should Know:

– Bill & Melinda Gates Foundation awards Caption
Health a $4.5M grant to support the development of an AI-guided lung ultrasound
system.

– The grant from the Bill & Melinda Gates Foundation
will be leveraged to create new AI technology that allows medical professionals
without prior ultrasound experience to perform lung ultrasounds, expanding
access to quality medical care.

---

Caption Health, a leading medical
artificial intelligence (AI) company, today announced that it has received
a grant from the Bill & Melinda
Gates Foundation in the amount of $4.95 million to support the development
of innovative AI technology for lung ultrasound. The grant was awarded to
Caption Health by the foundation due to the need to further develop solutions
that enable timely and accurate diagnosis of pneumonia, the leading killer of
children under 5, in resource-limited settings with a shortage of highly
trained physicians. 

Caption Health already has the first and only FDA cleared AI
platform that enables medical professionals without prior ultrasound experience
to perform cardiac ultrasound exams (Caption
AI). Like cardiac ultrasound, performing lung ultrasound requires a high
level of clinical skill and specific expertise, which has limited its broad
adoption. With this grant, Caption Health will be able to expand its
first-in-class AI technology to lung ultrasound, providing healthcare workers with
real-time guidance to acquire diagnostic-quality images for each lung zone and
automated interpretation to detect key lung pathologies.

Why It Matters

“Ultrasound can be challenging for clinicians without prior experience because it requires skill in both obtaining and interpreting images. Caption Health is the leader in developing artificial intelligence that combines image acquisition and interpretation to enable clinicians to perform ultrasound regardless of skill level,” said emergency medicine physician Dr. Chris Moore, Associate Professor of Emergency Medicine, Chief of the Section of Emergency Ultrasound, and Director of the Emergency Ultrasound Fellowship at Yale. “Expanding this AI to lung ultrasound and putting it in the hands of clinicians could have profound implications for the diagnosis and treatment of pneumonia, a leading cause of death in our youngest global citizens, as well as for COVID-19 and other lung conditions.”

Lung ultrasound enables the detection of a range of
pulmonary pathologies such as pneumonia and other consolidations, pulmonary
edema, pleural effusions and pneumothorax. Furthermore, it is non-invasive,
portable and does not expose recipients to harmful radiation. As the cost of
miniaturizing ultrasound hardware decreases, Caption Health’s AI technology
solves the remaining challenge currently limiting ultrasound’s widespread use:
enabling clinicians without lengthy specialized training to acquire and interpret
diagnostic-quality ultrasound images. 

As COVID-19 cases rise, lung ultrasound is playing a
critical role in the triage and monitoring of these patients. When patients
arrive in the Emergency Department with suspicion of COVID-19, lung ultrasound
can be used for early detection of pulmonary involvement, offering higher sensitivity than chest x-rays. For those who are
diagnosed with COVID-19, lung ultrasound can be used to grade the degree of
pulmonary involvement, and to monitor changes over time. Caption Health’s AI
technology will expand access to this powerful diagnostic tool by enabling
medical professionals without prior experience in lung ultrasound to perform
these exams, and could eventually lead to lung ultrasound becoming a routine
part of point-of-care assessments.

 “Pulmonary health and cardiovascular health are closely intertwined,” said cardiologist Dr. Randolph Martin, FACC, FASE, FESC, Chief Medical Officer of Caption Health. “Abnormalities or disease states in the lungs can directly cause prominent abnormalities of cardiac function, just as disease states in the heart can lead to marked abnormalities in the lungs. By taking our unique methodology for developing breakthrough AI for cardiac imaging and applying it to lungs, we will continue to broaden the impact we can have in helping with the management of patients with conditions affecting these two vital systems.”

Future Research Plans

Having demonstrated extensive clinical validation for its
cardiac ultrasound technology, including a multi-center prospective clinical
study and numerous published abstracts, Caption Health intends to seek similar
validation for its AI lung ultrasound technology to demonstrate the ability of
the technology to equip non-specialists to perform lung ultrasound exams.

Eli Lilly has said it will start shipping supplies of its COVID-19 drug bamlanivimab immediately, after claiming emergency-use authorisation (EUA) for the antibody.

The AbCellera-partnered drug, previously known as LY-CoV555 or LY3819253, can be used to treat mild-to-moderate COVID-19 in patients 12 years and older, who are at high risk for progressing to severe disease that might require hospitalisation.

President Trump has previously pledged to make antibody drugs available for free to anyone who needs them, and his administration has already signed a $375 million contract with Lilly to supply 300,000 doses of the drug within two weeks of the EUA.

The US has also taken an option on another 650,000 doses of the drug between now and the end of June 2021.

Lilly chief executive David Ricks has also said that patients should have no out-of-pocket costs for the drug, although healthcare facilities may charge a fee for the antibody’s intravenous administration.

The EUA as well as the US government contract came despite the results of the ACTIV-3 trial run by the National Institute of Allergy and Infectious Diseases (NIAID), which halted enrolment in the bamlanivimab arm because data to date suggested the drug was unlikely to be effective in hospitalised patients.

The FDA approved a 700mg single IV dose of the drug based on the phase 2 BLAZE-1 trial, which showed a reduced rate of hospitalisation in patients at high risk of COVID-19 progression who were treated with the antibody in the outpatient setting.

The NIAID is also running another study of Lilly’s antibody, ACTIV-2, which involves outpatients with mild-to-moderate COVID-19 symptoms.

Lilly says it will ship supplies of the drug to AmerisourceBergen, which will handle distribution once a week in accordance with the US government’s allocation plans. Where the drug is distributed will depend on confirmed COVID-19 cases across the US over the prior seven days.

Bamlanivimab – an antibody that neutralises the SARS-CoV-2 coronavirus – joins Gilead’s antiviral Veklury (remdesivir) on the list of drugs with EUAs for COVID-19, but is the first to be cleared for use outside hospitals.

Veklury’s EUA and subsequent full approval by the FDA covers the treatment of patients with COVID-19 who already require hospitalisation. The FDA has also issued EUAs for convalescent plasma for hospitalised patients, as well as for certain drugs that don’t target the virus directly but are used to support patients whilst in acute care.

Lilly notes that bamlanivimab should be administered as soon as possible after a positive COVID-19 test and within 10 days of symptom onset.

The company says it hopes to be able to produce up to one million doses of bamlanivimab 700mg by the end of 2020, “for use around the world through early next year.”

The FDA is meanwhile reviewing an EUA for Regeneron’s COVID-19 antibody therapy REGN-COV2, focusing on patients with mild to moderate disease who are at risk of progressing, and a decision on that could be made shortly.

REGN-COV2 also seems at risk of falling short in hospitalised patients, after a safety concern led to the pause of enrolment in a clinical trial in this setting earlier this month.

The post Lilly to ship COVID-19 drug straight away after FDA green light appeared first on .

What You Should Know:

– Cardiopulmonary digital health company Eko raises $65M
in Series C funding to close the gap between virtual and in-person heart and
lung care.

– The latest round of funding will enable Eko to expand
in-clinic use of its platform of telehealth and AI algorithms for disease
screening and to launch a monitoring program for cardiopulmonary patients at
home.

Eko, a
cardiopulmonary digital
health company,
today announced $65 million in Series C funding led by Highland Capital
Partners and Questa Capital, with participation from Artis Ventures, DigiTx
Partners, NTTVC, 3M Ventures, and other new and existing investors. The new
funding will be used to expand in-clinic use of the company’s platform of telehealth
and AI
algorithms for disease screening, and to launch a monitoring program for
cardiopulmonary patients at home.

Eko was founded in 2013 to improve heart and lung care for
patients through advanced sensors, digital technology, and novel AI algorithms.
The company reinvented the stethoscope and introduced the first combined
handheld digital stethoscope and electrocardiogram (ECG). Eko’s FDA-cleared AI
analysis algorithms help detect heart rhythm abnormalities and structural heart
disease. Eko seeks to make AI analysis the standard for every physical exam. The
company recently launched Eko AI and Eko Telehealth to combat the needs of the COVID-19
pandemic.

Eko Telehealth delivers:

– AI-powered and FDA-cleared identification of heart murmurs
and atrial fibrillation (AFib), assisting providers in the detection and
monitoring of heart disease during virtual visits

– Lung and heart sound live-streaming for a thorough virtual
examination

– Single-lead ECG live-streaming, enabling providers to
assess for rhythm abnormalities

– Embedded HIPAA-compliant video conferencing, or can work
alongside the video conferencing platform a health system has in place

Symptoms of valvular heart disease and AFib often go
undiagnosed during routine physical exams. With the development of Eko’s AI
screening algorithms, clinicians are able to harness state-of-the-art machine
learning to detect heart disease at the earliest point of care regardless if
the patient visit is in-person or remote.

“We are thrilled that our new investors have joined our journey and our existing investors have reaffirmed their support for Eko,” said Connor Landgraf, CEO and co-founder at Eko. “The explosion in demand for virtual cardiac and pulmonary care has driven Eko’s rapid expansion at thousands of hospitals and healthcare facilities, and we are excited for how this funding will accelerate the growth of our cardiopulmonary platform.”

The FDA may have been minded to approve Biogen and Eisai’s Alzheimer’s candidate aducanumab, but its clinical advisors have little doubt that the evidence for the drug is lacking.

The much-anticipated advisory committee meeting held on Friday to discuss the marketing application for aducanumab proved to be a fractious affair, with both the companies’ data – and some of the FDA’s interpretation of it – under fire by panellists.

After hours of debate, the verdict from the experts was pretty unequivocal – 10 panellists voted against the main study supporting the drug, with just one saying they were uncertain about the data. Not one was convinced that the clinical trial results proved that the drug was effective.

The resounding rejection of the clinical data submitted in support of the marketing application for aducanumab came after the FDA published briefing documents that indicated the agency’s clinical reviewer was in favour of approval, calling the results “persuasive” despite a fairly damning assessment by the agency’s statistical expert in an appendix.

Aducanumab is vying to become the first Alzheimer’s drug designed to tackle an underlying cause of the disease, and the first new treatment for the neurodegenerative disease in almost two decades.

The FDA has been working closely with Biogen on the application and could still decide to approve the drug despite its experts’ reservations. That said, the balance of the votes makes that outcome unlikely – despite impassioned testimony during the virtual meeting by patient organizations.

The agency is due to make a decision by 7 March, and a review is also underway at the European Medicines Agency (EMA). Analysts have predicted it could become a multibillion-dollar blockbuster if approved, although at the moment that looks unlikely without another clinical trial.

An approval could also be seen as vindication for the hypothesis that tackling the amyloid plaques that are seen in the brains of people with the disease helps to slow the onset of symptoms. Many people had given up on the hypothesis after dozens of failed studies involving anti-amyloid drugs.

Biogen ran two identically-designed phase 3 studies of aducanumab – EMERGE (Study 302) and ENGAGE (Study 301) – to try to demonstrate that the anti-amyloid antibody could slow down the loss of cognitive function in people with mild cognitive impairment due to Alzheimer’s.

Last year, it called a halt to the studies after a futility analysis found it unlikely that aducanumab would show an effect on cognitive decline, but a few months later said that EMERGE was positive after all, and would form the basis of a marketing application with a phase 1b extension study – called PRIME – used as supportive evidence.

After a positive discussion of the results from Billy Dunn, acting director of the FDA’s office of neuroscience, the panel voted on three aspects of the data set.

In the first, they came down 8 to 1, with 2 uncertain, that the EMERGE trial could not be viewed on its own as providing strong evidence supporting aducanumab’s efficacy without taking into account the negative ENGAGE data.

They then voted 7 against and 4 uncertain on a question asking whether PRIME provide supportive evidence of the effectiveness of aducanumab in Alzheimer’s, before rejecting the premise that EMERGE could serve as primary evidence supporting the efficacy of the antibody.

Overall, the conclusion was that a negative interpretation of the data is just as likely as the positive one put forward by the trial sponsors, with one suggesting Biogen had shot first, and painted a bullseye later.

Biogen issued a short statement after the meeting in which CEO Michel Vounatsos said: “We appreciated the opportunity to share our data with the advisory committee, and we will continue to work with the FDA as it completes its review of our application.”

Trading in the biotech’s stock was halted ahead of the advisory committee meeting, but looks likely to be under considerable pressure when it resumes as it has been fluctuating wildly in reflection of the fortunes of aducanumab.

