Unlocking the potential in rare disease research with decentralised trials

A survey by rare disease patient network Raremark found that 86% of the community members asked were interested in taking part in clinical trials. CEO Jeremy Edwards looks at how decentralised trial models can solve some of the challenges for clinical trial recruitment in rare disease.

With low and geographically dispersed patient populations in rare disease, deciding where best to place trial sites can be a headache for sponsors. Sites need to be close enough for travel, and the number of site visits and trial appointments must also be manageable for people living with the complications of having little-known and rarely-researched orphan diseases.

Decentralising trials, a topic revitalised by the COVID-19 pandemic, seems like a no-brainer, but would people with rare diseases be open to them? In fact, how do rare disease patients even feel about clinical trials? A survey of members of the Raremark community found that 86% of respondents were interested in taking part in a clinical trial now or in the future.  The people who took part were from across the world, living with hemophilia, idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease.

Research and development will always be important to rare disease patients

This finding may come as a surprise to anyone who read this CISCRP report last year. The report found that people’s willingness to take part in clinical trials in 2020 fell dramatically compared to previous years – however that survey was conducted with the wider public while the Raremark survey focused specifically on people with rare diseases.

Progress in research and development for better treatments will always be a priority for rare disease patients. There are around 7,000 known rare diseases and only a fraction of those have treatments available.

“For those participants that did not want to take part in a clinical trial, reasons given included not knowing where to start looking for information as well as the COVID-19 pandemic, hesitance about traveling and not wanting to disrupt their medication plans.”

Getting a diagnosis is not always easy; people have to battle many years because rare diseases are not as extensively researched or known about as more common ailments. Common experiences heard from people with rare diseases can be things like:

“It took me a full 10 years [to get a diagnosis]! I experienced my first symptoms when I was 12, they progressed at 13 and I got a correct diagnosis at 22.”

“No one ever knows what to do with you! It’s a pure guessing game!”

“Never heard of it before my mum was diagnosed, also nothing was actually explained to us, we found out most information on the internet!”

“I had to suffer hours of excruciating pain before I could be taken seriously.”

This illustrates the toll that having a rare disease can have on the individual and their family’s lives. This is a key reason why patients view the continued progress of research and development as important. For those participants that did not want to take part in a clinical trial, reasons given included not knowing where to start looking for information as well as the COVID-19 pandemic, hesitance about traveling and not wanting to disrupt their medication plans.

Positive patient feedback for decentralised trials

While the idea of decentralised and virtual trial methods – i.e. taking the trial to the patient, rather than the patient to the trial – has been a topic of interest for some time, COVID-19 has brought it to the forefront.

Investment into companies that offer decentralised and virtual trial solutions increased dramatically in 2020 and this is translating into practical discussions regarding the implementation of decentralised trials. With all of this in mind, Raremark was interested to learn what people with rare diseases think about them.

We asked people where they would prefer to receive their trial medication and monitoring appointments if they decided to take part in a clinical trial. 57% of participants said they would like to receive the trial medication through a mix of clinic, home, and nearby hospital visits, and 45% would like the same mix for how they are monitored during trials.

Only a few people surveyed said they wanted to have all their appointments only at the clinic or only at their home.

We also asked participants if they would have any concerns about taking part in a trial that would not require them to travel to the clinic for all their appointments – 87% of survey participants expressed no concerns about this approach to trials.

When asked about features of decentralised trial designs that people found most attractive, the top reasons given were the convenience of having more of the study take place at home and less time traveling, as well as the use of technology, both to communicate with research staff and to have their health monitored.

For the 13% of participants who did have some concerns about these types of trials, the reasons they gave for their reservations included possible adverse reactions to the drug, and not having specialists there to help them. A few others said they would struggle to comprehend all the trial literature on their own.

What does this mean for clinical trials in the future?

The COVID-19 pandemic has turned many aspects of clinical research upside down; however it will also be a powerful agent for change in the way the pharma industry conducts clinical trials. One of the barriers to uptake of virtual trials in the past has been the assumption that it requires a fully decentralised approach. We’re now seeing a shift in focus towards hybrid models where sponsors identify aspects of a trial that could be done virtually or at locations closer to patients’ homes.

Clearly, face-to-face visits will still be necessary, but through a decentralised approach these can be limited and balanced with home visits from nurses or by engaging local clinics and hospitals so some appointments can be conducted at the patient’s regular doctor’s office.

There are already tools available and more being developed that can help make decentralised trials a possibility, including software that capture patient consent easily, at home video consultation tools that make staying in contact with patients straightforward, apps for nurses to use when they’re making home visits, and even integrated wearable devices that collect the relevant data needed for a trial such as biomarker data, physical activity and motor symptoms.

Adopt a person-first approach to increase engagement and retention

The best way to keep people engaged in trials and reduce trial dropouts is taking a person-first, approach.

It is essential to remember that the people who take part in clinical trials may have jobs, work or childcare commitments, or the nature of their disease may mean certain tasks take priority over others.

To keep people living with rare diseases engaged, clinical trials must adapt to fit as seamlessly as possible into people’s lives. Decentralised and hybrid trial models can help by removing obstacles that people may find disruptive to their everyday life when taking part in a trial, such as the burden of traveling to trial sites multiple times.

Additionally, there are several ways to keep the people who opted to participate interested and engaged, whether a decentralised approach is adopted. These include access to information and keeping communication lines open before, during and after a trial.

Participants should always feel comfortable asking questions and voicing concerns, so it is important to use every opportunity to keep people informed and treat their time and commitment with respect.

A few simple ways to achieve this include:

  • Replying to patient emails and call-backs promptly – there’s nothing worse than spending lots of time looking for patients only to lose them because of a missed call
  • Catering for work schedules, planning calls and screening appointments accordingly – remember patients have lives too.
  • Addressing any anxieties patients and caregivers have, including coming off existing medication, as well as questions regarding the trial drug or the tests and procedures involved in the trial.

Conclusion

Shifting towards decentralised trial designs can help solve some of the challenges around recruiting patients for orphan drug trials. Designing trials where participants do not have to travel as often and where some appointments can be done virtually will make them more convenient and cause less disruption to people’s lives. Rare disease patients are open to these trial types but still need some in-person appointments, therefore a hybrid approach with a mix of in-person and virtual appointments is desirable.

Ultimately, by understanding the world that rare disease patients are living in and designing trials accordingly, sponsors can significantly improve clinical trial engagement and participation.

You can learn more about our findings and the opinions around clinical trials in rare disease here.

About the author

Jeremy Edwards is the CEO of Raremark. He has been focused on aiding the biopharmaceutical and health sciences industry in the development of key compounds and new therapies for over 25 years.  His background includes executive leadership positions across the clinical development continuum; from full-service CROs, to highly specialised imaging modalities, to niche patient-focused service providers.

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Allergic reactions prompt Moderna COVID vaccine pause in California

Public health authorities in California are seeking a halt on dosing of one lot of Moderna’s COVID-19 vaccine after reports of allergic reactions at one immunisation clinic.

According to state epidemiologist Dr Erica Pan, there were a higher-than-expected number of suspected allergic reactions at a community clinic being used to administer the shot, with some people needing medical attention in a 24-hour period.

For now there is little information about the Moderna vaccine reactions, other than they are centred around a specific manufacturing lot – number 041L20A –  and that “fewer than 10” cases of allergic reactions were reported.

More than 330,000 doses from that lot have already been administered in California since the start of the vaccine roll-out, according to state department of public health. The clinic in question switched to another lot of Moderna vaccine after closing for a few hours.

There were also reports of allergic reactions during the initial roll-out of the Pfizer/BioNTech vaccine, including some cases of anaphylaxis, which also resulted in a temporary pause in dosing at some centres.

Last week, the Centers for Disease Control and Prevention (CDC) published new data which identified 21 cases of anaphylaxis after administration of a reported 1.9 million first doses of the Pfizer/BioNTech shot, mainly within 15 minutes of the injection.

That was equivalent to 11 cases per million doses, according to the agency, which says the reactions can be managed using patient screening for allergies, observation periods after dosing and having epinephrine injections on hand as a precaution.

Both the Pfizer/BioNTech and Moderna vaccines are based on mRNA and use an excipient – called polyethylene glycol (PEG) – that some scientists suggest could be responsible for the allergic reactions, according to a report in the journal Science.

“Our goal is to provide the COVID vaccine safely, swiftly and equitably,” said Dr Pan in a statement.

“Out of an extreme abundance of caution and also recognising the extremely limited supply of vaccine, we are recommending that providers use other available vaccine inventory and pause the administration of vaccines from Moderna lot 041L20A until the investigation by the CDC, FDA, Moderna and the state is complete.

While no vaccine or medical procedure is without risk, the risk of a serious adverse reaction is very small, according to the department. At last count, California had recorded almost 3 million COVID-19 cases, with just over 33,000 deaths, placing it among the worst affected states in the US.

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A collaborative approach to greater diversity in clinical trials

The need for diversity in clinical trial populations has been a topic of discussion across regulators and the industry in general for decades. Despite the introduction of US policies, beginning with the 1993 National Institutes of Health (NIH) Revitalization Act which called for the inclusion of more women and communities of colour in clinical trials, clinical trial data has remained largely based on healthier Caucasian subjects with minimal representation from minorities (African American, Latinx, Asian, Native Americans), the elderly, young, and those with co-morbidities.

To encourage more of a focus on clinically relevant populations, the US Food and Drug Administration (FDA) recently released “Enhancing the Diversity of Clinical Trial Populations – Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry” to increase participant access to clinical trials and the enrolment of underrepresented populations to ensure clinical trial data reflects the population most likely to use the drug if approved1. The guidance encourages sponsors to remove overly restrictive and legacy exclusions, broaden protocol eligibility criteria, and improve trial recruitment practices so trial data is clinically relevant for the end user.

Historical performance data, like that provided in FDA Drug Trials Snapshots, has shown that using traditional recruitment practices by themselves does not enhance the diversity of clinical trial populations. Fundamental barriers and deeply rooted mistrust of medical research motives among communities of colour require a more thoughtful and deliberate approach to participant outreach. PPD has seen recent successes in the recruitment of more clinically relevant trial populations through the implementation of patient-centered trial solutions designed to address the most common barriers to clinical trial participation among these diverse patient populations – mainly trust, understanding, awareness, access, time, and cost, especially when delivered in collaboration with organisations focused on communities of colour and community leaders to ensure optimal receptivity.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Interstitial Lung Disease (ILD) Drug Development Summit

Network with over 50 other industry pioneers including Ionis Pharmaceuticals, Genentech, Vicore, and AnaMar to hear first-hand how the latest scientific research is innovating and upgrading ILD therapeutics at this trailblazing new meeting.

The 1st Interstitial Lung Disease Drug Development Summit is a ground-breaking new conference dedicated to helping you drive forward the development of effective therapies for chronic fibrosing ILDs and achieve success in anti-fibrotic drug development beyond IPF.

Join the world’s top ILD specialists as they guide you through the seminal research and insights you need to confidently define, understand, and develop clinically effective antifibrotic therapies against connective tissue disease, rheumatoid arthritis, hypersensitivity pneumonitis, Goodpasture’s syndrome, and much more.

To view the full agenda, visit our website here: https://ter.li/8ug2my

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Enabling new models of care: pursuing pharma’s partnership potential

There is a huge, ongoing shift in how health and wellness is approached in the UK, and the changes will have important implications for NHS-industry partnerships.

Transformative change is coming to the NHS and is set to radically alter how the UK’s health service cares for people at a population level.

The NHS Long Term Plan signposted this change, with its emphasis on preventative health, and the forthcoming expansion of the Integrated Care Systems programme continues this direction of travel on a path towards the long-cherished hope of joining up health and social care.

As new approaches to healthcare attainment take hold there will be some degree of uncertainty among pharmaceutical companies about where they fit into the new structures and the holistic care they seek to provide.

But they’re not the only stakeholders working to map out how to enable new models of care and what their role should be.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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‘No going back’ for clinical trials after COVID

Trial sites have adapted swiftly to the restrictions of COVID-19, and patients have seen many knock-on benefits as a result. The next step is ensuring the industry does not regress to old ways of working once the pandemic is over, say Karen McIntyre and Allyson Small.

COVID-19 has changed everything for clinical trials – but in most cases these are changes that were well overdue.