Biogen has a lot riding on aducanumab, as its pipeline has suffered recent setbacks including the failure of multiple sclerosis candidate opicinumab and a gene therapy for spinal muscular atrophy (SMA) at a time when big selling SMA drug Spinraza (nusinersen) is facing increased competition.

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President-elect Joe Biden made COVID-19 a linchpin of his campaign, criticizing President Donald Trump’s leadership on everything from masks and packed campaign rallies to vaccines.

That was the easy part. Biden now has the urgent job of filling top health care positions in his administration to help restore public trust in science-driven institutions Trump repeatedly undermined, and oversee the rollout of several coronavirus vaccines to a skeptical public who fear they were rushed for political expediency.

At the top of that list is a new commissioner of the Food and Drug Administration, an agency where Biden faces immense pressure to move faster than any other modern president as the pandemic rages and COVID deaths are expected to surge through the winter. That agency and its beleaguered personnel will be relied on to give the green light to vaccines and therapeutics to fight the COVID pandemic.

Biden is expected to swiftly announce his choices to lead the FDA and the Centers for Disease Control and Prevention, given their importance in informing the federal government’s COVID strategy, according to interviews with Biden advisers, former agency officials and Democrats with knowledge of the transition team’s inner workings. But how soon they’ll be able to begin work after Biden’s Jan. 20 inauguration is unclear.

The CDC director does not need Senate confirmation, avoiding a hurdle that could slow that process. That is not the case for the FDA commissioner, who now appears increasingly likely to face a Republican-controlled Senate that may not be as keen as Democrats to swiftly clear Biden’s nominees. As a result, even if Biden moves at breakneck speed to replace outgoing Commissioner Stephen Hahn, it could be weeks after Biden is in the White House before his pick could get to work.

In the meantime, the FDA will face critical decisions about vaccines needed to help put the nation on its path out of the pandemic. Biden will have to rely on a temporary head of the FDA to steer the 17,000-employee agency during one of the most challenging times in its history.

“It’s not ideal timing, for sure,” a former FDA official said. “It’s a huge job.”

The transition of power will occur at one of the most high-profile times for the FDA, as it vets multiple coronavirus vaccine candidates that could reach the public before the inauguration. The Trump administration could oversee emergency authorizations of initial vaccines from two front-runners, Pfizer and Moderna, that would be prioritized for health care workers and other groups at higher risk of severe COVID complications. But other companies’ vaccines that could be available for many more Americans — such as teachers, adults at lower risk of severe health consequences if they get sick, and children — are all but certain to fall under Biden’s FDA for review because the data on safety and efficacy isn’t expected until next year.

FDA’s credibility in vetting the safety and benefits of COVID products has been in question for months, fueled by Hahn’s inaccurate statements about certain treatments for sick patients. Further, infighting between officials there and political appointees at the White House and the Department of Health and Human Services persisted even in the weeks leading up to the election, with HHS Secretary Alex Azar openly plotting Hahn’s removal because of disagreements over vaccine standards, Politico reported in October.

In September, eight senior FDA officials who have served in multiple administrations took the extraordinary step of publishing an op-ed in USA Today stating they would work with agency leadership “to maintain FDA’s steadfast commitment to ensuring our decisions will continue to be guided by the best science.”

“Protecting the FDA’s independence is essential if we are to do the best possible job of protecting public health and saving lives,” the officials wrote.

“Trust has eroded so significantly in these institutions that have undermined public confidence, especially on vaccines,” a Biden adviser said of the FDA and CDC. “Change in leadership is critical.”

Getting new people into the federal government — where Biden is charged with filling roughly 4,000 jobs held by political appointees — is a mammoth slog on its own, let alone while moving to take over the U.S. pandemic response. Former President Barack Obama set the record for presidential appointments in the first 100 days, securing Senate confirmation for 69 appointees. The FDA commissioner wasn’t among them — Dr. Margaret Hamburg was not nominated until March 2009 and became commissioner that May. A similar timeline held for Trump’s first FDA commissioner, Dr. Scott Gottlieb, who began in May 2017.

“It is a difficult period because you’re going to have a lot of folks who need to get into place,” said Max Stier, CEO of the Partnership for Public Service, which advises presidential candidates and their teams installing new administrations. “The track record has not been good on getting people in quickly.”

At the outset of the Biden administration, it’s expected there will be a fair number of “acting” agency heads rather than Senate-confirmed appointees, Stier said. Biden has said he’ll trust the government’s scientists on COVID vaccines. Former FDA officials said in interviews that if there’s an acting official in charge when a specific vaccine is under review, it should not make a difference because the agency’s longtime scientists conduct the necessary scientific evaluations.

Where it could make a difference is in messaging and accountability, not just to the new president but to the public: The traditionally lower profile and temporary nature of an acting FDA commissioner is at odds with the highly visible role the commissioner is expected to play during a public health emergency, particularly in convincing people that vaccines are safe.

“An agency needs a face, and it’s hard for an ‘acting’ to be the face of the agency,” a former senior agency official said. “The work could be done, but the communication is always better if there’s an FDA commissioner who’s willing to take responsibility.”

The messaging role has taken on extraordinary importance since public confidence in a coronavirus vaccine has eroded significantly. A September Pew Research Center poll found that only 51% of U.S. adults would definitely or probably get a vaccine to prevent COVID-19 if it were available, a drop of 21 percentage points since May.

“Things can only be better,” said Michael Carome, director of the health research group at Public Citizen, a left-leaning group that advocates for consumer interests. “I think an acting commissioner under a Biden administration will be far more trusted than the current FDA commissioner, who has been kowtowed by the White House.”

FDA staffing policy outlines who should be the agency’s acting head in the event there isn’t a permanent commissioner. The most recent version, from 2016, says the position is delegated to the deputy commissioner for foods and veterinary medicine, a title that has since been recast as deputy commissioner for food policy and response. The job is currently held by Frank Yiannas, a longtime food safety expert who joined the agency in 2018 after the retirement of Stephen Ostroff, a veteran FDA scientist who served as acting commissioner twice. The FDA did not respond to questions about whether it had a new staffing policy.

Some administrations, however, have ignored that policy. The Trump administration, for example, briefly installed senior HHS official Brett Giroir, a political appointee, as acting FDA commissioner, a move criticized by Democrats in Congress.

But critical decisions await the new appointee.

The earliest officials would know whether COVID vaccines from Johnson & Johnson and AstraZeneca work is January or February, said Moncef Slaoui, the top scientific adviser for Operation Warp Speed, which is funding multiple coronavirus vaccines and treatments. Other efficacy trials won’t be completed until spring, he said in October.

Safety will take even longer to assess — Johnson & Johnson’s and AstraZeneca’s late-stage clinical trials were already paused earlier this year for safety reasons —and companies will seek emergency authorization or FDA approval only once both metrics are known.

After four years of politicization of the science agency, a Biden adviser said, most important was having a “trusted, credible voice to restore trust in a vaccine.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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Bristol-Myers Squibb posted a solid set of financial results for the third quarter, but shares slid on investor fears that a payout tied new product approvals was in jeopardy.

BMS’ stock fell a little over 2%, but the big casualty was a contingent value right (CVR) due to former shareholders in Celgene, which BMS acquired for $74 billion a year ago. Celgene investors accepted a $9 CVR to sweeten the deal, tied to FDA approval of three late-stage medicines.

The CVR lost around three quarters of its value after BMS revealed on its third-quarter results call that the FDA still hasn’t carried out an inspection of a manufacturing facility that will be used to produce B-cell lymphoma CAR-T therapy lisocabtagene maraleucel (liso-cel), one of the three drugs.

Liso-cel has to be approved by the FDA before the end of the year to satisfy the requirements of the CVR. One manufacturing facility in Bothell, Washington has been inspected by the US regulator, but another operated by Lonza in Texas has not.

As both facilities need to be inspection before approval, that has led to speculation that the agency may not be able to complete its review of liso-cel by its 16 November deadline.

FDA inspectors “are doing what they can to ensure that…staff are kept safe in this COVID pandemic,” said Samit Hirawat, BMS’ chief medical officer. “And because of the travel restrictions, we have to obviously honour their desire as to where they go and when they go.”

Some analysts appealed for calm, noting that the FDA would likely have notified the company already if an inspection would not be happening in time and a delay to the review would be needed.

The first of the three CVR-tied medicines – Celgene’s multiple sclerosis drug Zeposia (ozanimod) – was approved in March. The final one – multiple myeloma CAR-T idecabtagene vicleucel (ide-cel) – has to be approved by 31 March next year, just four days after the FDA’s action date.

Liso-cel is already due for an FDA verdict three months later than anticipated after the regulator delayed its review by three months in May.

Worries about the liso-cel deadline drove the value of the CVR to penny share territory, down from almost $1.90 a year ago, and also cast a shadow over BMS’ third-quarter results.

BMS revenues rocketed 75% to $10.5 billion thanks to the addition of Celgene, but were up 6% on a prof forma basis. The numbers came in around $200 million ahead of expectations, which BMS put down to a good performance by anticoagulant blockbuster Eliquis (apixaban) which grew 9% to $2.1 billion.

Blood cancer therapy Revlimid (lenalidomide) also grew 10% to $3 billion, but cancer immunotherapy Opdivo (nivolumab) slipped 2% to $1.8 billion. BMS said it expects its flagship PD-1 inhibitor to return to growth next year.

The results came a couple of days after BMS presented impressive data with its psoriasis candidate deucravacitinib, which outperformed Amgen’s rival Otezla (apremilast) in a head-to-head trial.

CEO Giovanni Caforio said: “Our financial strength and flexibility combined with our robust inline businesses, multiple launches and progress in our deep pipeline, including the promising results from the deucravacitinib trial, strongly position the company to deliver our mission and help more patients.”

The post Could an FDA inspection scupper CVR deadline for BMS’ liso-cel? appeared first on .

What You Should Know:

– The FDA just extended its research collaboration
agreement (RCA) with COTA, specifically looking at how COVID-19 is affecting
cancer.

– With this expanded agreement, COTA and the FDA will use real-world data to explore the impact of COVID-19 and the pandemic on cancer treatment, with the opportunity to expand into other oncology questions in the future.

---

COTA, Inc., a healthcare technology
company that uses real-world data to bring clarity to cancer care, today
announced it has extended its Research Collaboration Agreement (RCA) with the
U.S. Food and Drug Administration (FDA) for an additional three years. This
renewed RCA will expand on the objective to explore the applications of
real-world data in oncology, including the impact of COVID-19 and the
pandemic on cancer treatment. As the project advances, the research will
broaden to study other oncology care delivery questions.

Real-world data can provide critical insights into the
delivery of cancer treatment in the routine practice setting, as well as
potential long-term effects post-COVID-19 recovery. The expanded focus of this
RCA will enable the exploration of important research questions to help support
FDA’s understanding of how the COVID-19 pandemic continues to impact patients
with cancer.

Why It Matters

With over 8 million cases of COVID-19 in the United States,
there is a significant need to understand the pandemic’s impact on oncology
care. Additionally, an increasing body
of research has shown that oncology patients may be particularly
susceptible to harm during this pandemic – both in contracting the
condition itself or experiencing care delays.

Cancer patients are particularly at risk of severe complications with COVID-19, and there is currently no understanding of how this can affect their oncology care or progression. Through real-world data, we can begin to understand if COVID-19 should be considered as comorbidity – particularly around clinical trial criteria.

Shots:

• The acceptance marks the first marketing application accepted for achondroplasia in the US with an anticipated PDUFA date as of Aug 20, 2021. The US FDA is not planning to hold an advisory committee meeting to discuss the application
• Additionally, the company is expecting to complete enrollment in a P-II study assessing vosoritide in ~70 infants and young children with achondroplasia, aged 0-<60mos., for 52wks.
• Vosoritide (qd) is an investigational injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia and has received the US FDA’s & EMA’s ODD for the same

Click here to­ read the full press release/ article | Ref: PRNewswire | Image: BioMarin Careers

The post Biomarin Reports the US FDA’s Acceptance of Vosoritide’s NDA to Treat Children with Achondroplasia first appeared on PharmaShots.

Regulatory reviews of Biogen’s Alzheimer’s drug aducanumab are now ongoing on both sides of the Atlantic, but debate is still ongoing about whether the data behind the drug is strong enough to support approval.

The EMA has just kicked off its review of the anti-amyloid therapy, following in the footsteps of the FDA in the US which has been looking at the drug since August, but a new analysis of the mixed phase 3 data for aducanumab argues that an additional trial should be carried out.

The paper in the journal Alzheimer’s & Dementia, led by Mayo Clinic neurologist David Knopman, says that efficacy of aducanumab “as a treatment for the cognitive dysfunction in Alzheimer’s disease cannot be proven by clinical trials with divergent outcomes.”

Meanwhile, the paper also notes that Knopman has been excluded from an FDA advisory committee meeting due to discuss the data on Friday, ahead of a decision on the marketing application due in March.

The expert – who was an investigator in the phase 3 trials of Biogen’s drug – told Reuters he was recused from the panel because of his involvement in conducting clinical trials of aducanumab.

Aducanumab – which Biogen is developing with Japanese drugmaker Eisai – was all but abandoned in 2019 after the partners decided that two phase 3 trials of the drug were unlikely to show an effect on cognitive decline in Alzheimer’s.

Shares in Biogen were hit hard, as investors lost hope that aducanumab might be rescue the almost defunct amyloid hypothesis of Alzheimer’s disease, which holds that blocking the formation of amyloid plaques in the brain could delay the onset of dementia.

Just a few months later however they said a fresh look at the results of the EMERGE and ENGAGE studies had revealed that the initial futility analysis was “incorrect.” In fact, the drug reduced clinical decline in patients with early, a chance was put down to more exposure to a higher dose in additional patient follow-up.

Some patients showed statistically significant improvements on symptoms like memory, orientation, and language, as well as being able to carry out day-to-day tasks more easily.

There’s a lot riding on the FDA and EMA reviews. If approved, aducanumab will become the first therapy to reduce the clinical decline of Alzheimer’s and to change the course of the disease, says Biogen. It would also be the first amyloid-targeting drug to reach the market, after dozens of others have failed in clinical development.

Meanwhile, aducanumab is the big hope in Biogen’s late-stage pipeline, which otherwise is looking fairly thin, at a time when the biotech is facing the loss of patent protection for its blockbuster multiple sclerosis therapy Tecfidera (dimethyl fumarate).

Knopman and fellow authors argue in the Alzheimer’s & Dementia paper that Biogen’s interpretation of data in the two trials might not be correct.

They write that they have found alternative explanations for the apparent drug benefits unrelated to the treatment, and say that while there is evidence that aducanumab was working on amyloid and other biomarkers like tau protein as expected, “no evidence was presented to correlate biomarker changes to cognitive benefits.”

They also say there were differences in the placebo responses between the two studies, which could have contributed to the divergent results.

“Our analysis supports the conduct of a third, definitive phase 3 trial with high‐dose aducanumab [that is] optimally designed and adequately powered to prove efficacy,” they conclude.

The FDA has not commented on the reasons for Knopman’s exclusion from the advisory committee meeting publicly, but in these cases there is usually a conflict of interest.

Along with his involvement in EMERGE and ENGAGE, Knopman also serves on a data safety monitoring board for a tau drug for Alzheimer’s developed by Biogen, and is an investigator in a trial sponsored by Eli Lilly and the University of Southern California.

He also performs unpaid consultancy work for Samus Therapeutics, Third Rock, Roche, and Alzeca Biosciences, according to the paper’s conflict of interest statement.

The post Trials and tribulations in Biogen’s Alzheimer’s drug reviews appeared first on .

What You Should Know:

– Fern Health will reveal a
first-of-its-kind collaboration with Mass General Hospital where it will inform
existing and future digitally-delivered pain management programs through the
marriage of AI + predictive analytics with 10 million de-identified Mass
General patient records.

– MGH will validate emerging Fern Health products and pilot new products in clinical environments, setting the stage for Fern expansion into all aspects of non-invasive multimodal pain management.

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Fern Health, a digital health company pioneering virtual musculoskeletal pain programs and pain neuroscience education through employers, announced that it has expanded its collaboration with Massachusetts General Hospital (MGH) and the MGH Center for Innovation in Digital HealthCare (CIDH). MGH is the original and largest teaching hospital of Harvard Medical School and home to one of the world’s leading pain management clinics.

Fern Health’s relationship with MGH, formed 18 months ago, will now broaden to entail a multi-year collaboration in which MGH will validate emerging Fern Health product lines, pilot new products in a clinical setting, and investigate new scientific approaches to pain management.

The expansion supports Fern Health’s long-term vision of democratizing access to non-invasive multimodal pain management. Fern Health’s current product suite, which includes an evidence-based, digitally delivered musculoskeletal (MSK) pain management program, was originally developed with experts from within Mass General, in consultation with their clinical collaborators at the world-renowned Spaulding Rehabilitation Network. Fern’s biopsychosocial pain management solution was validated with the clinical rigor of MGH’s renowned hospital-based research enterprise.

“There are a multitude of gaps in the U.S. healthcare system that unfortunately fail our patients with chronic pain, from lack of access to high-quality pain care to the proliferation of costly and often ineffective treatments,” said Mihir M. Kamdar, MD, MGH Pain Physician and Digital Health Advisor. “Evidence-based models of care are still rare in digital health solutions even though they have the potential to address these gaps and give clinicians innovative and effective care options for their patients.”

Leverage Data-Driven Insights from De-Identified Patient Data

Fern Health will leverage clinical validation and implementation science, clinical protocol development, access to data-driven insights derived from de-identified patient data, third-party corroboration for peer-review publications, and FDA approval processes. 

“By evaluating digital health tools in a real-world clinical setting, we can provide distinctive insights, understand user preferences, and validate clinical protocols for optimal implementation and outcomes,” added Joseph C. Kvedar, MD, Senior Advisor, Virtual Care, Mass General Brigham; Professor of Dermatology, Harvard Medical School; and Senior Advisor, MGH Center for Innovation in Digital HealthCare. “This collaboration is designed to help advance pain management through digitally-delivered personalized exercise therapy, education, and health coaching—which early results suggest is occurring.” Dr. Kvedar is also President of the American Telemedicine Association (ATA).

Expansion into All Aspects of Non-Invasive Multimodal Pain Management

The collaboration also gives Fern Health substantial clinical and scientific data to expand into the broader ecosystem of digitally-delivered pain management platforms: 

– The Fern user experience will replicate how a patient might experience evidence-based, personalized treatment at a hospital-based pain management clinic. Rather than delivered in-person, treatment is delivered digitally and is accessible from anywhere.

– Informed by predictive analytics and an expansive MGH data set of 10 million de-identified patient records, personalized, evidence-based Fern patient treatment plans will leapfrog the performance of “one-size-fits-all” pain management platforms that are limited to publicly available data or their own user data.

– The collaboration will form the foundation from which Fern will launch new products and services rooted in collaborative care aimed at treating the whole person across physical, emotional, and behavioral considerations.

Why It Matters

One out of every two people suffer from MSK pain and the U.S. spends $380 billion on MSK conditions each year, contributing to MSK pain being the top driver of employer healthcare costs. Fern Health eases the burden on employers who face daunting pain management treatment economics. Provided as a benefits add-on for self-insured employers, Fern Health offers a biopsychosocial approach to pain management, including personalized restorative therapy, pain neuroscience education and virtual 1:1 health coaching. The company is currently engaged in pilot programs with several mid-size and large employers spanning the professional services, manufacturing and transportation sectors.

“At least half of the population suffers from physical pain and its cascade of effects across social, mental and emotional well-being,” said Travis Bond, CEO, Fern Health. “This initiative marries science, clinical rigor, artificial intelligence and incredibly rich historical patient data sets with digital care delivery. It’s a huge first step into a better future for pain management science and for the millions of people living with musculoskeletal pain today.” 

Scrutiny of drug prices will continue whether Trump or Biden wins. But pharma can take steps to address critics and cut costs before they reach a crisis point, according to a panel of industry experts.

What You Should Know:

– Northwestern Memorial Hospital is the first in the
nation to deploy FDA-cleared AI-guided ultrasound by Caption Health, including
measurement of ejection fraction – the most widely used measurement to assess
cardiac function.

– Caption Health’s AI-guided cardiac ultrasound enables clinicians – including those without experience – to accurately perform diagnostic-quality exams — accelerating the availability of information and saving lives.

– Caption AI has been shown to produce assessments
similar to those of experienced sonographers in work presented to the American
Society of Anesthesiologists.

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Northwestern Memorial
Hospital is the first hospital in the United States to purchase Caption Health’s
artificial
intelligence (AI) technology for ultrasound, Caption AI. The FDA cleared, AI-guided
ultrasound system enables healthcare providers to acquire and interpret quality
ultrasound images of the human heart, increasing access to timely and accurate
cardiac assessments at the point of care.

Performing an ultrasound exam is a complex skill that takes years to master. Caption AI enables clinicians—including those without prior ultrasound experience—to quickly and accurately perform diagnostic-quality ultrasound exams by providing expert turn-by-turn guidance, automated quality assessment, and intelligent interpretation capabilities. The systems are currently in the hospital’s emergency department, medical intensive care unit, cardio-oncology clinic, and in use by the hospital medicine group.

Democratize the Echocardiogram

Point-of-care ultrasound (POCUS) has a number of benefits. Increased usage of POCUS contributes to more timely and accurate diagnoses, more accurate monitoring, and has been shown to lead to changes in patient management in 47% of cases for critically ill patients. POCUS also allows patients to avoid additional visits to receive imaging, as well as providing real-time results that can be recorded into a patient’s electronic medical record.

“Through our partnership with Caption Health, we are looking to democratize the echocardiogram, a stalwart tool in the diagnosis and treatment of heart disease,” said Patrick McCarthy, MD, chief of cardiac surgery and executive director of the Northwestern Medicine Bluhm Cardiovascular Institute, a group involved in the early development of the technology. “Our ultimate goal is to improve cardiovascular health wherever we need to, and Caption AI is increasing access throughout the hospital to quality diagnostic images.” 

How Caption Health Works

Caption AI emulates the expertise of a sonographer by providing real-time guidance on how to position and manipulate the transducer, or ultrasound wand, on a patient’s body. The software shows clinicians in real-time how close they are to acquiring a quality ultrasound image, and automatically records the image when it reaches the diagnostic-quality threshold. Caption AI also automatically calculates ejection fraction, or the percentage of blood leaving the heart when it contracts, which is the most widely used measurement to assess cardiac function.

“Northwestern Medicine has been a tremendous partner in helping us develop and validate Caption AI. We are thrilled that they are bringing Caption AI into key clinical settings as our first customer,” said Charles Cadieu, chief executive officer and co-founder of Caption Health. “The clinical, economic and operational advantages of using AI-guided ultrasound are clear. Most important, this solution increases access to a safe and effective diagnostic tool that can be life-saving for patients.”

Pfizer could be just a few months away from getting FDA approval for its JAK1 inhibitor abrocitinib in atopic dermatitis, a drug that CEO Albert Bourla believes hasn’t been given the credit it is due by Wall Street analysts.

The US regulator has started a priority review of abrocitinib for mediate to severe atopic dermatitis in patients aged over 12, setting up a decision next April, while the EMA has also started a standard review that could lead to approval in the EU in the second half of 2021.

Bourla told analysts on Pfizer’s results call yesterday that abrocitinib is “the one potential near-term compound where we see the biggest difference compared with consensus.”

Pfizer sees abrocitinib and other JAK drugs coming through the pipeline for atopic dermatitis as expanding the number of patients getting treatment for the more severe end of the spectrum of symptoms – and it reckons that unlocks blockbuster sales potential.

“This is not a zero-sum game with the biologics in the treatment of moderate to severe atopic dermatitis,” said Bourla.

These patients currently rely on biologic drugs like Sanofi and Regeneron’s Dupixent (dupilumab), which dominates the category and saw sales rocket to more than $2 billion last year.

Sanofi sees plenty of additional upside, suggesting the drug could become a $10 billion brand at peak from expansion in atopic dermatitis as well as new indications like asthma, chronic rhinosinusitis with nasal polyps and eosinophilic oesophagitis.

Abrocitinib has previously been shown as effective in the JADE-MONO-1 and JADE-MONO-2 trials in subjects aged over 12, with a profile that looks like it could challenge its biologic rivals.