“For years and years, the industry has debated the practicalities and safety of decentralising clinical trials, using telemedicine, and where study activities should take place,” says Karen McIntyre, executive director, global lead Catalyst Program & site relationships at Syneos Health. “We were always having these discussions, but nothing moved forward.”

When the pandemic hit, regulators around the world rapidly updated their guidelines to reflect the realities of conducting trials amidst lockdowns and social distancing mandates.

“For example, drugs are now able to be delivered directly to patients to allow for a clinical trial visit to take place remotely,” says McIntyre.

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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How patient insights are changing trial solutions

Patients have been asking for patient-centric trial solutions for years – the industry just hasn’t been listening. That’s according to Medidata’s Anthony Costello, who was bringing patient feedback into product design long before COVID-19. He tells us what insights pharma has been missing out on and how they can be harnessed to build better solutions.

COVID-19 might have forced the industry to leave behind its reticence around remote and decentralised trials, but according to Anthony Costello, senior vice president of mobile health at Medidata, this reticence wouldn’t have existed in the first place had the industry been genuinely listening to patients.

“Patients have not been saying anything new during COVID-19, but the difference is that the industry has woken up and started paying attention,” he says.

“Patients have long wanted more and better technology to use in studies so that they don’t have to visit sites so often, but the industry has been very reluctant to go in that direction.”

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Pfizer Reports the First Patients Dosing of PF-06939926 in P-III CIFFREO Study for DMD

Shots:

  • The first patient has been dosed in P-III CIFFREO study assessing PF-06939926 vs PBO in 99 ambulatory male patients aged 4-7yrs. with DMD across 55 sites in 15 countries.
  • The first patient was dosed at a site in Barcelona, Spain on Dec 29, 2020. The 1EPs of the study is the change in NSAA @1yr, while patients will be followed in the CIFFREO study for 5yrs. after treatment with PF-06939926
  • PF-06939926 is an investigational rAAV9 capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor and has received the US FDA’s FT designation in Oct’2020 and ODD & RPD in May’2017

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Wall Street Journal

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IL-6 drugs do work in COVID-19, says UK, as it plans NHS use

New data means that IL-6 drugs from Roche and Sanofi that had been all-but written off as coronavirus therapies will now be offered routinely to COVID-19  patients in intensive care in the UK.

The renaissance of the two therapies comes on the back of the REMAP-CAP trial, which found that the IL-6 inhibitors RoActemra (tocilizumab) and Kevzara (sarilumab) reduced the relative risk of death by 24% when administered to COVID-19 patients within 24 hours of entering intensive care, and reduce the time spent in hospital by seven to ten days.

Both RoActemra and Kevzara have failed to hit their objectives in earlier studies, leading to speculation that inhibiting IL-6 wasn’t a valid approach to treat severe COVID-19, but the new independent study turns that view on its head.

Crucially, their benefits seem to stack with that of the corticosteroid dexamethasone, the first drug to be shown to improve survival in seriously-ill COVID-19 patients in the RECOVERY trial.

The death rate for those in intensive care units on dexamethasone and respiratory support alone was 35%, but reduced to 28% when RoActemra was administered as well.

“The data shows that tocilizumab, and likely sarilumab, speed up and improve the odds of recovery in intensive care, which is crucial for helping to relieve pressure on intensive care and hospitals and saving lives,” said UK deputy chief medical officer Prof Jonathan Van-Tam.

The data has emerged as the government unveiled figures showing there are currently around 30,000 COVID-19 patients in hospitals, up nearly 40% on the previous peak during the first wave of the pandemic in April.

There are already ample supplies of RoActemra in the UK, so that drug in particular will be recommended for use “immediately” in patients admitted to intensive care with the virus, it said, saying that could potentially save “hundreds of lives”.

Roche welcomed the results, saying it was still in the process of analysing data from the COVACTA and EMPACTA trials, which generated negative and positive results for its drug respectively in patients hospitalised with COVID-19 associated pneumonia.

“Previous trials using IL-6 receptor agonists have showed no clear benefit on either disease progression or survival in COVID-19 patients, but those studies included less severely ill patients and started treatment at different stages in the disease course,” said Professor Anthony Gordon of Imperial College London, the trial’s lead investigator in the UK.

“A crucial difference may be that in our study, critically ill patients were enrolled within 24 hours of starting organ support. This highlights a potential early window for treatment where the sickest patients may gain the most benefit from immune modulation treatment,” he added.

REMAP-CAP has been running since 2016, with the aim of putting dozens of drugs through their paces to see if they can improve the prospects of people with severe community-acquired pneumonia (CAP) caused by influenza, but was expanded to include COVID-19 patients after the pandemic took hold.

It included more than 800 pneumonia patients in intensive care with suspected or proven COVID-19, of which around three-quarters were recruited from UK NHS trusts, but hasn’t yet been subjected to the scrutiny of peer review.

“This news is a positive step in the fight against COVID-19, giving doctors and the NHS another weapon in their armoury to treat critically ill patients,” said Marius Scholtz, chief medical officer at Roche Products Ltd. “It also increases the collective scientific understanding of COVID-19.”

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True patient-focused research through decentralised and hybrid trials

How can pharma improve the patient-centricity of its trials during COVID-19 and beyond? Experts from across the sector give their thoughts on the key approaches and technologies that are driving patient engagement forward.

With COVID-19 presenting new barriers to running and recruiting for clinical trials, making studies patient-centric is more important now than ever before.

According to one analysis, conducted by Global Data, approximately 67% of trial disruptions during the early stages of the pandemic were due to the suspension of enrolment, followed by the delayed start of planned trials at 18.4% and slow enrolment at 14.4%.

Ensuring that trials are easy to access and don’t overly burden the patient is essential amidst these potential disruptions – but a truly patient-centric trial has benefits beyond enrolment.

Trials that are engaging and easy to partake in can lead to higher adherence, higher satisfaction, improved data quality and overall performance, and can even give participants a more positive view of the sponsor company in terms of their commitment to bring new treatments to patients.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Alnylam Reports Results of Vutrisiran in P-III HELIOS-A Study for Patients with hATTR Amyloidosis with Polyneuropathy

Shots:

  • The P-III HELIOS-A study assessing vutrisiran (25mg, SC, once every 3mos.) vs patisiran (0.3 mg/kg, IV, q3w) in 164 patients in a ratio (3:1) with hATTR amyloidosis with polyneuropathy
  • The study met its 1EPs & 2EPs i.e. change from baseline in the mNIS+7 @9mos. & changes in QoL assessed by Norfolk QoL-DN and gait speed assessed by the timed 10-MWT respectively. Additionally, therapy showed improvements in the 9mos. exploratory cardiac endpoint of NT-proBNP
  • The company plans to submit NDA to the US FDA in H1’21, following the regulatory filing in Brazil and Japan. Moreover, Alnylam intends to submit MAA in the EU on obtaining the results of 18mos. analysis, anticipated in late 2021

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

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Boosting the impact of patient services

New research from Accenture has revealed that adoption of patient support services hasn’t improved since 2015 despite increasing pharma investment. The company’s Jennifer Spada tells us how companies can boost awareness of their programmes to improve patient outcomes.

As part of its drive towards patient centricity, the pharma industry has increasingly been building patient support programmes that can offer beyond-the-pill services to patients. The market for patient engagement solutions was worth $8.8 billion in 2017 and is projected to reach $18.68 billion in 2022, an annual growth rate of 16.2%.

These programmes can help guide a patient through complex information about diagnosis and treatment choices, or aid them with information on the medical and financial aspects of care, and also include day-to-day care management such as medication reminders, symptom monitoring, and nursing support.

Research has shown that when patients utilise these services, adherence increases, quality of life improves, hospitalisations and ER visits are reduced, and survival rates rise.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Behind the scenes: How health systems, EHR vendors will give patients unprecedented access to their data

virtual care

Health systems and EHR vendors have been working for months to comply with the ONC’s final rule on interoperability and information blocking that goes into effect in April and is expected to grant patients unprecedented access to their health information. Here is a look at some of the issues they contended with.

Regeneron’s Antibody Cocktail Demonstrate Promising Results in Hospitalized Patients on Low-Flow Oxygen

Shots:

  • The company reported encouraging initial data from an ongoing P-I/II/III trial of its casirivimab + imdevimab (8,000/2,400mg) in hospitalized COVID-19 patients requiring low-flow oxygen. The results passed the futility analysis as seronegative patients treated with the Ab cocktail had a lower risk of death or receiving mechanical ventilation
  • In seronegative patients, the cocktail reduced the time-weighted average daily viral load through day 7 by -0.54 log10 copies/mL & through day 11 by -0.63 log10 copies/mL, at day 5, the relative reduction vs PBO was -1.1 log10 copies/mL
  • Both the Ab cocktail doses were well-tolerated. The Ab cocktail is designed to block the infectivity of SARS-CoV-2

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Knowledge Ecology International

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Vulnerable should all get COVID-19 shot before summer, says NHS chief

NHS England’s chief executive Sir Simon Stevens has said that all vulnerable people over the age of 50 will be offered a COVID-19 vaccine by “late spring” in a message to healthcare workers.

The prediction comes after the NHS announced on Christmas Eve that more than half a million people had received the Pfizer/BioNTech shot approved in early December, but will depend on additional vaccine supplies coming “on stream”, according to Stevens.

There are around 25 million people classed as vulnerable due to their age or health conditions, and hitting that target will likely rely on the much-anticipated approval of the AstraZeneca/University of Oxford COVID-19 vaccine, said to be coming this week.

The UK has ordered 100 million doses of the AZ vaccine, which unlike the Pfizer/BioNTech shot can be stored and transported at normal temperatures, making it easier to distribute. Around 40 million doses are due to be delivered before the end of March. A third shot from Moderna isn’t expected to be available in the UK until well into next year.

The Pfizer/BioNTech vaccine – which was approved by the EU last week – is being delivered via a network of more than 80 hospital hubs and over 500 GP-led vaccination centres, as well as in care homes in the UK.

That will likely have to be expanded even further if the AZ vaccine is approved and as the immunisation programme gathers pace.

Stevens’ forecast – delivered to staff at a vacciination centre – came alongside a warning that NHS workers are “back in the eye of the storm”, with the number of coronavirus patients in hospitals higher than at the peak of the first wave.

There were around 20,500 hospitalised cases as of this morning, above a peak of just under 19,000 as the first wave hit in April. The UK also recorded a record number of lab-confirmed new cases yesterday at more than 41,000, although that figures reflects a higher level of testing nationally.

At the same time, the new, more transmissible variant of SARS-CoV-2 that was first identified in the UK has been detected in more than 20 other countries around the world, including several EU member states, India, Canada, Japan and Hong Kong.

The leading vaccine developers have said there is no reason their shots will not work against the new variant.

So far there is little evidence of “anti-vaxxer” resistance to the vaccine in the UK. However, in Spain – where more than a quarter of people said they would not take the vaccine in a recent survey – the health ministry has suggested it will set up a registry of people who refuse to be vaccinated and share it with other EU members.

The European Commission has said it expects to deliver 200 million doses of the Pfizer/BioNTech vaccine to EU countries by September 2021.

Novavax vaccine starts US phase 3

Meanwhile, another COVID-19 vaccine has entered the late-stage testing phase in North America. US biotech Novavax has started a phase 3 trial of its recombinant protein-based shot NVX-CoV2373 in the US and Mexico, adding to an ongoing phase 3 trial that started in the UK that is due to read out next year.

The UK government has already signed an agreement with the US biotech to buy 60 million doses of the vaccine in August if trials work out.

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Amgen hands off leprosy, TB candidate to Australian non-profit

Amgen has decided that a drug for leprosy and tuberculosis it inherited as part of its acquisition of Celgene’s psoriasis blockbuster Otezla last year would fare better in the hands of a non-profit company, dedicated to bringing affordable medicines to poorer countries.

AMG 634 – a PDE4 inhibitor being investigated for TB and leprosy complication erythema nodosum leprosum (ENL) – is being licensed to Australia’s Medicines Development for Global Health (MDGH), with Amgen surrendering all rights to the drug.

The US biotech will continue to provide support for two phase 2 trials of AMG 634 in TB and ENL which are due to get underway next year by producing and supplying the drug and funding the ENL trial.

AMG 634 acts as an anti-inflammatory, so it aims to tackle the complications of these chronic infections rather than attacking the infectious organisms themselves.