In particular, Pfizer says the drugs seem to have an impact on itch – often cited as the most bothersome symptom by atopic dermatitis patients.

Bourla and Angela Hwang – group president of Pfizer’s biopharmaceuticals division – think analysts are overlooking the sheer scale of atopic dermatitis as a condition with around 60 million sufferers aged over 12 worldwide.

They are also discounting abrocitinib’s profile, and the fact that almost two-thirds of patients treated with Dupixent don’t achieve clear or almost clear skin at 16 weeks, leaving room for improvement.

“Of those 60 million, only 7% of them today are being treated with a systemic agent,” said Hwang on the call. “So the systemic market opportunity has real potential to more than double with the introduction of better systemic treatment, because the patient need is just so high.”

There’s a precedent for that level impact for new biologic drugs such as interleukin inhibitors for psoriasis, which doubled the market over a 10-year period.

Claiming just 8% of the systemic atopic dermatitis treatment market would equate to $3 billion in abrocitinib sales, said Hwang.

Pfizer’s drug seems to have a lead in atopic dermatitis over other JAK inhibitors, although AbbVie has trials on the go for its fast-growing Rinvoq (upadacitinib) rival in this indication – including a head-to-head comparison with Dupixent due to read out next year.

The post FDA sets April verdict for Pfizer’s “underestimated” atopic dermatitis drug appeared first on .

The Los Angeles-based startup received a breakthrough device designation from the FDA for using VR to manage chronic pain. It recently published the results of a 92-person virtual trial testing VR for pain management at home.

What You Should Know:

– Scopio Labs announced FDA clearance of its AI-powered
X100 microscope and decision support system with Full Field Peripheral Blood
Smear (Full Field PBS) application. 

– Using advanced computational photography imaging and tailored AI tools, Full Field PBS gives clinical laboratories an unprecedented ability to capture digital scans with full-field view of the monolayer and feathered edge at 100X oil immersion resolution level. 

– The global market for hematology analyzers and reagents
is currently $7.6 billion and is expected to reach $10.6 billion by 2025.

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 Scopio Labs, a leading provider of Full Field Morphology (FFM), announced that it was
granted FDA clearance to market and sell its X100 with Full Field Peripheral
Blood Smear (Full Field PBS) Application, unlocking the potential of in
vitro hematology diagnosis. Full Field PBS is also available in Europe with CE mark certification granted earlier this year.   

Why It
Matters

Blood is one
of the most foundational gateways to health information. Roughly 120,000
laboratories worldwide conduct 600 million PBS tests annually for the global
population, predominantly via manual microscopes. Even with the adoption of
digital tools, today’s solutions do not showcase all required regions of
interest in a PBS slide, only capturing snapshots of cells. Consequently,
technologists frequently default to the manual microscope for a more detailed
examination of the raw data. 

AI-Powered
Full Field Peripheral Blood Smear (Full Field PBS) Application 

To help improve diagnostic accuracy leveraging novel computer vision tools, Full Field PBS gives clinical laboratories an unprecedented ability to capture digital scans using advanced computational photography imaging and tailored AI tools. The Full Field PBS utilizes adaptive monolayer identification in support of long and short smears and automates the analysis process by pre-classifying 200 white blood cells (WBC), providing platelet pre-estimate, and enabling RBC morphology evaluation.  Accelerating routine analysis, Full Field PBS includes a tailored decision support system for pre-classification of white blood cells into 16 classes, red blood cell morphology evaluations, platelet location and count.

“Understanding the challenges lab technicians, hematologists and hematopathologists face when evaluating blood samples containing large numbers of morphologically-unique cells in a timely fashion, we designed our solution specifically for hematology labs where we can improve quality of care, consistency of results and reduce review time,” said Scopio Labs’ CEO and Co-Founder, Itai Hayut. “We are thrilled to receive FDA clearance following the successful completion of a multi-center study, as we bring our innovative solution to laboratories around the U.S. to help improve the outcome of diagnosis and care.”  

Recent Traction

Earlier this
year, the company closed a $16 million Series B funding round, bringing total funding to $30 million. Scopio Labs is setting its sights
on transforming all hematology applications, including bone marrow aspirates
(BMA) and body fluids. With a robust product development pipeline, and the
ability to detect morphological events on a cellular and subcellular scale,
Scopio Labs will open the door for morphology-based diagnostics, disease
monitoring and treatment adjustment for various blood cancers.  

What You Should Know:

– FDA awards AppliedVR Breakthrough Device designation for
treating treatment-resistant fibromyalgia and chronic intractable lower back
pain

– AppliedVR’s EaseVRx program helps patients learn self-management skills grounded in evidence-based cognitive-behavioral therapy (CBT) principles and other behavioral methods.

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AppliedVR,
a pioneer advancing the next generation of digital medicine, today announced
its EaseVRx product received Breakthrough Device designation from the U.S. Food
and Drug Administration (FDA) for treating treatment-resistant fibromyalgia and
chronic intractable lower back pain. EaseVRx is now one of the first virtual
reality (VR) digital therapeutics to get breakthrough designation to treat
conditions related to chronic pain.

What is the FDA Breakthrough Device Program?

The FDA Breakthrough Device Program helps patients receive more timely access to breakthrough technologies that could provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases or conditions. 

Clinical Trial Results/Outcomes

AppliedVR achieved this milestone after successfully
completing the first randomized controlled trial (RCT), evaluating VR-based
therapy for self-management of chronic pain at home. The RCT, which was
published in JMIR-FR,
found that a self-administered, skills-based VR treatment program for treating
chronic pain was feasible, scalable and was effective at improving on multiple
chronic pain outcomes – each of which met or exceeded the 30-percent threshold
to be clinically meaningful. On average, participants noted:

– Pain intensity reduced 30 percent;

– Pain-related activity interference reduced 37 percent;

– Pain-related mood interference reduced 50 percent;

– Pain-related sleep interference reduced 40 percent; and

– Pain-related stress interference reduced 49 percent.

EaseVRX Program Background

AppliedVR’s EaseVRx program helps patients learn self-management skills grounded in evidence-based cognitive-behavioral therapy (CBT) principles and other behavioral methods. The program was designed by AppliedVR, in partnership with the top pain experts and researchers, to improve self-regulation of cognitive, emotional, and physiological responses to stress and pain. AppliedVR has already been shown to be an effective treatment for acute pain in hospital settings. 

Why Virtual Reality Is An Effective Approach for Pain
Management

Lower back pain is one of the most common
chronic conditions that people face worldwide and represents one of the top
reasons why people miss work. Additionally, it’s an extremely
costly problem for insurers, especially as they look to cut costs related to back surgery. Recent research indicated that, when combined with neck pain,
lower back pain costs nearly $77 billion to private insurance, $45 billion to
public insurance, and $12 billion in out-of-pocket costs for patients.

Chronic pain more broadly also is a difficult and costly
problem that has contributed to many other major health problems in the U.S.,
including the opioid epidemic. A previous Johns Hopkins study in the Journal of
Pain found that chronic pain can cumulatively cost as high as $635 billion a year — more than the annual costs of
cancer, heart disease and diabetes — and lower back pain has been one of the most common reasons for prescribing opioids.
Cognitive behavioral therapies like VR are now seen by many providers as an
effective alternative or complement to pharmacological interventions that can
support their larger treatment tool belts.

“Since 1980, the American Chronic Pain Association has advocated a multidisciplinary approach to pain management—using a combination of medical and behavioral techniques to address pain,” said Penny Cowan, founder and CEO of the American Chronic Pain Association. “Virtual reality has the potential to be an important resource in this approach, helping people with pain to think differently about their conditions and learn strategies to reduce suffering and improve quality of life.”

Future Clinical Trials

AppliedVR is currently engaged in many other trials,
including feasibility studies with multiple well-known payers and with the
University of California at San Francisco (UCSF) to study how digital therapeutic platforms, including
virtual and augmented reality, can be used to improve care access for
underserved populations. AppliedVR also is advancing two clinical trials with
Geisinger and Cleveland Clinic to study VR as an opioid-sparing tool for acute
and chronic pain – specifically the company’s RelieVRx and EaseVRx platforms.
The National Institute on Drug Abuse (NIDA), part of the National Institutes of
Health (NIH), recently awarded $2.9 million grants to fund the trials.

While there are encouraging signs of reimbursement falling in step with the move towards a more value-based healthcare system, what is needed now to further encourage healthcare innovators is to properly rationalize approval processes imposed by the FDA and CMS.

Zosano Pharma has been hit by an FDA rejection of its marketing application for migraine drug Qtrypta, asking for new bioequivalence data.

Qtrypta takes the form of a transdermal patch loaded with zolmitriptan, a well-established medicine for acute migraine that has been available for years in various formulations including tablets and nasal sprays.

Zosano’s product uses microneedles to help the drug get absorbed into the blood, but the Complete Response Letter (CRL) from the FDA says it is concerned about inconsistencies in how much zolmitriptan was being delivered with Qtrypta.

Specifically, the FDA pointed to differences in zolmitriptan exposures between subjects receiving different lots of Qtrypta in the company’s trials, including some unexpectedly high results in five individuals.

It also mentions “inadequate pharmacokinetic bridging between the lots that made interpretation of some safety data unclear.”

The CRL comes just a couple of weeks after Zosano received a discipline review letter (DRL) from the FDA, a preliminary sharing of the agency’s stance, after which the company had already said it did not expect approval by the action date of 20 October.

Shares in the Nasdaq-listed biopharma lost around 25% of their value after the announcement, as investors tried to gauge how long the programme might now be delayed, and the implications for its delivery platform, which is the first microneedle patch to be Included in a new drug application to the FDA.

Qtrypta is Zosano’s lead product candidate, but the company is also working on a follow-up in phase 2/3 for cluster headache.

The FDA wants a new bioequivalence study between three of the Qtrypta lots tested during development, as well as product quality validation data, originally due to be filed after approval, as part of the new drug application (NDA).

The agency also says that inspection of Zosano’s contact manufacturing facilities for the product – while currently on hold because of the pandemic – will also have to be completed before Qtrypta can be approved.

That suggests the delay could be some time, but Zosano is keeping tight-lipped until it has a chance to meet with the FDA to discuss a way forward for the programme.

“We are working diligently to address the deficiencies identified by the FDA and look forward to the possibility of resubmitting our NDA,” said the company’s CEO Steven Lo.

“There are thousands of people suffering from migraine attacks that are not adequately addressed with available drugs, and we continue to believe that Qtrypta, if approved, could offer a much-needed new therapy for these patients,” he added.

The company has previously reported data showing that Qtrypta can deliver fast relief from a migraine with fewer than 2% of patients experiencing side effects common with triptan drugs, like dizziness and pins and needles.

Zosano recently signed a five-year deal worth $250 million deal with Eversana to commercialise and distribute Qtrypta if gets past the FDA.

The post Headache for Zosano as FDA turns down migraine patch appeared first on .

Pfizer said it will wait until after the US election to file its COVID-19 vaccine with the US regulator, as it waits for important safety data to become available.

The vaccine is being developed by Pfizer and development partner BioNTech and will wait until late November to make its application with the FDA.

In a letter published on its website Pfizer may have the data to say whether the vaccine is effective this month, based on the findings of the ongoing 40,000 person clinical trial.

But CEO Alfred Bourla cautioned that the safety data will only be ready in the third week of November.

There have already been two safety scares in COVID-19 vaccine trials – Johnson & Johnson earlier this week put its phase 3 trial on hold because of an undisclosed illness in a patient.

A US trial of AstraZeneca’s shot is also on hold after a similar incident, although studies in other parts of the world have restarted.

As vaccines will be given to healthy individuals, regulators have far less tolerance for adverse events.

There are further concerns that political interference during the election build-up could undermine the credibility of a mass vaccination programme.

Bourla said: “So let me be clear, assuming positive data, Pfizer will apply for Emergency Authorization Use in the US. soon after the safety milestone is achieved in the third week of November.”

Despite the concerns of scientists president Donald Trump has said that there would be a vaccine available before the election on 3rd November and is hoping that approval could be the “October Surprise” that could boost his popularity ahead of the vote.

Rival Moderna has said it could apply for an Emergency Use Authorization for its rival vaccine as soon as November.

The European Medicines Agency has already started a rolling review of the Pfizer/BioNTech and AstraZeneca vaccines.

The post Pfizer to wait until after election to file COVID-19 vaccine in US appeared first on .

What You Should Know:

– FUJIFILM Medical Systems U.S.A., Inc. and Volpara
Solutions announced the extension of their partnership to provide mammography
facilities and clinicians with breast imaging solutions designed to improve
image quality, streamline workflow and accurately assess a patient’s breast
density.

– Building on a successful 6-year partnership, Fujifilm’s
customers using ASPIRE Cristalle with Digital Breast Tomosynthesis (DBT) now
have access to the latest innovations from Volpara’s Breast Health Platform.

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FUJIFILM
Medical Systems U.S.A., Inc., a provider of diagnostic imaging
and medical informatics solutions, and Volpara Solutions, a
purpose-driven software company on a mission to prevent advanced-stage breast
cancer, today announced an expanded partnership to provide mammography
facilities and clinicians with breast imaging solutions designed to improve
image quality, streamline workflow and accurately assess a patient’s breast
density.

Building on a successful 6-year partnership, Fujifilm’s
customers using ASPIRE
Cristalle with Digital Breast Tomosynthesis (DBT) will now have access
to the latest innovations from Volpara’s Breast Health Platform. Volpara®Live!
helps reduce patient recalls due to poor image quality by giving mammographers
instant feedback on positioning and compression—which the FDA attributes as the
cause of most image deficiencies—for adjustment before the patient leaves the
room. Volpara Enterprise provides a comprehensive analysis of quality on
every mammogram and tomosynthesis image taken at the facility to identify
opportunities for improvement.

Early Detection is Critical to Breast Cancer Survival

Dense breast tissue is associated with an increased risk of developing breast cancer. Volpara’s  Enterprise includes a module that uses proprietary x-ray physics, AI, and machine learning to generate an accurate volumetric measure of breast composition. It provides a repeatable, consistent, and objective means of scoring breast density.

“Early detection is critical to breast cancer survival.  It’s essential that clinicians and patients have as many resources available to them to quickly and accurately find any possible signs of disease,” said Christine Murray, Women’s Health Product Manager, FUJIFILM Medical Systems U.S.A., Inc. “Fujifilm is thrilled to expand our relationship with Volpara Solutions to offer our customers the clinical decision-support tools they need to improve mammography quality and enhance patient care.”  

Scynexis has filed for FDA approval of its novel drug ibrexafungerp which – if approved – would be the first broad-spectrum antifungal to reach the US market for vaginal yeast infections in more than two decades.

The Jersey City, US-based biotech has submitted ibrexafungerp (SCY-078) on schedule as a treatment for the vulvovaginal candidiasis (VVC), a common yeast infection affecting one in three women during their lifetime.

The FDA has already granted Qualified Infectious Disease Product (QIDP) as well as fast-track status to the drug, setting up a possible approval in June 2021 and a launch next summer.

Currently there is only one approved drug for VVC – oral fluconazole – but a sizeable proportion of patients don’t respond well to the treatment. Resistance rates are rising and the drug is unable to be used in pregnant women as they are at a higher risk of VVC infections.

Ibrexafungerp is a member of the glucan synthase inhibitor class – which covers fluconazole as well as newer agents like caspofungin and micafungin that are administered intravenously – but has a new triterpenoid structure.

That makes it suitable for both IV and oral dosing, and also makes it fungicidal, in other words it kills fungi cells unlike some current drugs which only block fungal growth.

The filing in VVC is based on the results of two phase 3, placebo-controlled studies, VANISH-303 and VANISH-306, which had clinical cure rates of 50.5% and 63.3 and mycological cure rates of 49.5% and 58.5%, respectively.

That level of efficacy improves on results with oral fluconazole, according to analysts at Ladenburg Thalman, who think there is a potentially big market for ibrexafungerp, particularly among the approximately 650,000 women in the US who suffer recurrent VVC infections.

They think a lot of the drug’s use will come second-line behind fluconazole, but also point to market research by Scynexis that suggests 29% of physicians would use the drug as a first-line option, increasing to 59% for fourth-line treatment of recurrent VVC.

Assuming the drug is approved in the US next year and costs around $300 to $400, analysts believe sales could reach almost $240 million by 2025 then rise to $400 to $600 million.

Aside from targeting VVC, Scynexis is running two phase 3 trials of ibrexafungerp in refractory invasive fungal infections caused by Candida auris, called FURI and CARES, as well as the CANDLE study in recurrent VVC and SCYNERGIA, a mid-stage study of ibrexafungerp plus voriconazole in patients with invasive pulmonary aspergillosis.

“We believe that ibrexafungerp may be the modern antifungal therapy, that will have utility for a broad range of today’s fungal infection patients as fluconazole did almost 30 years ago,” said Scynexis’ chief executive Marco Taglietti.

The post Scynexis bids to end novel antifungal drought, filing ibrexafungerp in US appeared first on .

What You Should Know:

– Nationwide disease registry Corrona has acquired
virtual patient community HealthUnlocked, creating a first-in-class patient
experience ecosystem.

– The acquisition will enable Corrona to expand its broad
set of capabilities–ranging from highly granular and longitudinal structured
data across eight registries.

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Corrona, LLC 
(“Corrona”), a nationwide disease registry and is now the largest
registry in the world collecting data on Rheumatoid Arthritis (RA), today
announced it has acquired
London-based HealthUnlocked, the
world’s largest virtual patient community.  

Acquisition Will Establish Innovative Snapshot of Patient

Founded in 2010, HealthUnlocked leverages an innovative
social platform to connect 1.3 million patients, representing over 300 disease
states and conditions with more than 500 patient advocacy groups. Corrona’s
investment in HealthUnlocked helps to further expand the patient engagement
offerings that began in late 2019 with Corrona’s acquisition of HealthiVibe, a recognized leader in
patient experience and engagement. HealthUnlocked provides an additional
dimension of cultivated data by leveraging a social network of 1.3 million
patients across hundreds of condition-specific communities, moderated by over
500 patient advocacy groups, capturing insights to better understand what
matters most to these patients. HealthUnlocked will be integrated into
Corrona’s HealthiVibe business unit to establish an innovative and holistic
snapshot of the patient. 

By building out its patient experience business, Corrona is working to create an industry-leading ecosystem centered around the patient voice. This ecosystem directly supports both the 21st Century Cures Act and the FDA’s Patient-Focused Drug Development initiative, and establishes Corrona as a leader in patient insights and real-world evidence with both a scalable technology platform and short- and long-term outcomes and safety data. This data powerhouse is the first of its kind to provide such a wholistic, 360⁰ degree view of a patient while ensuring data integrity. 

“By combining with HealthUnlocked, we are expanding our broad set of capabilities–ranging from highly granular and longitudinal structured data across our eight registries, to broader patient insights from HealthUnlocked,” said Abbe Steel, Chief Patient Officer of Corrona. She continued, “The HealthUnlocked communities provide access to engaged patients across the globe, allowing us to better understand the patient experience and what matters most to patients. Our organization is positioned to expand its expertise in gathering, analyzing, and applying deep patient insights to optimize the patient journey and bring significant value to our clients.”

Harmony Biosciences has scored an expanded use for its Wakix narcolepsy drug to include cataplexy – a symptom of the sleep disorder that can cause total body collapse.

Wakix (pitolisant) was first approved in August 2019 for treatment of excessive sleepiness in adults with narcolepsy.

It is the only FDA-approved drug to treat cataplexy associated with narcolepsy not scheduled as a controlled substance by the US Drug Enforcement Administration.

Cataplexy is a temporary loss of muscle tone and often triggered by strong emotions such as excitement or laughter. Effects can be severe and cause knee buckling or the body to collapse.

Last year, the FDA rejected Wakix for the additional use.

After receiving the Complete Response Letter rejecting Wakix for cataplexy, Harmony met with the FDA who agreed to review a new analysis of the HARMONY1 trial.

Following review of the data, the agency acknowledged it showed a statistically significant reduction in the rate of cataplexy for Wakix compared with placebo.

The new indication adds to Wakix’ use in a market dominated by older drugs such as modafinil for improving wakefulness, and others treating cataplexy attacks such as sodium oxybate and venlafaxine.

Last year, Jazz Pharmaceuticals’ Sunosi (solriamfetol) was approved to improve wakefulness in adults with narcolepsy or obstructive sleep apnoea.

The company added another alternative, Xywav (calcium, magnesium, potassium, and sodium oxybates) earlier this year although these drugs lack the cataplexy use on their label.

The post Harmony’s sleep disorder drug Wakix gets US label extension appeared first on .

Shots:

• Eli Lilly reports additional data on its SARS-CoV-2 neutralizing Ab programs including interim data on combination therapy in diagnosed patients with mild-to-mod. COVID-19 and plans to make therapies available to patients
• The new analysis P-II BLAZE-1 study assessing LY-CoV555 (2800mg) + LY-CoV016 (2800mg) vs PBO demonstrated reduced viral [email protected] meeting its 1EPs, reduction in symptoms and COVID-related hospitalization and ER visits
• Based on the combination regimen data, along with the previous findings for LY-CoV555, Lilly has submitted the initial EUA for LY-CoV555 monothx. and plans to initiate a large open-label pragmatic study in COVID-19 outpatients in Oct’2020. Additionally, Lilly anticipates the data supporting BLA submission for dual regimen as early as Q2’21

Click here ­to­ read full press release/ article | Ref: Eli Lilly | Image: GMP News

The post Eli Lilly Seeks the US FDA’s EUA for its COVID-19 Antibody Treatment first appeared on PharmaShots.

The FDA granted an emergency use authorization to an algorithm developed by Dascena to predict which Covid-19 patients might face unstable blood pressure or respiratory decline.

What You Should Know:

– Virta is expanding its suite of treatment options to
include prediabetes reversal, obesity reversal, and type 2 diabetes management.

– By making this crucial expansion, Virta can scale its
treatment to support the tens of millions of additional patients with
prediabetes and obesity, as provide an on-ramp to reversal for those with T2D
that aren’t yet ready to reverse. 

---

Virta Health, the
leader in type 2 diabetes reversal, today introduced the addition of new
services including prediabetes reversal, obesity treatment, and provider-led
management for type 2 diabetes. The expansion provides payers and covered
beneficiaries a single, full-service virtual clinic that offers
industry-leading outcomes for the most critical needs in metabolic health.

Virta’s fully-virtual, high-touch model demonstrates hope
for change, and stands in stark contrast to approaches that only slow the
diabetes downward spiral, as opposed to reversing it. Virta provides
individualized guidance from medical providers and behavioral specialists,
whenever and wherever it is needed. Patients interact with their dedicated
clinical team often multiple times per day. This novel telehealth
approach—called Continuous Remote Care—ensures successful adoption of Virta’s
individualized medical nutrition therapies and long-lasting results.

---

Why It Matters

Nearly half of adults in the United States suffer from obesity,
prediabetes, or type 2 diabetes. Thirty people die per hour of diabetes-related causes. The
economic burden continues to grow, and people with diabetes incur nearly $17,000
in medical expenses per year. They are also at high risk for severe illness
from COVID-19, and risk of dying from the disease is twice as high compared to
those without diabetes.

Obesity and prediabetes patients will benefit from the same
treatment that delivers the sustained type 2 diabetes reversal outcomes in Virta’s
clinical trial and commercially-covered population. Patients receiving type 2
diabetes management will receive support from a provider-led care team, with
personalized guidance and an option for a seamless transition to Virta’s
reversal treatment. All patients will receive individualized care via Virta’s
provider-led Continuous Remote Care platform.

Virta Type 2 Diabetes Reversal Results

Virta’s results in type 2 diabetes reversal have fueled continued triple-digit year-over-year growth for the company while creating strong demand to bring Virta’s evidence-based approach to other metabolic conditions. In Virta’s peer-reviewed clinical trial results, 60% of people at one-year reverse type 2 diabetes, and 94% reduce insulin use or eliminate it altogether.

Additionally, patients completing one year of the Virta
Treatment experience 14% weight loss. This figure exceeds the goal of the
National Diabetes Prevention Program and the FDA benchmark for weight loss
drugs by nearly 200%.

“This expansion provides our commercial partners and patients with the transformational outcomes they’ve come to expect from Virta, but don’t receive from other solutions on the market,” said Sami Inkinen, CEO & co-founder of Virta Health. “We can now meet every patient wherever they are on their metabolic health journey, while uniquely offering a path to reversing their chronic disease.”