TB – caused by Mycobacterium tuberculosis – remains a massive public health problem worldwide, affecting 10.4 million patients every year and causing over a million deaths. About a quarter of the world’s population carry the bacterium, and have a 5% to 15% chance of developing the disease.

Current treatments are often inadequate, with resistance emerging to many of the mainstay drugs used to treat the infection, and if it isn’t treated effectively can leave patients with permanent, clinically significant lung damage.

Leprosy meanwhile generally doesn’t hit the headlines like TB, but despite being curable with antibiotic therapy still causes more than 200,000 infections every year, according to the World Health Organization.

Caused by the bacterium Mycobacterium leprae, the infection develops slowly – sometimes it can take a year or more for symptoms emerge – if untreated it can cause progressive and permanent damage to the skin, nerves, limbs, and eyes.

Treatment typically requires months or even years of multidrug therapy with antibiotics – generally with dapsone, rifampicin and clofazimine – but compliance over that long period can be problematic, particularly in countries with less sophisticated healthcare systems.

A significant number of patients go on to develop ENL, an autoimmune complication that can occur many years after being cured of leprosy and can cause permanent nerve damage and disability.

MGDH has already demonstrated that it can bring drugs to market for diseases that mainly afflict low- and middle-income countries, launching moxidectin – the first new treatment for river blindness (onchocerciasis) in 30 years – in 2018.

Its founder and managing director, Mark Sullivan, said the company is “honoured to take over the stewardship of this compound from Amgen… and we will now undertake full development of AMG 634 in hopes of bringing it to patients in need of a treatment for their disease.”

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NHS leaders urge UK government to extend Brexit transition period

NHS leaders have written to the UK prime minister urging him to extend the Brexit transition period by a month to avoid plunging the healthcare service into chaos on 1 January.

The letter, published today by the NHS Confederation – a membership organisation representing NHS leaders from all parts of the health service, outlines concerns about the impact a no-deal outcome will have on delivering care to patients amid winter pressures and a rapid rise in COVID-19 infections.

“The failure to secure a Brexit deal will throw the NHS into further chaos and it will risk the health of patients and the working conditions of our staff,” wrote NHS Confederation chief executive Danny Mortimer. “The NHS might not be perceived to be on the Brexit negotiating table, but the disruption shockwaves from a no-deal outcome could push the NHS’ ability to function over the edge.”

The south east of England is expecting the worst impact, as ambulances and clinicians trying to reach patients face delays due to major congestion on the roads of Kent.

Mortimer added: “They will face these barriers to their critical work while facing some of the highest levels of COVID-19 infections and the additional risks that the increased traffic, lorry parks and congestion could increase the demand for NHS services in the South East. The South East will face disruption even if a deal is reached. Should a deal not be reached, the magnitude and extent of disruption will be of a much greater order.”

Other areas of concern for the NHS Confederation include medical supply shortages, lack of clarity on rules and reimbursement arrangements for EU citizens requiring treatment in the UK and the loss of participation in EU-wide data-sharing platforms to exchange information about health threats and the testing of new treatments.

Uncertain future

Meanwhile, new research from the Nuffield Trust and academics from the Universities of Oxford, Sheffield and Michigan warns of a “perilously uncertain” future facing the UK health sector at the end of the Brexit transition period.

Their study examined nine areas of possible impact: health systems delivery, health systems workforce, medical supplies, health systems financing, leadership and governance, communicable diseases, non-communicable diseases, public health capacity and governance.

It highlights how a combination of migration barriers, an unknown level of disruption to medicines and devices, a prolonged economic slowdown, and barriers to investment in science will impact the health sector almost “immediately and into the future”.

Mark Dayan, Brexit programme lead at the Nuffield Trust, said there were many serious questions that needed answering over the future of the sector beyond the UK’s exit from the European Union.

“There are a particular set of fairly immediate issues which should be in sharp focus – from the double whammy of COVID-19 and Brexit-related workforce shortages and economic fallout to the very real danger of supply chains of medicines and medical devices being disrupted.

“But it doesn’t end there. There are some deeply concerning and unresolved issues that may affect health in the UK over many years to come and potentially risk the health of the UK population.”

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3 Patient Lessons: What Cancer Patients Teach Me

By YASMIN ASVAT

An estimated 1.8 million people in this country may face a cancer diagnosis this year, in what has already been a bleak year of isolation and loss.  

While news of the COVID-19 vaccine rolling out across the U.S. offers hope in a year of 311,000 deaths,  11 million  people face the financial pressure of unemployment, and, approximately 43 percent of the nation reports some symptoms of anxiety or depression.  

It is understandable that a cancer diagnosis now may be too much to bear. And yet, somehow, many patients cope with the diagnosis and the associated uncertainty, fragility, and the threat of mortality with remarkable resilience.  

As a clinical psychologist in the Supportive Oncology program at a major Midwestern cancer center, I witness these quiet heroics every day. 

Since the beginning of the pandemic earlier this year, I have been striving to listen, empathize, support, and help cancer patients cope as their lives have been disrupted by both a cancer diagnosis and COVID-19. These are lessons these patients have taught me. 

Courage is being faced with doing something that utterly terrifies you, and you do it anyway. One of my patients described that leading up to the day of chemotherapy treatment, she is highly anxious, has racing thoughts and worries, and has trouble concentrating and sleeping. The morning of treatment, she vents to her partner about how she doesn’t want to go to the clinic. During the drive, she braces herself repeating, “I don’t want to do this” over and over again. 

Once in the clinic, she tells some of her nurses that she doesn’t want to be there because she worries about COVID-19 exposure, despite all the precautions the clinics have in place. She tells another set of nurses that she is scared of the side-effects of treatment – the disabling fatigue, the nausea, the suppressed immune system. 

And yet, despite her fears and her protests, she stays. The nurses hook her up to the needle, attached to a tube, attached to a bag, attached to an infusion pump that delivers the toxic and cancer-fighting chemotherapy. 

She stays for her children and grandchildren and the desire to see them grow, for her partner, for the hope of a cancer-free future, for her eagerness to live.  She goes home relieved that one more treatment is completed and anxiously anticipates the next round. From this remarkable woman I have learned what it means to live the adage credited to Nelson Mandela, that “courage is not the absence of fear, but the triumph over it.” You may be terrified – and yet you do what needs to be done, anyway.

When you have clarity about what matters, even impossible, heart-breaking decisions are clear. Few decisions in life are more poignant, heartbreaking, and difficult than deciding how to die. Many spend most of their lives conveniently shoving the reality of mortality to the back of their minds, necessarily so, in order to continue persevering through the expected and unexpected challenges of living. Many patients make decisions about their deaths with certainty and acceptance. 

One of these patients was a Latina in her early 50s, Spanish-speaking who battled a particularly aggressive form of metastatic colorectal cancer for two years, with extensive surgery and multiple rounds of chemotherapy. All the while, her priorities were beautifully simple – to spend time with her husband of three decades, enjoy the company of her three children, dance to her favorite music, play with her beloved dog, and find comfort in her faith. 

For as long as her body was able to sustain itself, she did exactly that – she lived, laughed, danced and tolerated all the side-effects of treatment with dignity. And when the day came that saw herself spending her time lying in bed because moving was too painful, sleeping because without medication the pain was so unbearable it would make her scream, she knew what she had to do because she knew what mattered. She said she was living a half-life where she was a shell of herself, breathing but not really living, seeing her family but not really participating in their lives, listening to music but not being able to dance.  

Her doctor suggested yet another round of chemo, with no assurances that it would help, and she declined. She resolved to speak with her family about hospice care. There was sadness, naturally, but no despair. There was wishing she had more time, and there was gratitude for the time she did have. She said there were no regrets, and there was comfort in knowing her family would lean on each other to heal. Knowing what mattered made her choice crystal clear – in the words of German philosopher Nietzsche, she chose to “die proudly when it is no longer possible to live proudly.” 

Being flexible is mandatory for our survival. One of my patients said she was crushed to realize that she would likely be spending the Christmas holiday without her grandchildren. She faced a particularly grueling treatment that knocked her down for eight days, unable to eat, severely fatigued, and spending most of her days sleeping. 

By the ninth day, she started feeling a little better and was able to do a few things around the house and in her beloved garden, and another two weeks later she was back in clinic for the next round of chemotherapy.  She handled this ordeal with the utmost grace and willingness to sacrifice six months of her life for the hope of the rest of her life. 

But faced with the prospect of sacrificing her cherished Christmas season, she almost broke. We talked about safe options to celebrate the holidays, and we philosophized about her values and what brings meaning to her life. In the midst of circumstances that are unprecedented, uncertain, and threating to her physical health and emotional wellbeing, she chose to adapt to survive. 

So she rallied, creatively and flexibly, and planned a virtual baking session — she would make the dough, her husband would drop it off to the grandchildren, and they would Facetime while the grandkids baked and decorated the cookies. She also planned to have gifts and a holiday meal delivered to her daughter’s home. And her daughter adapted by planning a holiday drive-by so her mother could see her family briefly and from a safe distance. 

In true Darwinian fashion, “it is not the strongest of the species that survives, nor the most intelligent; it is the one most adaptable to change.” 

Cancer patients adapt so that they may yet hold on to a slice of what brings their lives joy and meaning. It is a lesson everyone can learn. 

Yasmin Asvat, PhD is a licensed clinical psychologist at Rush University Medical Center with eight years of experience providing mental health services to chronically ill patients, primarily cancer patients. She is a Public Voices Fellow through The OpEd Project.

AstraZeneca’s Tezepelumab Fails to Meet its Primary Endpoint in P-III SOURCE Study for Asthma

Shots:

  • The P-III SOURCE study involves assessing Tezepelumab (210mg, q4w) vs PBO in 150 adult patients as add-on therapy with patients maintained on their currently prescribed ICS + LABA, with/ out other asthma controller therapy for 48wks.
  • The trial did not meet its 1EPs i.e., reduction in the daily OCS dose, without loss of asthma control, the safety profile was consistent with previous trials. The therapy’s other efficacy parameters were similar to those observed in previous trials, including the P-III NAVIGATOR trial
  • Tezepelumab is a human mAb targeting TSLP, being developed in collaboration with AstraZeneca and Amgen

Click here ­to­ read full press release/ article | Ref: AstraZeneca  | Image: Physicians Practice

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2020 in review: COVID-19 and patient centric clinical trials

The trials and tribulations of 2020 have brought the vital role of research, pharma, and biotech into sharp focus. But how has the push to develop treatments and vaccines for SARS-CoV-2 affected the industry’s commitment to patient centricity?

This time last year, researchers and industry players were working hard to embed patient centricity and engagement into their everyday work.

Then COVID-19 hit, and organisations big and small were forced to pivot operations to tackle the real and present danger of the pandemic. Since then, we have seen some mind-boggling scientific achievements, with innovations in vaccine development being just such one example.

But how has this global push for SARS-CoV-2 vaccines and treatments affected the research community’s commitment to patient centricity? We take a look back over the last 12 months to find out.

Green shoots of engagement

At the start of the year, the industry was busy incorporating the patient voice into trials in a bid to overcome the recruitment and retention problem – the figures showed that fewer than 5% of all those eligible to take part in research signed up, and the global average dropout rate was around 30%.

“The pandemic also accelerated the adoption of many patient centric study practices. Remote monitoring, for example, went from a nice to have to a necessity overnight.”

While the research community was aware of the benefits of patient centric trials, however, they were still unclear on how to put that into practice, said patient engagement agency, Couch, back in February.

Said the team: “In the Annual Patient Centricity Benchmark Survey, when asked about training or preparing people to behave in patient-focused ways, over half of employees from biopharmaceutical and medical device companies said: ‘We are actively looking at how to teach this to our people’.

“Only 22% selected: ‘We know exactly what and how to teach this to our people’.”

That said, the community was going in the right direction. In March, we reported on how researchers were increasingly using methods such as patient-reported outcomes, remote reporting, and lay summaries to boost engagement.

Widespread interest

But then the pandemic piqued people’s interest in medical research at previously unimaginable scale.

The RECOVERY Trial, a multi-arm RCT studying the efficacy of several repurposed treatments in COVID-19, recruited a staggering 2,000-plus people across 16 NHS sites in little more than three weeks. At the same time, thousands more signed up to report their health status to the King’s College London COVID Symptom tracker app every day.

Speaking to pharmaphorum in May, Dr Sheuli Porkess, Executive Director of Research, Medical and Innovation at the Association of the British Pharmaceutical Industry (ABPI) said, if harnessed correctly, such changes could benefit research efforts for years to come.