The guidance calls for a median two months’ worth of follow-up safety data from well-designed Phase III studies as a precondition for a vaccine receiving an EUA. The Wall Street Journal reported that the White House had backed down in its opposition to the guidance, which pushes the date of an EUA past Election Day.

As trust in the Food and Drug Administration wavers, several states have vowed to conduct independent reviews of any COVID-19 vaccine the federal agency authorizes.

But top health experts say such vetting may be misguided, even if it reflects a well-founded lack of confidence in the Trump administration — especially now that the FDA has held firm with rules that make a risky preelection vaccine release highly unlikely.

At least six states and the District of Columbia have indicated they intend to review the scientific data for any vaccine approved to fight COVID-19, with some citing concern over political interference by President Donald Trump and his appointees. Officials in New York and California said they are convening expert panels expressly for that purpose.

“Frankly, I’m not going to trust the federal government’s opinion and I wouldn’t recommend [vaccines] to New Yorkers based on the federal government’s opinion,” New York Gov. Andrew Cuomo said last month.

“We want to make sure — despite the urge and interest in having a useful vaccine — that we do it with the utmost safety of Californians in mind,” Dr. Mark Ghaly, California’s health and human services secretary, said at a recent news conference.

The District of Columbia, Colorado, Michigan, Oregon and West Virginia also have said they’ll review vaccine data independently.

But scientists who study vaccine policy said such plans could backfire, confusing the public, eroding confidence in any eventual vaccine and undermining the best strategy to end the pandemic, which has sickened nearly 7.5 million Americans and killed more than 210,000.

“Do you really want a situation where Texas, Alabama and Arkansas are making drastically different vaccine policies than New York, California and Massachusetts?” asked Dr. Saad Omer, an epidemiologist who leads the Yale Institute for Global Health.

Separate state vaccine reviews would be unprecedented and disruptive, and a robust regulatory process already exists, said Michael Osterholm, an epidemiologist and director of the Center for Infectious Disease Research and Policy at the University of Minnesota.

“States should stay out of the vaccine review business,” Osterholm said. “I think the Food and Drug Administration is doing their job right now. Unless there’s something that changes that, I do believe that they will be able to go ahead.”

The administration has given reasons for states to worry. Trump has repeatedly signaled a desire for approval of a vaccine by the Nov. 3 election, arousing fears that he will steamroll the normal regulatory process.

The president wields “considerable power” over the FDA because it’s part of the executive branch of government, said Lawrence Gostin, faculty director of the O’Neill Institute for National and Global Health Law. The president nominates the FDA commissioner and can replace that official at any time.

Trump has already contradicted the advice of his own scientific advisers in order to promote unproven therapies to fight COVID-19. The FDA approved two treatments — hydroxychloroquine and convalescent plasma — without strong evidence of safety and efficacy after Trump pushed for the therapies to be widely available.

Late Monday, The New York Times reported that top White House officials planned to block FDA guidelines that would bolster requirements for emergency authorization of a COVID vaccine — because the new guidelines would almost certainly delay approval until after the election.

The White House’s actions undermine the agency, said Dr. Paul Offit, an infectious disease expert at Children’s Hospital of Philadelphia and a member of the FDA advisory committee on vaccines.

“Trump has perverted the FDA,” Offit said. “He has scared people into thinking that normal systems aren’t in place there anymore.”

But the FDA seems to be maintaining plans that would make it virtually impossible for a vaccine to be approved by Election Day.

Dr. Peter Marks, who heads the FDA division responsible for vaccine approval, has repeatedly said career scientists at the agency are working to ensure that political pressure isn’t a factor in any decision.

FDA reviewers are determined to “keep our hands over our ears to the noise that’s coming in from all sides and keep our eyes on the prize,” Marks said Monday in a JAMA webinar.

On Tuesday, the FDA pushed back against White House interference by publishing stricter guidance for vaccine developers on its website. The document instructs vaccine companies to follow patients for two months after their last shot in order to give researchers more time to detect serious side effects and ensure the vaccine works.

For now, supporters of the normal regulatory process are pinning their hopes on two advisory groups of respected scientists who will evaluate vaccines for safety and efficacy and send their recommendations to federal agencies.

The FDA’s advisory group, known as VRBPAC, will review data submitted by the pharmaceutical companies and the agency for any vaccine. The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices, or ACIP, will weigh in on its use. Their recommendations aren’t binding, but the federal government has rarely contravened them.

Before jumping to independent reviews, states should allow ACIP and VRBPAC to do their jobs, said Dr. Marcus Plescia, chief medical officer of the Association of State and Territorial Health Officials. That’s the best defense against any political pressure, he said, and individual states likely wouldn’t have access to the data — or, perhaps, the expertise — to conduct their own reviews.

ACIP Chairman Dr. José Romero, who also is the chief medical officer for the Arkansas Department of Health, said the group has been meeting regularly since spring to discuss COVID vaccines and they’ve been able to proceed “in an unfettered fashion.”

“I have not felt pressured by the CDC, other government agencies or pharmaceutical companies to arrive at any particular recommendation,” he said.

Other safeguards are in place as well. Trump cannot simply override the FDA’s authority to approve drugs and vaccines, which comes from Congress.

“The president can influence the FDA, but it must be consistent with the FDA’s statutory mandate,” Gostin said. “The White House may not, for example, direct the agency to ignore science or use a lower scientific standard.”

Congress could sue the FDA for failing to follow its own standards, and a judge could issue a temporary restraining order blocking release of a COVID vaccine, Gostin said. Courts would require the FDA commissioner or health and human services secretary to have “valid, evidence-based reasons” for any decision.

“The commissioner or secretary may not act arbitrarily or according to political preferences alone,” Gostin said.

Individual states could not overrule the FDA’s authorization or approval of a vaccine, but they could wield their power in other ways. States distribute vaccines through contracts with the CDC, noted Dr. Kelly Moore, associate director of immunization education for the Immunization Action Coalition. They could say, “‘We will not place any orders until we’re sure,’” she said.

States probably could not prevent private companies, such as pharmacy chains, from distributing vaccines that are shipped directly to them. Pharmacies would likely sue any states that try to prevent them from distributing vaccines, Gostin said.

Although federal and state agencies play a crucial role in ensuring patient safety, they’re not the only entities looking out for patient interests, said Dr. Joshua Sharfstein, a former FDA deputy commissioner who is now a vice dean at the Johns Hopkins Bloomberg School of Public Health. Doctors and other medical providers won’t recommend a vaccine they don’t trust, he said.

“We have an entire health care system standing between politics and the patients,” Sharfstein said. “I think doctors are going to be very concerned if a vaccine is rushed.”

Even pharmaceutical companies that stand to profit from vaccines have a huge stake in protecting the integrity of the approval process. Nine rival vaccine makers took the unusual step last month of pledging not to release a COVID vaccine until it has been thoroughly tested for safety.

The bigger consideration, however, is how state-by-state vetting would affect consumer trust in a COVID vaccine — or any vaccine in the future, Plescia said. A recent KFF poll found 54% of Americans would not submit to a COVID vaccine authorized before Election Day.

“Are people going to mistrust the entire process?” he said. “We will get through COVID one way or another, but if we undermine confidence in public health, that would be a disaster.”

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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The FDA may have been blocked from issuing stricter guidelines for emergency approval of COVID-19 vaccines by the Trump administration, but it has still managed to make its new approach public.

The regulator published “advice” to vaccine developers as part of documentation prepared for a vaccine advisory panel scheduled to convene later this month.

The five-page document sets out the information the FDA wants to see on safety and effectiveness and chemistry manufacturing and controls (CMC), and represents a tightening of the criteria for an emergency-use authorisation (EUA).

For example, the agency wants to see median follow-up duration of at least two months after completion of the full vaccination regimen in phase 3 studies – which would make it almost impossible for a shot to get the green light before the US election given that the front-runner jabs require two doses given at least 3-4 weeks apart.

The Trump Administration blocked the FDA from introducing new guidelines because they would set back the timeline for a COVID-19 green light by several weeks, scuppering the President’s plans to get a shot available before Americans go to the polls on 3 November.

Trump has also accused the FDA of succumbing to political pressure to delay approval of a vaccine, which the agency denies, and says industry had objected to the tougher standards. That prompted this response from former FDA Commissioner Scott Gottlieb:

Consumer group Public Citizen meanwhile said today that the suppression of the FDA guidelines “represents the latest but most dangerous politicisation of the US public health response to the catastrophic pandemic.”

There has been speculation for some weeks that the Trump administration could try to authorise a vaccine without oversight from the FDA, and the agency’s decision to set out its criteria publicly suggests it is resisting any such move.

Trump has previously said that a vaccine may be available this month, and the administration has pledged to provide tens of millions of doses before the end of the year.

Michael Carome, director of Public Citizen’s Health Research Group, said that the interference from the White House is an “appalling action that will only feed public distrust in any future COVID-19 vaccine marketed under an EUA, fostering vaccine hesitancy and prolonging the pandemic.”

Industry is also concerned. Just last week, the BIO trade organisation sent an open letter to Health & Human Services Secretary Alex Azar asking that all new guidance developed by the FDA concerning EUA for vaccines to prevent the spread of COVID-19 be released.

“The…guidance would provide scientists and researchers greater regulatory clarity and strengthen public confidence in any future vaccine that may be authorised or approved,” said the letter, signed by BIO chief executive Michelle McMurry-Heath.

The current FDA Commissioner Stephen Hahn has previously insisted that the agency will not be swayed by political pressure during presidential election campaigning.

The FDA guidelines reportedly also envisage that an independent advisory committee should be convened to go over the data before it can consider EUA. With the election now less than a month away, the timeline for setting up and running an advisory committee would also make it highly unlikely that a vaccine could be available before then.

Among the leading vaccines, Pfizer/BioNTech’s RNA-based candidate looks like it is the only one with a chance of seeking an EUA ahead of the election. Moderna has said it doesn’t expect results with its RNA shot until later that month, while AstraZeneca’s adenoviral-based vaccine is still on clinical hold in the US.

The latest twist in the coronavirus vaccine story comes during another politically fractious week in the coronavirus fight, with Trump now back at the White House after contracting COVID-19.

He immediately courted controversy by downplaying the seriousness of the virus, removing his face mask while posing for pictures despite still being infectious, and claiming that vaccines are “coming momentarily.”

Meanwhile, Facebook has deleted a post in which Trump had claimed COVID-19 was “less lethal” than the flu, placing it behind a warning about “spreading misleading and potentially harmful information.”

The post FDA gives COVID-19 vaccine ‘advice’ after White House blocks guidance appeared first on .

As the coronavirus pandemic broke out across the country, health care providers and scientists relied on the standard method for detecting respiratory viruses: sticking a long swab deep into the nose to get a sample. The obstacles to implementing such testing on a mass scale quickly became clear.

Among them: Many people were wary of the unpleasant procedure, called a nasopharyngeal swab. It can be performed only by trained health workers, putting them at risk of infection and adding costs. And the swabs and chemicals needed to test for the virus almost immediately were in short supply.

Some places, like Los Angeles County, moved early to self-collected oral swabs of saliva and sputum, with the process supervised at drive-thru testing sites by trained personnel swathed in protective gear. Meanwhile, researchers began investigating other cheaper, simpler alternatives to the tried-and-true approach — including dribbling saliva into a test tube.

But the transition has not been immediate. Regulators and scientists are generally cautious about new, unproven technologies and have an understandable bias toward well-established protocols.

“Saliva is not a traditional diagnostic fluid,” said Yale microbiologist Anne Wyllie, part of a team whose saliva-based test, called SalivaDirect, received emergency use authorization from the Food and Drug Administration in August. “When we were hit by a virus that came out of nowhere, we had to respond with the tools that were available.”

Eight months into the pandemic, the move toward saliva screening is gaining traction, with tens of thousands of people across the country undergoing such testing daily. However, saliva tests still represented only a small percentage of the more than 900,000 tests conducted daily on average at the end of September.

Yale is providing its protocol on an open-source basis and recently designated laboratories in Minnesota, Florida and New York as capable of performing the test. Besides the Yale test, the FDA has authorized emergency use of several others, including versions developed at Rutgers University, the University of Illinois at Urbana-Champaign, the University of South Carolina and SUNY Upstate Medical University. A further advance, an at-home saliva test, could be headed for FDA authorization, too.

Since the start of the pandemic, the Trump administration’s approach to testing has been hampered by missteps and controversy. As a key health agency during an unprecedented emergency, the FDA’s effectiveness relies on public trust in how it balances the need for speed in authorizing innovative products, like saliva tests and vaccines, with ensuring safety and effectiveness, said Ann Keller, an associate professor of health policy at the University of California-Berkeley.

“You obviously want to get new tests into the mix quickly in order to address the emergency, but you still need to uphold your standards,” Keller said. The White House’s public pressure on the FDA has complicated the agency’s efforts by undermining its credibility and independence, she said.

Respiratory viruses colonize areas inside the nasal cavity and at the back of the throat. Besides the nasopharyngeal approach, nasal samples obtained with shorter and less invasive swabs have proven effective for the coronavirus and have become widely adopted, although they also generally require a health care worker’s involvement. The millions of rapid tests that will be distributed across the country, per a recent White House announcement, rely on nasal swabs.

In the early months of the pandemic, some studies reported significant levels of the virus in oral secretions. In a Hong Kong study published in February, for example, the virus was found in the saliva of 11 of 12 patients with confirmed coronavirus infection.

In Los Angeles, which began using the oral swab test in late March, more than 10,000 samples are collected per day, said Fred Turner, chief executive of Curative, the company that developed it.

Turner sees an advantage to the swabbing strategy. The self-swab procedure takes only 20 to 30 seconds, while producing enough saliva for testing can take people two to three minutes, and sometimes longer, he said. “That might not sound like much difference,” Turner said, “but it is when you’re trying to push 5,000 people through a test site.”

Curative’s three labs process tens of thousands of tests from jurisdictions across the country in addition to L.A., Turner said. A test developed at SUNY Upstate Medical University, which is expected to become available at state labs around New York, also uses an oral swab.

For the Curative test, a health care worker is supposed to oversee the sample collection —reminding people to cough to bring up fluids, for example. When investigators at the University of Illinois launched what they called a “Manhattan Project” to develop a saliva test by mid-June, they hoped to make it possible for people to visit a collection site, drool into a test tube, seal it and drop it off without the aid of a health care worker.

The university is now testing more than 10,000 people a day at its three campuses and is seeking to expand access to communities across the state and country, said chemistry professor Paul Hergenrother, who led the research team. Like the similar Yale test, it is being made freely available to other laboratories. The University of Notre Dame, in Indiana, recently adopted it.

Like tests using nasopharyngeal and other kinds of nasal swabs, these saliva tests are based on PCR technology, which amplifies small amounts of viral genetic material to facilitate detection. Both the Yale and University of Illinois tests have managed to simplify the process by eliminating a standard intermediate step: the extraction of viral RNA. Their protocols also don’t require viral transport media, or VTM — the chemicals generally used to stabilize the samples after collection.

“You don’t need swabs, you don’t need health care workers, you don’t need VTM, and you don’t need RNA isolation kits,” Hergenrother said.

In correspondence published in the New England Journal of Medicine, the Yale team reported detecting more viral RNA in saliva specimens than in nasopharyngeal ones, with a higher proportion of the saliva tests showing positive results for up to 10 days after initial diagnosis.

The National Basketball Association provided $500,000 in support for the Yale project, said David Weiss, the NBA’s senior vice president for player matters. He said the Yale team’s decision to eliminate the process of RNA extraction, which separates the genetic material from other substances that could complicate detection, involved trade-offs but did not compromise the value of the test.

“Any molecular test that has an RNA extraction step is almost by definition going to be more sensitive, but it will also be more expensive and take longer and use supplies that are in shorter supply,” he said. “If we’re trying to look at surveillance testing to open up schools and nursing homes, a test that’s still very sensitive and a lot cheaper is an important innovation.”

Prices for coronavirus tests vary widely, running upward of $100. Tests based on the Yale or University of Illinois protocols, which require only inexpensive materials, could be available for as little as $10. The Curative testing service, which includes collection and transportation of samples as well as the laboratory component, averages around $150 per test depending on volume, said Clayton Kazan, chief medical director of the L.A. County Fire Department, which uses the tests.

Despite the advances in sample collection, tests using PCR — polymerase chain reaction — technology still require laboratory processing. Researchers have been investigating other approaches, including saliva-based antigen tests, that could be self-administered at home and would provide immediate results. (While PCR can detect coronavirus genetic material, antigen tests look for viral proteins that can signify a current infection.)

At least one company has announced it is seeking emergency use authorization for a saliva antigen test, although two others have dropped plans to develop their own versions as infeasible, according to The New York Times. Meanwhile, scientists at Columbia University, the University of Wisconsin and elsewhere are investigating the use of saliva with other kinds of rapid-test technologies.

“There’s tons of interest” in an at-home saliva test, noted Yvonne Maldonado, chief of pediatric infectious diseases at Stanford University School of Medicine.

“People really do want to get that pregnancy-type kit out there,” she said. “You could basically send people a little packet with little strips, and you pull off a strip every day and put in under your tongue.”

This KHN story first published on California Healthline, a service of the California Health Care Foundation.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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What You Should Know:

– physIQ has been selected by the National Institute of Health (NIH) to develop an innovative AI-based COVID-19 digital biomarker solution to address the COVID-19 pandemic.

– Early detection of COVID-19 decompensation in patients
is complicated by infrequent and non-specific clinical data. The first-in-kind
tool will collect and analyzes continuous physiologic data could provide early
clinical indicators of COVID-19 decompensation.

The National Cancer
Institute (NCI) and the National
Institute of Biomedical Imaging and Bioengineering (NIBIB) of the National Institutes of Health (NIH), have
awarded physIQ a contract to develop an
AI-based COVID-19 Decompensation Index (CDI) Digital Biomarker to address the
rapid decline of high-risk COVID-19 patients.

Why It Matters

Today, high-risk COVID-19
patients and their providers are finding out too late that in the disease
continuum they are getting sicker and need urgent care. The new early warning
system under development could allow providers to intervene sooner when a
COVID-19 patient is clinically surveilled from home and begins to worsen.
Rather than relying on point measurements, such as temperature and SpO2, that
are known to be lagging or insensitive indicators of COVID-19 decompensation,
continuous multi-parameter vital signs will be used to establish a targeted
biomarker for COVID-19.

Despite the technological advances and attention paid to COVID-19, the healthcare community is still monitoring patient vitals the very same way as we did in the 1800s,” said Steven Steinhubl MD, Director of Digital Medicine at Scripps Translational Science Institute (STSI) and a physIQ advisor. “With the advances in digital technology, AI and wearable biosensors, we can deliver personalized medicine remotely giving caregivers new tools to proactively address this pandemic. For that reason alone, this decision by the NIH has the potential to have a monumental impact on our healthcare system and how we manage COVID-19 patients.”

COVID-19 Decompensation Index (CDI) Digital Biomarker Development

PhysIQ will develop and validate a CDI algorithm that builds off existing wearable biosensor-derived analytics generated by physIQ’s pinpointIQTM end-to-end cloud platform for continuous monitoring of physiology. The data will be gathered through a clinical study of COVID-19 positive patients in collaboration with the University of Illinois Hospital and Health Sciences System (UI Health) and build upon work already in-place for monitoring COVID-19 patients convalescing at home.

In the development phase of this project, physIQ and its clinical partner will monitor participants who are confirmed COVID-19 positive, whether recovering at home or following discharge from the hospital. During the validation phase, physIQ will evaluate lead time to event statistics, decompensation severity assessments, and the ability for CDI to predict decompensation severity.

“The application of the CDI may provide a universal indicator of decompensation,” said Karen Larimer PhD, ACNP-BC, study PI and physIQ’s Director of Clinical Development. “Application of this technology could detect COVID-19 decompensation and prevent hospitalization or morbidity events in both scenarios.”

The study is designed to capture data from a large, diverse
population to investigate CDI performance differences among subgroups based on
sex/gender and racial/ethnic characteristics. This project will not only enable
the development and validation of the CDI, it will also collect rich clinical
data correlative with outcomes and symptomology related to COVID-19 infection.

This index will build on physIQ’s prior FDA-cleared, AI-based multivariate change index (MCI) that has amassed more than 1.5 million hours of physiologic data, supporting the development of this targeted digital biomarker for COVID-19. This will enable new research and further insight into using digital health to advance the public health response.

Good bacteria, those living in symbiosis with us, are nourished by fruits, vegetables, grains, and beans, whereas bad bacteria, those in dysbiosis with us and possibly contributing to disease, are fed by meat, junk food and fast food, seafood, dairy, and eggs, as you can see at 0:12 in my video Microbiome: We Are What They Eat. Typical Western diets can “decimate” our good gut flora.

We live with trillions of symbionts, good bacteria that live in symbiosis with us. We help them, and they help us. A month on a plant-based diet results in an increase in the population of the good guys and a decrease in the bad, the so-called pathobionts, the disease-causing bugs. “Given the disappearance of pathobionts from the intestine, one would expect to observe a reduction in intestinal inflammation in subjects.” So, researchers measured stool concentrations of lipocalin-2, “which is a sensitive biomarker of intestinal inflammation.” As you can see at 1:13 in my video, within a month of eating healthfully, it had “declined significantly…suggesting that promotion of microbial homeostasis”—or balance—“by an SVD [strict vegetarian diet] resulted in reduced intestinal inflammation.” What’s more, this rebalancing may have played a role “in improved metabolic and immunological parameters,” that is, in immune system parameters.

In contrast, on an “animal-based diet,” you get growth of disease-associated species like Bilophila wadsworthia, associated with inflammatory bowel disease, and Alistipes putredinis, found in abscesses and appendicitis, and a decrease in fiber-eating bacteria. When we eat fiber, the fiber-munching bacteria multiply, and we get more anti-inflammatory, anti-cancer short-chain fatty acids. When we eat less fiber, our fiber-eating bacteria starve away.

They are what we eat.

Eat a lot of phytates, and our gut flora get really good at breaking down phytates. We assumed this was just because we were naturally selecting for those populations of bacteria able to do that, but it turns out our diet can teach old bugs new tricks. There’s one type of fiber in nori seaweed that our gut bacteria can’t normally breakdown, but the bacteria in the ocean that eat seaweed have the enzyme to do so. When it was discovered that that enzyme was present in the guts of Japanese people, it presented a mystery. Sure, sushi is eaten raw, so some seaweed bacteria may have made it to their colons, but how could some marine bacteria thrive in the human gut? It didn’t need to. It transferred the nori-eating enzyme to our own gut bacteria.

“Consequently, the consumption of food with associated environmental bacteria is the most likely mechanism that promoted this CAZyme [enzyme] update into the human gut microbe”—almost like a software update. We have the same hardware, the same gut bacteria, but the bacteria just updated their software to enable them to chew on something new.

Hardware can change, too. A study titled “The way to a man’s heart is through his gut microbiota” was so named because the researchers were talking about TMAO, trimethylamine N-oxide. As you can see at 3:33 in my video, certain gut flora can take carnitine from the red meat we eat or the choline concentrated in dairy, seafood, and eggs, and convert it into a toxic compound, which may lead to an increase in our risk of heart attack, stroke, and death.

This explains why those eating more plant-based diets have lower blood concentrations of TMAO. However, they also produce less of the toxin even if you feed them a steak. You don’t see the same “conversion of dietary L-carnitine to TMAO…suggesting an adoptive response of the gut microbiota in omnivores.” They are what we feed them.

As you can see at 4:17 in my video, if you give people cyclamate, a synthetic artificial sweetener, most of their bacteria don’t know what to do with it. But, if you feed it to people for ten days and select for the few bacteria that were hip to the new synthetic chemical, eventually three quarters of the cyclamate consumed is metabolized by the bacteria into another new compound called cyclohexylamine. Stop eating it, however, and those bacteria die back. Unfortunately, cyclohexylamine may be toxic and so was banned by the FDA in 1969. In a vintage Kool-Aid ad from 1969, Pre-Sweetened Kool-Aid was taken “off your grocer’s shelves,” but Regular Kool-Aid “has no cyclamates” and “is completely safe for your entire family.”

But, if you just ate cyclamate once in a while, it wouldn’t turn into cyclohexylamine because you wouldn’t have fed and fostered the gut flora specialized to do so. The same thing happens with TMAO. Those who just eat red meat, eggs, or seafood once in a while would presumably make very little of the toxin because they hadn’t been cultivating the bacteria that produce it.