“The studies are being covered on the news and that’s great for letting people know how they can get involved in research right now. What’s more, that ongoing exposure to discussions around trials and what people do when they are in a trial will, in the future, help people to say ‘yes, I want to be involved’.  I think we really need to look into what it was that enabled people to sign up so quickly.”

The pandemic also accelerated the adoption of many patient centric study practices. Remote monitoring, for example, went from a nice to have to a necessity overnight. In doing so, it proved it could provide robust data at the same time as reducing participant burden.

Casualty of speed?

But while the pandemic certainly created a collaborative discovery atmosphere, it also introduced an element of intense urgency – and this has, arguably, had a detrimental effect on engagement in research.

In November, pharmaphorum reported from the Pioneering Partnerships conference, organised by the ABPI, the National Institute for Health Research (NIHR), and the Association of Medical Research Charities (AMRC). We asked if rapid progress and patient engagement were mutually exclusive.

NIHR director, Jeremy Taylor, said: “One of the consequences of the system commissioning lots of urgent COVID-19 research was that, to a certain extent, patient and public involvement got bypassed. For various reasons it was too difficult or too time consuming to do when everybody was in a frightful rush.

“Patient and public involvement turned out to be less embedded than we thought, so I think COVID has been a bit of a shock to the system. It’s made us think that maybe we have been a little too complacent.”

Lessons to learn

The last 12 months have been something of a whirlwind for everyone, but the healthcare and research community have been in the eye of the storm.

In 2021, the research community can build on the widescale adoption of remote monitoring and huge increases in study recruitment rates, but it must also put what it has learned about “doing” engagement at speed and at scale into practice.

  • Want to read more about how COVID-19 has impacted in patient centricity? Check out the latest edition of pharmaphorum’s Deep Dive magazine, which is dedicated to the topic.

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Finding agility in unprecedented times

Nicole Farmer, general manager, UK & Ireland at Sanofi Genzyme, tells us how the company aimed for agile adaptation during COVID-19, and how these lessons are driving patient centricity going forward.

2020 has been a watershed year for every company in the industry – and for Nicole Farmer, general manager for the UK & Ireland at Sanofi Genzyme, that meant moving into a pharma leadership role at a time when things couldn’t be more uncertain.

Nevertheless, Farmer – who was appointed in May at the height of COVID-19 – says that her main goals when coming into the company hadn’t changed due to the pandemic.

These goals were: creating an environment where people have the courage to be proactive and not wait to be told what to do; pushing beyond current limitations; and keeping customers and patients at the heart of why Sanofi does what it does.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Health inequalities: A societal challenge that needs a cross-sector solution

COVID-19 has laid the UK’s health inequalities bare and created an imperative for the NHS to develop a step-change in how it cares for diverse and marginalised communities.

Health inequality is the “greatest societal challenge of our time”, according to a briefing document calling for a system-wide solution.

The report was published by the Association of the British Pharmaceutical Industry (ABPI) and the NHS Confederation as part of the NHS Reset project, which has been working to set a post-pandemic roadmap for health and social care.

Said the authors: “The pandemic has thrown into sharp focus the issue of health inequalities in the UK and exposed the consequences of a long-standing failure to tackle this deep-rooted and multi-faceted problem.

“The first wave had disproportionate health, economic and social impacts on people in lower socioeconomic groups and those with black, Asian and minority ethnic backgrounds.”

It highlights the need for a clear strategy, pointing to a recent survey that found nine out of ten NHS Confederation members agreed that “the time to act is now”. Just 41%, however, said they had the tools, knowledge, and support to deliver the necessary change.

Societal challenges

In November, the two organisations held a roundtable with more than 20 NHS and industry leaders, who discussed “tangible approaches” to tackling “the greatest societal challenge of our age”.

Working under Chatham House rules, they agreed that reducing avoidable and unfair differences in health outcomes would require action in four key areas: data quality, community engagement, access to services, and risk identification and stratification.

“Participants heard that the health system is capturing patient ethnicity data around 65 per cent of the time. More complete – and more comprehensive – data is needed to obtain a full picture of how ethnicity affects health outcomes,” said the report.

The meeting agreed primary care had the biggest potential to rapidly increase the volume of captured ethnicity data, using opportunities such as vaccination programmes and changes to practice registration details.

It also discussed the need to involve and listen to local communities, and the possibility of funding community champions employed through voluntary and community sector organisations.

Equitable access

Much has been published on the increase of digital health during the pandemic, but, as the report stresses, digital exclusion and digital poverty can compound health inequalities.

According to NHS England, people from excluded groups or living in deprived areas often lack the skills, ability and means to get online. This can block access to initiatives such as virtual consultations, which have been soaring in popularity.

Restoring services in the aftermath of the pandemic was also felt to pose particular challenges relating to equity of access.

“Concerns were raised that providers simply working through waiting lists using standard processes, without viewing them through the lens of inequalities, could actively exacerbate the problem.

“A possible way to resolve this unintended outcome would be to use a clinical prioritisation process to identify who will benefit most from intervention,” said the authors.

“The first wave had disproportionate health, economic and social impacts on people in lower socioeconomic groups and those with black, Asian and minority ethnic backgrounds”

Wider determinants of health

Healthcare systems need to be able to proactively identify populations at high risk by virtue of their socioeconomic status and ethnicity, as well as their clinical and co-morbidity data.

The impact of poverty in particular was discussed as a factor requiring much more consideration in terms of risk stratification.

“For example, in some areas, people continued to work during lockdown despite being in high-risk COVID-19 localities, due to intense financial pressures and economic insecurity.”

As set out in the Long-Term Plan, integrated care systems (ICS), which bring health, social care, the voluntary sector, and local authorities together, are playing an increasing role in the NHS.

“ICS and their constituent place-based partnerships were viewed as potentially playing a role by convening organisations outside healthcare, such as housing associations, to ensure they are enabled to play a role in discussions about the wider determinants of health.”

The authors agreed that this approach presented a “particular opportunity to make system-wide improvements to determinants that have traditionally been out of scope for the NHS, such as air quality”.

However, it was also felt that this should be complemented by neighbourhood-level cross-sector multidisciplinary teams, able to provide health and care interventions, as well as connecting individuals to the wider community.

Call to action

Building these recommendations into the fabric of society, even with cross-sector involvement, will certainly be a challenge.

But, said the report, the NHS Confederation and the ABPI are “committed to a long-term partnership to help build the right tools, techniques, and capabilities to meaningfully address health inequalities”.

That includes developing a suite of datasets and methodologies to improve risk stratification, as well as recommendations on how to improve diversity, inclusion, and patient experience in research.

“Both organisations are eager for as many NHS and industry colleagues as possible to become part of this process, contributing expertise to co-create solutions to the greatest societal challenge of our age,” the authors concluded.

To get involved, contact Nasima Hossain on [email protected] or Su Jones at [email protected]

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Amgen’s Riabni (biosimilar, rituximab) Receives the US FDA’s Approval for Multiple Diseases

Shots:

  • The approval is based on trial assessing Riabni (375 mg/m2, IV) vs Rituxan once weekly for 4wks. followed by dosing @12wks. & 20wks. in 256 patients in a ratio (1:1) with grade 1, 2, or 3a follicular B-cell NHL & low tumor burden
  • The WAC of Riabni will be 23.7% lower than the Rituxan in the US and will be available at a WAC of $716.80/ 100mg and $3,584.00/ 500mg single-dose vial
  • Riabni is a biosimilar to Rituxan, approved for the treatment of NHL, CLL, GPA, MPA and ill be made available in the US in Jan 2021

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

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Patient experiences are evolving. How can pharma keep up?

By listening to the people who are navigating this new landscape everyday.

Integrating personal experiences and patient-reported data, Health Union recently hosted a one-day virtual event entitled
The COVID-19 Effect: How Pharma Can Adapt to the Evolving Patient Experience, featuring real-time conversations between people living with chronic conditions and industry experts. The sessions focused on the unique experiences of each of the advocates interviewed throughout the coronavirus pandemic, and covered topics such as telehealth, social distancing, isolation and more. 

Access any or all of the session recordings on demand, and hear from patient advocates living with migraine, MS, psoriasis, and lung cancer; plus a telehealth-focused roundtable featuring advocates living with Parkinson’s disease and IBD.

Visit health-union.com to access the recordings on-demand.

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Patient journeys in the era of COVID-19

OPEN Health’s Richard Jones and Sumira Riaz assess the pandemic’s implications for understanding patients’ experiences and how the pharma industry can support them

It’s impossible not to view healthcare in 2020 through the lens of the current global pandemic and COVID-19 is certainly set to cast a long shadow over patient needs and engagement.

Even when the virus is tamed, and recent advances with vaccines are grounds for much optimism, the huge societal and healthcare changes that we’ve seen take place this year will undoubtedly have left their mark in all sorts of altered, and new, approaches.

The way in which patients make their way through the healthcare system has shifted enormously, bringing disruption to the patient experience. COVID-19 has driven massive uptake of different types of digital support and interventions, most notably with telehealth.

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Novartis’s Entresto Receives the US FDA’s Advisory Committee Recommendation to Treat Patients with HFpEF

Shots:

  • The US FDA’s CRDAC voted 12 to 1 supporting the use of Entresto (sacubitril/valsartan) in the treatment of patients with HFpEF
  • The decision was based on efficacy & safety analyses, including findings from a pre-specified subgroup analysis of PARAGON-HF (P-III study in HFpEF) and additional evidence from PARAMOUNT (P-II trial in HFpEF), as well as PARADIGM-HF (P-III trial in HFrEF)
  • Entresto (sacubitril/valsartan) is a prescription medicine used to reduce the risk of CV death & heart failure hospitalization in people with long-lasting heart failure

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

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How pharma needs to change for the era of digital health

Experts from Healthware Group explore how companies can rebuild their approach to digital from the ground up for the benefit of patients.

Digital is not just a nice-to-have for pharma companies – it’s a necessity for ensuring that patients have the best possible outcomes in modern healthcare systems.

With digital tools and techniques being able to improve almost every aspect of a pharma company’s business, from R&D to sales, there’s no excuse for not implementing digital transformation at every level – and that’s not even mentioning new opportunities for life science firms to produce digital products that can complement or even replace traditional medicines.

Roberto Ascione, founder and CEO of Healthware, says that this landscape means that companies like his need to expand to be “communicators, connectors and builders” all at once – and this is a philosophy he has built up within Healthware over the last two and a half decades.

 

• Read the full article in pharmaphorum’s Deep Dive digital magazine

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Proposed HIPAA changes generally favorable, though new burdens may arise, experts say

HIPAA

The HHS has proposed changes to the HIPAA Privacy Rule — the biggest in seven years, a healthcare lawyer said. But while the changes aim to improve information sharing, they could also bring about challenges for providers and payers.

Pandemic has little impact on pharma’s reputation, says report

The pandemic has had very little positive impact on the public’s perceptions of pharma although the industry has fared much better than politicians, according to a report.

According to the report from Takeda UK, only 17% of respondents said their impression of pharmaceutical companies had improved, based on how the industry had reacted to the coronavirus pandemic over the previous six months.

Findings were based on the report entitled Pharma: Repurposed? where Takeda commissioned Ipsos MORI to conduct online interviews among adults aged 16-75 from the UK.

In all, 1,104 interviews were conducted in October 2020.

Aside from the 17% who said their impression of pharma companies had improved during the pandemic, 54% said their impression had stayed the same and 16% said their impression had got worse.

Less than half of respondents trust the pharma industry to act in the best interest of society, while 82% of respondents trust healthcare professionals to act in society’s best interest.

The pandemic has had a positive impact on UK adults’ perceptions of healthcare professionals, with 44% of respondents saying their impression had improved.

In contrast, UK adults’ impression of politicians is markedly worse, with 51% of respondents saying their opinion of government ministers had worsened over the last six months.

When asked to give an opinion on a range of organisations and roles, just 38% of those interviewed had a favourable opinion of pharma companies.

Over a third of respondents had neither a favourable or unfavourable view of the pharma sector, suggesting the industry needed to engage more with the public on its role and activities.

Healthcare professionals were highly regarded by 74% of those interviewed as well as healthcare charities and scientists within the pharma industry (53%) were well regarded.

Total unfavourable views of government ministers stood at 55% and other politicians at 54%.

Jon Neal, managing director for UK and Ireland at Takeda, said: “The pharmaceutical industry has faced several reputational challenges over the years. However, perhaps now more than ever, the development of new treatments relies on cross-sector collaborations and public trust and willingness to engage with the sector.

“Therefore, we need to address perceptions of the industry to improve the future of healthcare and ultimately save lives.

“We hope this report will lead to further discussion about how the industry can convey a stronger purpose to build trust with both patients and other healthcare organisations.”

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Roche Launches Cobas PIK3CA Mutation Test for Patients with Advanced or Metastatic Breast Cancer

Shots:

  • Roche launches the cobas PIK3CA mutation test for patients with advanced/ m-BC in countries accepting the CE mark
  • The IVT test is a real-time PCR test for the qualitative detection & identification of 17 mutations in exons 2, 5, 8, 10 & 21 in the gene encoding the catalytic subunit of PIK3CA in DNA isolated from FFPET and is intended to identify patients with m-BC harboring mutations
  • This test reports automated results, with flexible throughput to process 30 samples/ run on the widely available cobas z 480 analyzers. The test can detect 17 mutations in the PIK3CA gene and can help clinicians to identify patients who may benefit from PI3K targeted therapy

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

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Deep Dive: Patients and Partnerships

As this issue’s title suggests, patient centricity is about more than just talking points and marketing strategies – it requires companies to truly listen to, engage with and partner with the patients they serve, and in putting this month’s Deep Dive together it’s been great to see that so many pharma firms are now taking this to heart.

Not only that, but we are seeing patient insights being considered at every stage of a product’s lifecycle, from product design to clinical trials and even post-marketing patient services. In this issue you can read expert viewpoints from across the industry on how pharma can optimise its patient engagement at all these stages.

Finding agility in unprecedented times
Nicole Farmer, general manager, UK & Ireland at Sanofi Genzyme, tells us how the company aimed for agile adaptation during COVID, and how these lessons are driving patient centricity going forward

‘No going back’ for clinical trials after COVID
Trial sites have adapted swiftly to the restrictions of COVID-19. The next step is ensuring the industry does not regress to old ways of working once the pandemic is over

True patient-focused research through decentralised and hybrid trials
How can pharma improve the patient-centricity of its trials during COVID-19 and beyond? Experts from across the sector give their thoughts on the key approaches and technologies that are driving patient engagement forward

How patient insights are changing trial solutions
Patients have been asking for patient-centric trial solutions for years – the industry just hasn’t been listening. Medidata’s Anthony Costello tells us what insights pharma has been missing out on and how they can be harnessed to build better solutions

Boosting the impact of patient services
New research from Accenture has revealed that adoption of patient support services hasn’t improved since 2015 despite increasing pharma investment. Jennifer Spada tells us how companies can boost awareness of their programmes to improve outcomes

3 practical steps for improving patient support
Research Partnership’s Emilie Braund and Harrison Gaiger dig down into the top insights pharma companies can harness to make their patient support programmes as powerful as possible

Enabling new models of care: pursuing pharma’s partnership potential
There is a huge, ongoing shift in how health and wellness is approached in the UK, and the changes will have important implications for NHS-industry partnerships

A collaborative approach to greater diversity in clinical trials
Despite new policies, trial data has remained largely based on healthier Caucasian subjects. Evidera’s Rhonda Henry explores ways to change this

Patient journeys in the era of COVID-19
OPEN Health’s Richard Jones and Sumira Riaz assess the pandemic’s implications for understanding patients’ experiences and how the pharma industry can support them

Patient partnerships: putting relevance into relationships
Emma Sutcliffe explains how to build partnerships that are not defined by the volume of the interactions but instead by the value that each interaction brings to all stakeholders

COVID-19 – Delivering a pandemic of change to digital medical education
Medscape’s Adrian Duncan explores findings from an FDA trial looking at the impact of digital medical education on antibiotic prescribing

How pharma needs to change for the era of digital health
Experts from Healthware Group explore how companies can rebuild their approach to digital from the ground up for the benefit of patients

 

• Read the latest issue Deep Dive: Patients and Partnerships 2020 in full

Patients and Partnershipspharmaphorum’s digital magazine Deep Dive provides objective, issue-driven views, analysis, high-level interviews and unique research for pharmaceutical companies, biotech firms and the wider healthcare sector.

In 2020 Deep Dive will have special focuses on disruptive technologies in pharma, R&D innovation, market access and commercialisation, oncology, sales & marketing innovation, digital health and patient engagement. Subscribe to future issues of Deep Dive.

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CMS proposed rule requires payers to streamline prior authorizations

The rule would require payers in the Medicaid, CHIP and QHP programs to build and maintain application programing interfaces to improve data exchange and the prior authorization process. But the rule does not include Medicare Advantage plans, which the American Hospital Association called “disappointing.”

Roche Launches Elecsys SARS-CoV-2 Antigen Test to Support High-Volume COVID-19 Testing

Shots:

  • Roche has launched Elecsys SARS-CoV-2 Ag test as an aid in the diagnosis of SARS-CoV-2 infections, in the markets accepting the CE Mark. Additionally, Roche has also filed a EUA to the US FDA
  • The test showed 94.5% sensitivity across 200 PCR confirmed symptomatic individuals & 99.9% specificity across 2747 PCR negative symptomatic & screening individuals in clinical studies. The company plans to ramp up production to have a double-digit million number of tests/ month, in early 2021
  • The test is an immunoassay intended for the qualitative detection of SARS-CoV-2 present in the respiratory tract including nasopharynx & oropharynx. The test is the latest addition to Roche’s COVID-19 portfolio

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

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Does a lack of diversity in clinical trials reflect a lack of diversity among researchers?

Embedding diversity into clinical research does not start and end at trial recruitment – the community needs processes and institutions that reflect society and provide representation.

Greater representation among study designers, research teams, and funding committees would lead to greater diversity among research participants and help to tackle health inequalities.

Speakers at the virtual National Institute for Health Research (NIHR) Academy Members’ Conference said the inclusivity conversation had moved from “why” to “how”, and that it was time to look at wider structures.

Professor Sandra Eldridge, equality, diversity, and inclusion lead for Bart’s and the London School of Medicine and Dentistry, said:  “I would suggest that the reasons our trials are not as inclusive as researchers would like are related to wider societal issues.

“Clinical trials are part of research, and research is part of society. While we may be able to improve things through trial design, we certainly can’t solve all the issues just by doing that.”

The barriers are multiple, she went on, citing a lack of trust in experts, institutions, and authority among some groups, which can be seen in current concerns over COVID-19 vaccines, and in the research process as a whole.

“The literature shows that people who feel this lack of trust acutely use words like ‘discrimination’, ‘deception’, and ‘exploitation’. They also talk about the idea of researchers being parachuted in to do research, without really involving or getting to know the group,” said Prof Eldridge, adding that there were a number of reasons for this mistrust.

“Clinical trials are part of research, and research is part of society. While we may be able to improve things through trial design, we certainly can’t solve all the issues just by doing that.”

“The first is historic racism in research, such as the infamous Tuskegee experiments, and the racism that people experience within the health service and wider society.

“Also, I think there is a lack of knowledge about research processes and safeguards among the general population and, coupled with that, a suspicion of large rich organisations such as drug companies.” Importantly, she went on, there is a “lack of visible role models within established research”.

Equality versus equity

Representation among research teams and funding committees is crucial to building processes, institutions, and interventions that work for the whole of society.

Achieving this means actively seeking out people in previously marginalised groups and making sure their voices are heard, said Professor Lucy Chappell, NIHR research professor in obstetrics at King’s College London.

Prof Chappell said: “We talk a lot about equality and that’s important. But perhaps even more important is equity – how are we genuinely ensuring everyone has an equitable opportunity to apply for research, lead research, and be on decision making bodies?

“If we put out adverts for funding committee members, for example, and are surprised when we don’t get a balanced representation of applicants, we need to ask ourselves what we are doing within that process that systematically prevents or discourages certain groups from applying.”

Gender and ethnicity are not the only considerations, she went on, adding that cognitive diversity, or the meeting of multiple – even conflicting – representative perspectives, was also crucially important.

“If I have a lot of intelligent people together in a room but they all have the same view, I’m not going to get diversity. It’s not about having clones or a tick box exercise, it’s about having diversity in the way people approach problems.”

In the same vein, research institutions should be looking for passion, ahead of the usual accomplishments and awards in their staff, said Prof Jill Manthorpe, director of the NIHR Health and Social Care Workforce Research Unit.

“Passion is the prod that makes people say: ‘but what about patient views, what about the views of the people we’re not hearing?’ Passion enables people, and spurs them to go the extra mile,” she said, using examples of community work and volunteering.

“It’s about long-term relationships, not just parachuting in for a project. It’s about what we as researchers can bring to communities, and how enriching that is for us over the years.”

The 7%

Policies and tools to help organisations build ethnic diversity into processes, such as Trial Forge’s INCLUDE Ethnicity Framework, have been launched in recent years. What is less easy to pin down, though, is socioeconomic background, said Prof Manthorpe.

She pointed to the “7% problem”, referring to the 7% of people attending fee-paying schools who also hold most of the UK’s top jobs.

“How do we find and encourage people who didn’t have those educational opportunities? How do we differentiate between people who have had a very privileged education, from those who haven’t had the ability to build up robust networks early on in life?”

Older methods of establishing background, such as the age at which someone left school or whether they had an outdoor toilet growing up, are increasingly outdated and irrelevant, she said.

“We don’t know the answer as to how do you work out people’s socioeconomic status in a respectful and easily accomplished way.

“But I hope that we will get more people engaging with this, helping to answer some of those questions on how we reach people who are perhaps the hardest to hear,” she said, adding that all researchers wanted to “extend the ladder” to those who follow them.

Representation

There is lots of discussion about making research more diverse, and various initiatives have sought to alleviate the problem that can lead to devastating health inequalities in recent years.

But by making processes and infrastructure more inclusive, researchers will no longer need to worry about reaching underserved communities to make sure their interests are represented – because they will be part of them.

About the author

Amanda Barrell is a health and medical education journalist, editor and copywriter. She has worked on projects for pharma, charities and agencies, and has written extensively for patients, healthcare professionals and the general public.

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Pfizer and BioNTech ‘s BNT162b2 Receive Health Canada Authorization to Combat COVID-19

Shots:

  • Health Canada has granted authorization under interim order for the emergency use of BNT162b2. The distribution of the vaccine in Canada will be prioritized according to the populations identified in guidance from the NACI
  • BioNTech will hold the regulatory approval in Canada while Pfizer will have the commercialization rights. The approval is based on data that was filed through the rolling submission regulatory pathway and data from the P-l/ll clinical trial that began recruiting in Jul’2020 & enrolled ~44,000 patients across ~150 sites in multiple countries
  • The companies will supply ~20M doses of vaccine to Canada through 2021

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

The post Pfizer and BioNTech ‘s BNT162b2 Receive Health Canada Authorization to Combat COVID-19 first appeared on PharmaShots.

What HCPs think about COVID-19’s impact on cancer

COVID-19’s knock-on effect on cancer patients is of concern by HCPs, with delayed or decreased diagnosis seen as a key worry. CREATION.co’s Lara Meyer explores why HCPs need more support.

The COVID-19 pandemic has been mainstream headline news throughout 2020 and continues to have a profound impact on all our lives. A key concern that has emerged from the crisis is how COVID-19 will affect other areas of healthcare, particularly cancer diagnosis and treatment.

To understand what healthcare professionals (HCPs) felt about this topic, CREATION.co investigated public social media, in collaboration with Sermo’s HCP survey platform, Sermo RealTime, which provides real time access to physician insights.

We looked at conversations that HCPs were having online to provide a full picture of their concerns and needs. By designing fast surveys based on insights from the online conversation we were able to get a powerful depth of insight. For the survey we recruited oncologists from the United States, United Kingdom and Spain.

HCPs concern for decreased cancer diagnosis

During the COVID-19 pandemic, delayed or decreased cancer diagnosis was highlighted by HCPs as a key concern. Prominent industry figures, such as oncologist and ex-director of the WHO Cancer Programme Professor Karol Sikora, shared news articles and utilised their networks to raise awareness of decreased cancer diagnosis.

However, only a fraction of HCPs explained why they experienced a decrease. Knowing the “why” can help to address the specific challenges that hospitals are facing to ensure patients are receiving the care they need.