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Here’s the link to my video on TMAO: Carnitine, Choline, Cancer, and Cholesterol: The TMAO Connection. For an update on TMAO, see How Our Gut Bacteria Can Use Eggs to Accelerate Cancer, Egg Industry Response to Choline and TMAO, and How to Reduce Your TMAO Levels.

Interested in more on keeping our gut bugs happy? See:

• Microbiome: The Inside Story
• Prebiotics: Tending Our Inner Garden
• How to Change Your Enterotype
• Paleopoo: What We Can Learn from Fossilized Feces
• Gut Dysbiosis: Starving Our Microbial Self
• How to Develop a Healthy Gut Ecosystem
• How to Become a Fecal Transplant Super Donor
• The Role of the Gut Microbiome in Autism
• Gut Microbiome – Strike It Rich with Whole Grains
• Flashback Friday: Effect of Sucralose (Splenda) on the Microbiome
• Flashback Friday: What’s Your Gut Microbiome Enterotype?

In health,

Michael Greger, M.D.

PS: If you haven’t yet, you can subscribe to my free videos here and watch my live presentations:

• 2019: Evidence-Based Weight Loss
• 2016: How Not To Die: The Role of Diet in Preventing, Arresting, and Reversing Our Top 15 Killers
• 2015: Food as Medicine: Preventing and Treating the Most Dreaded Diseases with Diet
• 2014: From Table to Able: Combating Disabling Diseases with Food
• 2013: More Than an Apple a Day
• 2012: Uprooting the Leading Causes of Death

Shots:

• The US FDA has granted ODD and RPD to the Taysha Gene Therapies’ TSHA-101 for GM2 Gangliosidosis. The company expects the therapy to enter the clinic by the end of 2020
• The US FDA’s two designations demonstrated the strength of the translational data package supporting TSHA-101 for GM2 Gangliosidosis
• TSHA-101 is an AAV9-based gene therapy, currently under development for rare, neurodegenerative disease that causes a progressive dysfunction of the CNS

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Nordic Life Science

Shots:

• The US FDA and EMA has received ODD to Galecto’s GB0139 for the treatment of IPF. GB0139 showed significant reduction of YKL-40 biomarker in fibrosis, inflammation, tissue remodeling diseases in its first clinical study after 14 days of treatment
• The EMA cited GB0139’s clinically relevant biomarker data in IPF patients which provides financial incentives, encouraging the development of drugs targeting rare diseases
• GB0139 (formerly TD139) is an inhaled galectin-3 inhibitor, being evaluated in P-IIb GALACTIC-1 study in 450 patients with IP across 100 centers in the US the EU and Canada

Click here, ­to­ read full press release/ article | Ref: PRNewswire | Image: Galecto

President Donald Trump told the American people this week that convalescent plasma is a potential new treatment for COVID-19. His announcement followed the Food and Drug Administration’s decision Sunday to grant fast-track authorization for its emergency use as a treatment for hospitalized COVID patients.

This “emergency use authorization” triggered an outcry from scientists and doctors, who said the decision was not supported by adequate clinical evidence and criticized the FDA for what many perceived as bowing to political pressure.

With all the news swirling around convalescent plasma this week, we thought we’d break it down for you.

1. Convalescent plasma contains antibodies against disease. Donations are being promoted as a potential COVID-19 treatment.

“Convalescent” refers to recovery from a disease. And plasma is the yellowish, liquid part of blood in which blood cells are suspended.

When someone is infected with a virus, the body generates antibodies to fight off the viral particles. Enter COVID-19. If an individual who has recovered from this virus donates their plasma, scientists can isolate the antibodies from the plasma and give it to patients who are still in the early stages of their COVID-19 infection. This infusion, in theory, should help people fight off the virus while their own body catches up and makes its own supply of antibodies.

It’s not a new concept. An infusion of antibodies via plasma has been used as a treatment for other types of diseases, such as rabies.

2. Some experts took issue with the data presented to approve the treatment and thought the FDA action crossed a political line.

An FDA emergency use authorization allows companies and medical providers to deploy unapproved treatments or medical products in a crisis. The FDA said health care providers would be authorized to distribute COVID convalescent plasma to treat suspected or confirmed patients with COVID-19 while in the hospital.

Before the authorization, some top researchers and clinicians at the National Institutes of Health felt there was not sufficient scientific evidence to support pushing the treatment forward.

“A randomized placebo control trial is the gold standard,” said Dr. Howard Koh, who was an assistant secretary at the Department of Health and Human Services from 2009 to 2014 under President Barack Obama. “If you don’t have that standard and don’t have some evidence from a high-quality study or [a randomized controlled trial], you are left with suboptimal science and treatments in the long run that may not prove to work.”

Koh also said that for other COVID-19 treatments including the medication remdesivir, a randomized clinical trial had been done before the FDA OK’d it for emergency use.

When the emergency authorization for convalescent plasma was announced, HHS officials pointed to findings from a Mayo Clinic preliminary analysis as the rationale. The analysis has not been reviewed by other scientists and doctors.

Suspicions of a political motive behind the decision were heightened because the authorization came one day before the start of the Republican National Convention.

“The timing raises so many questions,” said Koh, also a professor of the practice of public health leadership at Harvard University. “I think this announcement shakes the confidence of the medical community in the rigor of the FDA decision-making process.”

Trump tweeted just a day before the FDA’s action, “The deep state, or whoever, over at the FDA is making it very difficult for drug companies to get people in order to test the vaccines and therapeutics. Obviously, they are hoping to delay the answer until after November 3rd. Must focus on speed, and saving lives!”

Scott Gottlieb, a former Trump administration FDA commissioner, offered his take in a tweet the day after the announcement: “Plasma may provide a benefit, and it could be meaningful for certain patients, but we need more evidence to prove it. The data FDA had supports an authorization for emergency use, where the standard is ‘may be effective’ but we need better studies to confirm preliminary findings.”

3. Dr. Stephen Hahn, the current FDA commissioner, misrepresented the data on the treatment’s effectiveness during Sunday’s press conference. Hahn later corrected himself.

The Mayo Clinic analyzed outcomes of patients who were given a low dose of plasma and those given a high dose. Those who got the high dose had a lower seven-day mortality rate (8.9%) compared with the seven-day mortality rate of those given a low dose (13.7%).

Dr. Adam Gaffney, a critical care doctor and instructor in medicine at Harvard Medical School, said these two variables were used to calculate what is known as a “relative risk reduction,” or the percent difference between the risk of two different treatment outcomes. In this case, the risk reduction between the high dose and low dose of plasma is 35%.

That’s the number Hahn misrepresented.

“Many of you know I was a cancer doctor before I became FDA commissioner, and a 35% improvement in survival is a pretty substantial clinical benefit,” said Hahn. “What that means is — and if the data continue to pan out — 100 people who are sick with COVID-19, 35 would have been saved because of the administration of plasma.”

But, that was an incorrect statement. Hahn had confused relative risk with absolute risk, as many members of the medical community later pointed out. Absolute risk reduction refers to the number of people who experienced reduced mortality from a treatment compared with the rest of the entire population who didn’t get the treatment. The absolute risk reduction in this situation is probably closer to 3-5 cases out of 100.

On Monday night, Hahn issued a tweet to set the record straight: “I have been criticized for remarks I made Sunday night about the benefits of convalescent plasma. The criticism is entirely justified. What I should have said better is that the data show a relative risk reduction not an absolute risk reduction.”

Hahn also noted in the Twitter thread that the agency’s decision was not political, but “made by FDA career scientists based on data submitted a few weeks ago.” He also said the approval was not final and the FDA could revoke authorization if needed.

4. President Trump referred to the use of blood plasma during the RNC, and is likely to do so throughout the remainder of his presidential campaign.

During the first night of the Republican National Convention, in a meeting with a group of first responders, Trump told a police officer who had recovered from COVID-19 that her blood was “valuable.”

“Once you’re recovered, we have the whole thing with plasma happening. That means your blood is very valuable, you know that, right?” Trump said. Vice President Mike Pence also mentioned it in his Wednesday night speech.

5. Critics of the convalescent plasma treatment say there must be randomized clinical trials to prove its effectiveness.

Koh said receiving convalescent plasma doesn’t appear to be dangerous, but a recent study in China did report that 2 in about 100 people experienced adverse events associated with the treatment.

And multiple experts said a randomized clinical trial is necessary to ensure that the mortality outcomes shown in the Mayo Clinic analysis weren’t confounded by other factors.

A randomized clinical trial would involve one group receiving a placebo and another group receiving the treatment. Who is assigned to each group would be completely random to eliminate bias.

Gaffney said he noticed that patients in the low-dose plasma group seemed to be sicker than those in the high-dose plasma group — which could have affected the Mayo Clinic’s findings.

“To ensure that the effect we see is the effect of the intervention, and not a manifestation of differences in how sick the two groups are,” the trial has to be randomized, said Gaffney.

The Mayo Clinic analysis also reported that some patients who received plasma also took remdesivir or steroids, which could have influenced their mortality outcomes.

Dr. Eric Topol, director of the Scripps Research Translational Institute, said, at best, he sees the outcomes of this analysis as a hypothesis that needs to be tested in a randomized clinical trial. “No survival benefit has been proven,” he wrote in an email.

Kaiser Health News (KHN) is a national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation which is not affiliated with Kaiser Permanente.

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Already under fire for what some view as a premature authorisation of convalescent plasma for COVID-19, the FDA is now being accused of a blunder that could render current supplies unusable.

Bioindustry association MichBio says the FDA’s labelling requirements for COVID-19 convalescent plasma (CCP) – which were published alongside the emergency use authorisation – could lead to “hundreds, if not thousands, of in-date, ready to transfuse CCP units across the country being rendered unusable.”

The problem lies with the requirement that each unit of plasma includes information on the concentration of antibody titres, something which wasn’t required for CCP units collected ahead of the EUA on 23 August.

MichBio adds that while it may be possible to relabel some of these units appropriately, that represents “a monumental undertaking by blood centres, and any transfusion services that have units in-house are unable to relabel them.”

Convalescent plasma treatment involves giving COVID-19 patients antibody-rich blood plasma from people who have already recovered from the disease, and has already been used in 70,000 people in the US under an Expanded Access Programme launched in April.

Two US lawmakers – Reps Debbie Dingell (D-MI) and Fred Upton (R-MI) – have already picked up on the issue and have written to FDA Commissioner Stephen Hahn, asking that the FDA “take prompt action to ensure that these requirements do not unduly inhibit patient access to convalescent plasma.”

They say that delays to treatment with CCP “could have an impact on patient outcomes, with preprint data indicating that time to transfusion is a key factor correlated with lessening the severity or shortening the length of illness.”

The preprint data referred to is a Mayo Clinic study used to support the EUA based on data taken from patients treated with plasma under the Expanded Access Programme.

It’s another embarrassment for the FDA, coming after Commissioner Hahn was forced to back-pedal on statements he made during a press conference to announce the EUA last Sunday, particularly that the use of CCP could improve survival in COVID-19 patients by 35%.

“What that means is…in 100 people who are sick with COVID-19, 35 would have been saved because of the administration of plasma,” said Hahn.

As it stands, only one or two people out of 100 would be expected to die if they had COVID-19, so the claim makes little sense.

Moreover, the figure is a misinterpretation of data from the Mayo study, which compared plasma with high antibody titres to plasma with low titres, not plasma versus no plasma, and revealed a relative rather than absolute risk reduction – as conceded by Dr Hahn later.

Criticism of the FDA is coming from many sides, including influential Scripps Research scientist Eric Topol, who wants the FDA to hold a second press conference to correct the mis-communication, and believes the credibility of the agency is at an all-time low.

The overblown claims also led to accusations that the FDA was playing politics, and pandering to President Trump’s desire to show evidence of progress on COVID-19 as the US election looms, a claim that Dr Hahn also denies, saying “the decision was made by FDA career scientists based on data submitted a few weeks ago.”

Nevertheless, the labeling controversy further undermines that credibility and according to MichBio “if not rapidly corrected, will lead to significant delays in transfusion of patients across the country for the foreseeable future, or, put transfusion services licenses at risk for wilfully violating FDA requirements.”