Using Sermo RealTime, we asked physicians to rank why they believe there has been a decrease in diagnosis at their hospital or practice. We discovered that cancellation of appointments by hospitals was perceived to have caused the most impact. The reason ranked as the next factor, was hospital staff being diverted to COVID-19 efforts. Understanding these reasons could help with resource allocation and impact assessments.

On social media, HCPs chose to encourage their peers to continue supporting their oncology patients during the pandemic. Again, key online influencers, such as Dr Tatiana Prowell – a well-known medical oncologist specialising in breast cancer, led a call to prioritise patients and raise awareness of decreased diagnoses. We have seen many HCPs supporting their peers online throughout the pandemic.

Uncertainty around cancer treatment

A significant part of the HCP conversation online discussed treatment of oncology patients. Physicians shared their concerns about delaying or changing treatment approaches and the impact this would have in the long term. When surveying physicians using Sermo RealTime, 79% of HCPs shared that they had delayed their patients’ treatment, while 52% of HCPs opted to change their patients’ treatment approach either by switching the drug their patient is on, or changing the administration timing or dose.

For the pharmaceutical industry, this knowledge could help teams in their communication plans to support HCPs with updated information or guidelines about treatments.

We regularly see HCPs share treatment guidelines on social media to provide support when there is confusion around new or existing treatment approaches, often creating their own guidelines when none exist.

And in our survey of physicians, 58% of respondents shared that regulatory guidelines have been their go-to source for information and advice for treatment during the COVID-19 pandemic, alongside consulting their peers. However, even HCPs’ go-to source did not always provide as much support as they would have liked, with some HCPs sharing that they are still unsure of the correct treatment for patients during the COVID-19 pandemic or for cancer patients that have COVID-19.

After surveying HCPs about how confident they were about the information and advice they have received about continuing treatment for their COVID-19 positive or negative cancer patients, 70% of physicians shared that they were “somewhat confident but consulted with their peers”, showing how important peer support is during this time.

HCPs look to the future

As COVID-19 continues to affect countries around the world, HCPs are concerned about the short-term and long-term implications the pandemic will have on patient diagnosis and care. Despite having to respond reactively day-to-day, and the focus on the here and now, the future is still on HCPs’ minds.

Dr Stephanie Graff, a breast cancer oncologist, shared her concerns about “what this might mean long term—stage at diagnosis for example”, and how to bring patients safely back to care.

When physicians in our survey were asked what some of the key concerns are for them going forward, they shared the backlog of patient cases that will need manual review and further investigations, switching to less effective or immunosuppressive treatments, and patients’ hesitancy or distress preventing them getting treatment. Others also shared the same concern as Dr Graff, that cancer patients present at a later stage because of backlogs and hesitancy to come to hospitals having a much larger impact on the treatment approach for these patients.

Across both open and closed online networks HCPs are concerned about the future of patient care.

The online HCP conversation continues at a steady rate each day with oncologists, nurses and specialists continuing to share their concerns and needs online with peers. Throughout the year, HCPs continue to seek the answers they are looking for and share resources online.

These concerns all present opportunities for pharmaceutical companies, hospitals, advocacy groups and medical organisations to support HCPs in very specific areas. Listening to the voices of HCPs online, especially as they are more active during this time, can help uncover key areas for engagement and support.

About the author

Lara MeyerAt CREATION.co, Lara supports clients in scoping and delivering projects. Her pharmaceutical experience includes laboratory research, as well as in marketing and strategy and she recently completed an MSc in Global Management from the London School of Economics. Working with CREATION.co she leads a team of insight analysts compiling reports.

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Sharing experiences to level the healthcare playing field

Amanda Barrell speaks to Alfred Samuels on his experience of prostate cancer and diversifying clinical trials to include the Black, Asian and minority ethnic (BAME) community.

Alfred Samuels’ stage 4 metastatic prostate cancer brought his 30-year career, which had seen him travel the world with the likes of Beyoncé, Oasis, and Bob Dylan, to a shuddering halt.

Now, through his books and advocacy work, he is determined to help others prepare themselves for the “horrendous” cancer journey and help level the healthcare playing field for the BAME community.

In 2011, Alfred was a fit and healthy 54-year-old providing security for music’s A-listers on global tours. But then he started experiencing an excruciating pain which radiated from his lower back and into his leg.

“It’s really important for men, their partners and families to understand that there is much more to this disease than you think”

At A&E, he was told it was “probably sciatica”, but was advised to ask his GP to refer him for an MRI scan just in case.

“They messed me around for three months before agreeing to a scan. Then, two weeks after the MRI, the diagnosis came in. It was stage 4 metastatic prostate cancer and it had spread to six locations in my body,” said Alfred.

“I went for some tests and the prognosis came back – I was given six months.”

While he was fortunate enough to secure a place on the STAMPEDE clinical trial, during which he received abiraterone and hormone injections, the side effects were “not pleasant”.

“I was hospitalised on a couple of occasions because the pain was horrendous, and they hadn’t got the pain relief medications sorted out properly. I collapsed due to compression of the lower back. I had brain fog and fatigue. I went from someone who was able to run and sprint quite easily to being like a 90-year-old man. It was not a pleasant journey.

“It was untimely, unwanted, and as far as I’m concerned, undeserved,” he said. “My career was over, you’ve got all these mental issues, and then there’s the financial toxicity of it all.”

At the time of presentation, Alfred’s prostate-specific antigen (PSA) was more than 500 ng/ml, compared to the “normal” level for his age at the time, between 2 ng/ml and 4 ng/ml.

However, within months his PSA had dropped to less than 0.01 ng/ml, so Alfred stuck with the punishing protocol.

“One year became two years, two years became three years, and it’s probably then that I started to believe in myself, believe that I could beat it,” said Alfred.

Publishing the journey

Since then, he has published two books based on his experiences and the diaries he has kept of his journey. Invincibility in the Face of Prostate Cancer: Coming Out the Other Side charts his time in the clinical trial, and Motivated to Inspire focuses on the post-treatment period.

“I think it’s really important for men, their partners and families to understand that there is much more to this disease than you think.  You really have to work as a team to get through it,” said Alfred, adding that his wife, Grace, had been invaluable support throughout his journey.

“Interacting with the medical fraternity needs to be explained to people so they get a better understanding of what they’re going to go through. It’s a journey full of pitfalls and pain, and you’ve got to be able to condition yourself for that.”

Focus on inclusivity

Alfred is an ambassador for Cancer Research UK, which funded the STAMPEDE trial, and works with groups including The Urology Foundation and Orchid. He also sits on a patient consultation panel for pharmaceutical company Parexel.

Much of the work he does centres around raising awareness of the patient experience, and the issues of diversity and inclusion are always top of his mind.

“Black people are not getting the best deal when it comes to healthcare, and where prostate cancer is concerned, black men are disproportionately affected,” said Alfred, explaining that Caucasian males had a one in eight chance of developing the condition, while the figure is one in four among black men.

A big part of the problem, he went on, was a lack of BAME representation in clinical trials, and he urged anyone from underserved communities to take part in research if they could.

“There are medications that are just not being tested on people from the BAME community. We will react differently to Caucasian people, so without more people from BAME backgrounds getting involved, we can never be sure that the information we are getting is correct,” he said.

Asked how the industry could be more inclusive, he said it was about trust and understanding.

“If you don’t take the patient’s voice into consideration, you have no understanding of what’s going on for them out there and you end up in a sticky situation.

“I think pharmaceutical companies are cottoning on to that now, but there’s still a lot of mistrust. It is about building relationships and getting to know the communities you are working with.

“Are you speaking in a language that they understand? Are the concepts broken down so that they are digestible?

“It’s not rocket science,” said Alfred, adding that the industry is now starting to understand the importance of such issues, and put plans in place to address them.


Patient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 2 million patients and caregivers worldwide.

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Survey Finds Telehealth Is Convenient Alternative, Will Likely Continue Beyond the Pandemic

A new Health Union survey reveals that people with chronic conditions feel that telehealth, despite its convenience and increased use throughout the COVID-19 pandemic, is generally less preferable than in-person visits, but can still serve as an occasionally suitable alternative. The survey is the fifth in Health Union’s ongoing COVID-19 Consumer Attitudes and Health Behaviors Survey series that captures “snapshots in time” that track the perspectives and health behaviors of people with chronic conditions throughout the pandemic.

Telehealth use continues to increase during the pandemic for people living with chronic health conditions. Nearly three-fourths of respondents of this wave 5 survey said they have had at least one telehealth appointment, up from 63% from the wave 4 survey, which fielded in July.

Fortunately, two-thirds of respondents who have had telehealth appointments considered their experiences to be positive, with convenience being a primary reason. And 44% of all respondents said they were “extremely likely” to consider using telehealth after the pandemic is under control. This number was even higher for people living with autoimmune conditions, such as ankylosing spondylitis (61%), Crohn’s disease (61%) and plaque psoriasis (56%).

However, the survey findings reveal that increased use doesn’t necessarily translate into a clear preference for telehealth.

Read the full article on health-union.com.

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PRISYM 360 Achieves SAP® Certification as Integrated with Cloud Solutions from SAP

PRISYM 360 Achieves SAP® Certification as Integrated with Cloud Solutions from SAP

The latest version of the PRISYM 360 intelligent data import connector now has SAP-certified integration using SAP Cloud Platform Integration Suite

Wokingham, United Kingdom — 7 December 2020 — PRISYM ID, a leading provider of regulated content and label management solutions for the life sciences sector, announced today that PRISYM 360 version 1.10 has achieved SAP® certification using SAP Cloud Platform Integration Suite. The integration helps organizations to transfer labeling data from SAP technologies into the PRISYM 360 label management platform, easing the process of producing compliant labels and booklets for clinical trials, medical devices and other life sciences organisations.

The new interface supports cloud-based SAP applications as well as on-premise components. It is available as a standard product for PRISYM 360 deployments and its SAP-certified integration with cloud solutions from SAP offers businesses assurance that they are safely integrating the two systems in an approved way.

PRISYM 360 and SAP technologies communicate through a web service using standard SAP components with no intermediate stages.  This helps to eliminate the need for manual data transfers and reduce the risk of human error, providing certainty while streamlining the process.

The SAP® Integration and Certification Center (SAP ICC) has certified that PRISYM 360 version 1.10 leverages SAP Cloud Platform Integration Suite for integration to SAP.

“PRISYM ID has achieved SAP-certified integration with SAP solutions before, helping to ensure that accurate data is used automatically for labeling purposes,” said Chris Lentz, VP SaaS and SAP Business Development at PRISYM ID. “This latest intelligent data import connector for PRISYM 360 delivers enhanced capabilities, enabling customers to transfer their clinical or medical device labeling data into PRISYM 360 using the latest cloud and on-premise components from SAP and eliminating the need for manual transfer of data. This SAP certification of PRISYM 360 gives users continued peace of mind that their systems are integrated using the latest protocols and following recommended procedures.”

– Ends –

About PRISYM ID
PRISYM ID provides regulated content and label management solutions designed specifically for life science companies and medical device manufacturers, improving patient safety and health outcomes whilst ensuring regulatory compliance. PRISYM ID’s unique products and technologies are used to assure compliance globally, for 10 million+ medical device, clinical trial and pharmaceutical products annually. www.prisymid.com

Any statements in this release that are not historical facts are forward-looking statements as defined in the U.S. Private Securities Litigation Reform Act of 1995. All forward-looking statements are subject to various risks and uncertainties described in SAP’s filings with the U.S. Securities and Exchange Commission (SEC), including its most recent annual report on Form 20-F, that could cause actual results to differ materially from expectations. SAP cautions readers not to place undue reliance on these forward-looking statements which SAP has no obligation to update and which speak only as of their dates.

SAP and other SAP products and services mentioned herein as well as their respective logos are trademarks or registered trademarks of SAP SE (or an SAP affiliate company) in Germany and other countries. See https://www.sap.com/corporate/en/legal/copyright.html for additional trademark information and notices. All other product and service names mentioned are the trademarks of their respective companies.

For more information:

Andrew Baud, Distil

[email protected]

+44 (0) 7775 715775

 

 

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Kite Reports Results of Yescarta in P-II ZUMA-5 Study for Adult Patients with R/R Indolent Non-Hodgkin lymphoma

Shots:

  • The P-II ZUMA-5 study involves assessing Yescarta (axicabtagene ciloleucel) in patients with r/r iNHL after at least 2L of systemic therapy. After a single infusion of Yescarta, 92% of patients responded, including 76% of patients achieving a CR @median follow-up of 17.5 mos., results presented at ASH
  • The data supports the US FDA’s acceptance of BLA and is currently under PR for r/r FL, MZL, after two or more prior lines of systemic therapy, with an anticipated PDUFA date as Mar 5, 2021
  • Yescarta is the first CAR T-cell therapy to be approved by the US FDA for patients with r/r large B-cell lymphoma after two or more lines of systemic therapy

Click here ­to­ read full press release/ article | Ref: Businesswire | Image: Seek Vector Logo

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The urgent need for education and awareness about mitochondrial disease

Delayed diagnosis, misdiagnosis, and no cure or disease modifying treatments – children living with mitochondrial disease face all the issues often associated with rare diseases. It’s a problem that Michelle Bamber, whose two little girls both have the life-limiting condition, knows only too well.

Lily was a happy, contented baby, and her parents had no cause for concern until she went for her two-year check and was put on a pathway to diagnosis.

She is now 11, “healthily unhealthy”, and has just started secondary school. On the surface things are going well, but it has been a difficult journey for the family.

“Lily was a normal baby, and she met all the normal milestones,” says Michelle. “It was only when she was coming up to two that that we started to see issues that we weren’t really happy with.

“There was a delay in getting her two-year check, but when we did, she was referred to the Child Development Centre.”

Initially, they were told Lily probably had cerebral palsy, but as she continued to develop her healthcare team decided she needed further assessment. What followed was a litany of tests and invasive procedures as doctors tried to get to the bottom of her symptoms.

Michelle said: “She was walking, then she had a CT scan, and stopped walking. That obviously rung alarm bells. She had a lumbar puncture, a muscle biopsy, various scans. She had three general anaesthetics in the space of seven weeks.

“The internet told us some very disturbing things: that my child wouldn’t live past the age of five or that children don’t really live longer than two years after diagnosis”

“In August 2011, they said it was a form of mitochondrial disease, but didn’t know what one at that point. They just said it was a neurodegenerative, progressive disease, very similar to Parkinson’s.”

Lily was kept in hospital for a week where she was treated with IV vitamins, then the family were discharged with no further information.

“It was a total shock. We were just sent away from the hospital; we did not know anyone else who had it and we were very much in the dark about what was going to happen. We were on our own,” says Michelle.

“Where do you turn apart from Google? The internet told us some very disturbing things: that my child wouldn’t live past the age of five or that children don’t really live longer than two years after diagnosis.”

Priceless support

It was at this point that Michelle discovered Lily’s Foundation, a patient advocacy group established by Liz Curtis in memory of her own Lily, who died from mitochondrial disease at just eight months old.

Through the foundation, which Michelle actively fundraises for, the Bambers have met other families who are affected by mitochondrial diseases and have been able to access evidence-based information and support.

Mitochondrial disease, or ‘mito’, is an umbrella term for a group of medical disorders caused by mutations in the mitochondria, or the cell “powerhouses”, she explained.

The genetic condition affects different people in different ways, and the symptoms might include seizures, fatigue, vision and hearing loss, cognitive disabilities, respiratory problems, and poor growth. It can affect any of the body’s organs and systems, including the brain, heart, lungs, gut, liver, and skin. Crucially, there is no cure, and while work is ongoing to develop pioneering genetic therapies, the current standard of care relies heavily on symptom management.

That’s what makes the support provided by Lily’s Foundation invaluable, says Michelle, who explained that Willow, who was born in 2013, had been diagnosed at just four months in part due to the family’s new-found awareness of the signs.

“Usually, the foundation put on family weekends so all the families can come together. The kids go off and have fun with the volunteers at the kids’ clubs.

“The parents have lectures from the mitochondrial specialists and get all the latest advice and news on what’s happening in the medical world.”

Extended support

While the charity has not been able to host such events during the COVID-19 pandemic, it has been able to offer support to many more families thanks to a sudden uptick in awareness.

Back in May, the son of long-standing Coronation Street characters Leanne Battersby and Steve McDonald, Oliver, was diagnosed with mito after suffering seizures.

“That’s, obviously, creating a good bit of awareness and phone calls to the Lily Foundation have really increased. More people have been welcomed into the Lily family and are not living on their own with whatever their child’s going through anymore,” says Michelle.

She also hopes the storyline, which the charity and specialist doctors were consulted on, will help to address a lack of awareness among healthcare professionals that can often result in delayed or incorrect diagnosis.

“A lot of people report that they are having seizures and are diagnosed with having epilepsy, for example. Sometimes it can take a long time to get to that bigger picture and see that a patient has this, this, and this and that it is all because of mitochondrial issues,” Michelle explains.

Asked what parents who suspect their child has a rare disease like mito should do in this situation, Michelle said they should “trust their gut”.

“If you think there’s an issue, then keep talking to the doctors. Unfortunately, a lot of people find that they’re not listened to – they have to keep going back and keep insisting that things aren’t right,” she says.

For more information about the Lily Foundation, click here.


Cambridge Rare Disease Network Exploring Rare Diseases is produced in partnership with Cambridge Rare Disease Network (CRDN). CRDN is building a vibrant network of patients and stakeholders to share knowledge and foster innovation that leads to better diagnosis, treatment and support for those living with a rare disease.

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Jazz Pharma and PharmaMar’s Zepzelca Fail to Meet its Primary Endpoint in P-III ATLANTIS Study for SCLC

Shots:

  • The P-III ATLANTIS study involves assessing Zepzelca (lurbinectedin, 2.0mg/m2) + doxorubicin vs topotecan/ CAV in 613 adult patients in a ratio (1:1) aged ≥18yrs. with SCLC whose disease progressed following one prior Pt.-containing line
  • The study did not meet its 1EP of OS in the ITT population while there were no adverse effects on OS with the experimental arm. The safety data were consistent with the known safety profile of Zepzelca monothx. with no new safety signals observed
  • Zepzelca is an alkylating drug that binds guanine residues within DNA. The therapy received the US FDA’s accelerated approval in Jun’2020 for m-SCLC with disease progression on or after Pt.-based CT

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

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Social media erupts as anti-vaxxers respond to COVID-19 vaccine approval

In the midst of the jubilation about the UK’s emergency approval of Pfizer/BioNTech’s COVID-19 shot in the UK came the depressingly inevitable round of anti-vaccine social media activity and lobbying.

The green light for BNT162b was swiftly followed by posts on Twitter likening the vaccine to thalidomide – the drug that notoriously resulted in thousands of children being born with birth defects in the 1960s.

That ignores the fact that the thalidomide tragedy itself was responsible for the introduction of evidence-based medicine and reforms to the regulatory system that keep patients safe today.

While thalidomide is trending in the UK, it’s worth noting that many tweets are being posted by people slamming the #antivaxxers – a hashtag that is currently also riding high.

The Medicines and healthcare products regulatory Agency (MHRA) has been warning for some against anti-vaccine rhetoric that it fears could derail the coronavirus vaccination programme – which would be larger than any adult campaign carried out to date.

Pre-empting the backlash, MHRA’s chief executive Dr June Raine said at a Downing Street press briefing that despite the speed of its review, completed just three weeks after the final data were made available, no corners had been cut.

The vaccine had been approved after “an extremely thorough and scientifically rigorous review of all the evidence of safety, of effectiveness and of quality,” she asserted, adding: “The safety of the public will always come first.”

As the vaccine starts to be distributed, the National Institute for Biological Standards and Control (NIBSC) will be carrying out independent lab tests to confirm that every single shot that goes out meets the required standards for safety and quality, according to Raine.

Anti-vaxxers have been responsible for promulgating a series of fantastical rumours and conspiracy theories about coronavirus vaccines, including a persistent claim that vaccination will result in people being implanted with a microchip that will be used to track them.

Other false claims are that RNA-based vaccines like BNT162b can alter a recipient’s DNA and that the shots will contain tissue from aborted foetuses.

While some of these are frankly comical, the fear is that the spread of misinformation into mainstream media sources could result in fewer people taking up the opportunity to be vaccinated, undermining the programme.

Research published by the Vaccine Confidence Project – a unit of the London School of Hygiene & Tropical Medicine – found that misinformation around a COVID-19 vaccine induced a fall in the willingness to receive it among those who would otherwise “definitely” vaccinate.

VCP’s study found that only 54% of UK people would definitely have a COVID-19 vaccine – higher than the 41% seen in the US – with most of those who were reluctant citing safety concerns or a sense the threat posed by the pandemic had been overblown.

That’s already fewer than is required for herd immunity – a level of protection that would impact on virus transmission – but most worrying was that exposure to misinformation reduced the proportion of those definite responses by more than 6%.

“I hope that enough people take these vaccines, but I think it is going to be much more of a challenge than is recognised,” VCP director Heidi Larson told the Financial Times this week.

Speaking to the BBC today, Pfizer’s country manager for the UK, Ben Osborn said that “after the provision of clean water, vaccines are…the single most effective public health intervention we can make”. He also stressed that the study behind the approval was assessed by an independent panel with no links to Pfizer and BioNTech.

Health secretary Matt Hancock has also responded to questions about the anti-vax movement today, telling LBC radio that “the good news is that it’s not growing”.

“We monitor this very carefully and actually the number of people who want to have the vaccine is increasing,” he said

“The regulators are fiercely independent – they would not approve this if it wasn’t safe.”

800,000 doses of BNT162b have passed batch testing and should be ready within the next few days, and will be prioritised for elderly people in care homes and care home staff, followed by over-80s and health and care service workers.

The UK has ordered 40 million doses – enough for 20 million people – with several million doses expected to be available by year-end. Scottish leader Nicola Sturgeon said the first vaccines would be available in Scotland from Tuesday next week.

Twitter has also seen a debate about how the UK was able to become the first country in the world to approve the vaccine.

Hancock said the country was able to move quickly because of Brexit, but Raine emphasised in the press briefing that the approval was “made under provisions under European law which exist until January 1”.

The European Medicines Agency will meet on 29 December to decide if the safety and efficacy of Pfizer and BioNTech’s vaccine supports its approval.

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Member of COVID R&D Alliance Report the First Patient Enrollment in COMMUNITY Study

Shots:

  • Amgen, UCB, and Takeda reported that the first patient has been enrolled in the COMMUNITY trial. The study will test whether Amgen’s Otezla, Takeda’s lanadelumab, and UCB’s zilucoplan can reduce the severity of COVID-19 in hospitalized patients by moderating the immune system’s response to the disease
  • The focus of the trial is to identify an effective treatment for hospitalized COVID-19 patients, who are Grade 2 to Grade 5 on a clinical severity status 8-point ordinal scale. Trial sites are being established in hospitals across the globe to support enrollment in communities
  • This is the first time that the global pharmaceutical has come together to launch an adaptive clinical trial

Click here ­to­ read full press release/ article | Ref: Takeda | Image: Stat

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‘Knowledge is power’ for rare diseases and NAbs

Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies.

Neutralising antibodies (NAbs) present a unique challenge to researchers looking to treat patients with AAV treatment. As AAV is a virus derived from the common cold, the immune system can easily have levels of pre-existing NAbs that will recognise and neutralise AAV, rendering it ineffective.

In serious cases, higher levels of pre-existing NAbs can even cause a dangerous immune reaction when such therapies are administered.

This is particularly pertinent in rare diseases, as a number of gene therapy products use viral delivery methods to deliver the transgene to target organs.

“NAbs are antibodies that are part of the humoral response of the adaptive immune system,” explains Rich Horgan, founder and president of Cure Rare Disease (CRD), a nonprofit biotech developing CRISPR therapeutics for Duchenne muscular dystrophy (DMD). “They defend against foreign pathogens or infectious particles, and are specific to the targeted antigen, neutralising its effect and rendering it no longer pathogenic by binding to it.

“The goal is to not have the body neutralise the therapeutic, and therein lies the issue with NAbs. For gene therapy treatments using viral delivery methods such as AAV, NAbs are an issue that we must all contend with for both initial patient dosing as well as subsequent redosing.

“Our belief is that patients should understand the challenges associated with drug development and how those challenges impact them personally… It is emotionally challenging for patients who must put their faith in the hands of pharma companies”

“Regardless of route of administration, the virus can be identified as a foreign pathogen causing the adaptive immune system to try and eliminate it through NAbs or other means.”

A further issue is that patients can, and do, develop NAbs from environmental exposure. For example, since AAV is derived from a common virus, patients can develop NAbs against AAV from common colds or other infections – and ultimately, even low levels of antibodies can prevent successful transduction of a viral-based therapeutic.

Clinical trials will screen potential patients for NAbs against the delivery vector to understand the level of NAbs present in the patient (known as the titer level) before dosing them with the experimental drug – but there is currently no standardisation for measuring NAb titer levels in a patient, and each pharma company develops their own assay for testing.

“Moreover, there is not a generally agreed upon threshold for a dose/no-dose decision,” says Horgan. “Ultimately, titer levels are related to safety, as dosing a patient who has a higher titer level (however that may be defined) is more likely to trigger an immune response to the treatment, an extreme example being the infamous case of Jesse Gelsinger.

“As additional gene therapy trials advance, it is important that bodies like the FDA begin to standardise NAb measurement assays and acceptable titer levels for inclusion in clinical trials.”

But although companies are able to screen patients in an attempt to avoid complications, only those already in line for clinical studies qualify, even though for many patients it’s critical to know this information in advance as they consider potential trials.

“To date, there hasn’t been a widely available mechanism for patients and patient families to get insight into whether or not they have a significant level of NAb activity other than through enrollment in a clinical trial,” says Horgan.

Because of this, Horgan believes there needs to be more conversations surrounding this issue, and much more education of patient communities regarding the issue of NAbs and the challenges they create towards participation in a gene therapy study.

“Our belief is that patients should understand the broad challenges associated with drug development and how those challenges may impact them personally. While drug development is technically challenging, it is even more emotionally challenging for patients and patient families who must put their faith in the hands of pharma companies to develop effective treatments for their disease.

“Given the rise of many gene therapies, especially those in the rare disease space, neutralising antibodies stand as a significant challenge to the patient community and it is critical that patients understand that a) these neutralising antibodies exist and can potentially prevent patients from participating in a gene therapy clinical trial and b) collaborative development efforts are needed to ensure that patients can get access to life-saving gene therapies if they possess a significant level of NAbs.”

As such, CRD has rolled out a new system to allow patients to get an understanding of where their neutralising antibody levels stand – knowing that these levels may change over time and that medical decisions should not be based on these results.

The system offers community access to preclinical neutralising antibodies screening. CRD enrolls patients into their research study, then sends a patient kit for local blood collection through a lab. Patients are then informed of the research results several weeks later, and are provided genetic counselling to understand and emotionally handle the results.

“We’ve gone to great lengths to provide a high level of counselling as we know, from personal experience, the results are more than data points – they are the hopes and dreams of a family counting on a potentially life-saving gene therapy,” says Horgan.

“Ultimately, we believe that patients should have knowledge of the impact of neutralising antibodies and must advocate for the advancement of technologies so that all patients may be eligible for viral-based therapeutics. Time and access are of the essence.”

Horgan says that the long-term benefits for families who choose to get screened comes from this generated knowledge.

“Knowledge is power. While the information provided does not change the reality of the situation, we hope that it encourages families to ask more questions and to advocate for the development of technologies to enable dosing with a viral-based therapeutic.

“While non-viral gene therapies will someday be possible, leading researchers believe it will be a number of years before this is realistically feasible.

“Moreover, we want to help patients avoid the immense emotional disappointment of a situation where the patient is found to be ineligible for a gene therapy study due to NAbs even though they never knew that NAbs were an issue to begin with.

“By rallying together a community, we can catalyse the development of technologies to enable dosing (and re-dosing) of advanced therapeutics so that all have access.”

About the interviewee

Rich Horgan is the founder and president of Cure Rare Disease. He has a deep passion for Duchenne muscular dystrophy (DMD) and other rare diseases. With a younger brother impacted by DMD, Rich has a strong interest in accelerating promising treatments for the disease. He has formed a collaboration with world-class researchers and clinicians to pioneer the rapid development of customised therapies for Duchenne and other rare diseases.

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How the rare disease community has developed fertile ground for progress

Amanda Barrell explores how a perfect storm of changing economics, advances in technology, and the increasing volume of the patient voice is stoking change in the rare disease space.

New models of drug development are fuelling life-changing advantages in the rare disease space, previously an economic no-go area for pharma and biotech companies.

That was among the discussion points during Fighting Rare Diseases – The Science, Economics and the Patients, a webinar hosted by o2h Group.

Chairing the event, the company’s Prashant Shah, said: “Rare disease, by definition, means it affects a fairly small number of patients. But the economics are beginning to change, the return on investment is changing, and there is a lot more interest now.

“There are more organisations coming into play and patient groups and charity groups are becoming ever more active. I think there’s more hope for those suffering from rare disease than ever before.”

The interplay between technological advances, new models of drug development, and an increase in patient centricity, has created fertile ground for progress, the panel said.

“Partnering with patient groups has really been our superpower from the beginning, because they are the experts”

Michael Binks, vice president of Rare Disease Research at Pfizer, said: “There’s been a growing awareness of the magnitude of the unmet need, that there are 7,000 identified diseases…and very few therapies available for the majority of them.

“Key factors have been the emergence of communities around some of these diseases that have driven major legislative change and ensured that regulators are more flexible.”

This shift in the regulatory environment has made developing medications for the 300-500m people affected by rare disease globally more economically feasible, said Binks, whose company is focusing on gene therapies in the rare disease space.

Shifting tide

There’s never been a better time for rare disease patients, said Tim Guilliams, CEO of AI-powered biotech company, Healx, who believes that technology such as machine learning (ML) is enabling researchers to take a new view on drug development

His company’s approach is to work with patient groups to understand unmet needs, then use ML to identify existing drugs that could tackle that need and bring them to trial.

“Drug discovery is really hard, and ML is not a magic wand. It’s really just bringing that component to the table to try to move as quickly as you can to get treatments into the clinic,” he said, adding that the method also needed the input of “amazing” pharmacologists, clinical experts, and patient advocates.

“Partnering with patient groups has really been our superpower from the beginning, because they are the experts,” he said.

Return on investment

Shah asked Binks and Guilliams if this paradigm shift in terms of patient involvement was contributing to higher returns on investment in clinical trials.

“It’s hard to put an absolute number on, because each disease has different endpoints, different number of patients that you can enrol, etc. but yes, we believe we can get the cost of clinical trials down significantly because of our model,” said Guilliams.

Binks said that working with patients early on could cut overall costs by reducing the likelihood of study failure.

“Running high-quality clinical trials is expensive. It is sometimes made more expensive by the frequency of failure because we don’t have an adequate understanding of the patient population or the disease.

“Bringing the patients and their families into the conversation early does help to define the clinical development path.”

Market research

Nicola Miller, editor-in-chief at Rare Revolution Magazine and co-founder and trustee of the Teddington Trust for those affected by Xeroderma Pigmentosum, said it made perfect business sense to involve patients in drug development early on.

For a start, she said, there is an assumption within the research community that everyone with a rare disease is seeking a curative treatment, yet many people accept their condition as part of who they are.

“We have all heard stories of where scientists have gone down a particular route, but they haven’t actually thought of engaging with the population as to what is the most debilitating part of their condition,” said Nicola.

“They could be developing something for photo sensitivity for a particular condition, for example, but generally people can cope with that, what they don’t want is a neurological decline which is going to impact their life.”

While some organisations were working well with patient groups, others appeared to be involved in more of a “box ticking operation” which doesn’t benefit anyone, she went on.

“There are huge sums of money and huge amounts of technology being ploughed into this area at the moment, so let’s make sure it’s going into the most beneficial point for the patients,” she said.

Rare future

All three panellists agreed that there was an abundance of hope on the horizon for people living with rare diseases – so long as the whole community continues to work together to overcome the challenge.

“So many things are moving in the right direction: diagnosis, possible treatments, technology, and empowerment. It’s really incredible what is happening in this space now because that just wasn’t the case 20 years ago,” said Guilliams.

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COVID-19 lessons for how pharma should support patient advocacy

Trio Health’s Neil McGregor-Paterson asks whether the healthcare industry has paid enough attention to the patient voice during COVID-19 and looks at the many ways pharma can improve the patient experience across the sector going forward.

Never has the health and care voluntary sector (HCVS) played such a critical role in supporting our nation´s health and wellbeing. Since the start of the COVID-19 pandemic, the sector has stepped up – and in – to deliver support and services where the NHS and social services were unable to. All with a speed and agility that could never be replicated by providers.

Even now, the sector continues to plug services while NHS and social care retains its focus on COVID-19. But this level of involvement cannot and will not last. And, as the sector plans for a precarious and challenging future, there are several established and emerging themes that can help inform pharma on approaches to support it.

The importance of the long-term and sustainability

A recent report from National Voices, The Neurological Alliance and the Arthritis and Musculoskeletal Alliance showed that while there had been a significant increase in demand for HCVS services supporting those with long-term conditions, the pandemic has had a devastating effect on the sector’s finances. Of the 40 organisations that participated in their research, almost a third predicted a 40% drop in fundraising income in the period June 2020 to June 2021, and a further third a 25% drop.

Commenting on the current and future funding challenges facing the HCVS, Colin McGregor-Paterson, CEO for Oasis Partnership, a charity providing social and psychological support services to improve health and wellbeing, said, “The crisis in funding has significantly impacted on many organisation’s capacity and capability to deliver in the here and now, while challenging the sustainability of many. Collaboration with industry has never been more important, and this needs a commitment to the longer-term if the UK’s vibrant advocacy sector is to be sustained.”

This is a sentiment backed up by Annabel Cowper, director of corporate affairs at Biogen: “As we move beyond the acute COVID-19 crisis, it’s important that our industry understands the significant pressure the patient advocacy sector is under. Our support for the sector has never been more critical, but in a funding constrained environment, this support needs to be carefully targeted if we are to maximise patient gain.”

“Considering this is the greatest crisis this country has faced since the second world war, the voice of the patient and their advocates appears to have been noticeably absent in national COVID-19 planning”

Keeping the patient voice centre stage

Health and social care policy makers and providers are committed to ensuring that the voice of those with lived experience, and those organisations that advocate for them have a place around the planning table. Yet, considering the greatest crisis this country has faced since the second world war, the voice of the patient and their advocates appears to have been noticeably absent in national COVID-19 planning.

Who knows if things might have been different had policy makers and their advisors listened to the voice of patients? Maybe peoples’ fear of using NHS services during the pandemic would have been predicted and mitigated from the outset, possibly preventing thousands of avoidable deaths.

For industry, this should serve as a salutary reminder of the importance of listening to the voice of patients and their advocates as plans are being developed and signed-off for 2021 and beyond.

Addressing health inequalities

Even before COVID-19, in the UK there was a persistent 20-year gap in the number of years lived in good health between those living in the most and least affluent areas. And, for the first time in a decade, life expectancy has stalled for many, and declined for the poorest 10% of women.

COVID-19 has exacerbated health inequalities, while exposing the fact that underlying health inequalities, while not new, have been overlooked for a long time. This has resulted in an increased focus on addressing these among policy makers, providers and importantly the HCVS.

While pharma has a great track record in supporting equity of access to medicines and services, the need for improved data, measurements, reporting, and comparisons, coupled to the complexity of addressing health inequalities means that ex-US, pharma has traditionally steered clear of bespoke health inequalities initiatives beyond health literacy type projects and/or funding for third party organisations. However, times have changed, and just as the HCVS is increasingly stepping up to help address health inequalities, so companies focused on those conditions that disproportionately affect poorer and disadvantaged communities must also step up and collaborate with them.

“The pharmaceutical industry could become an important voluntary sector partner in addressing health inequalities if it is prepared to go beyond patient advocacy and get behind whole communities that are taking action that increases resilience and creates health,” said Merron Simpson, chief executive of New NHS Alliance, a national cross sector movement addressing health inequalities through Health Creation. “On a practical level there are several roles they could play such as supporting digital literacy or helping communities get access to and interpret local datasets. The most important thing is to listen to the priorities of local people.”

Community up vs. national down

The people and community response to COVID-19 has been phenomenal. Beyond the NHS Volunteer Responders programme, three million people collaborated with local organisations outside the system. They came together with speed to self-organise and meet the specific needs of their communities. From streel-level WhatsApp groups to locality-wide delivery of medicines, the benefits of community resilience have been recognised at the highest levels of government, while its link to digital health and power shifting to communities has been made.

In his report to the Prime Minister, entitled Levelling up our communities: proposals for a new social covenant, Danny Kruger MP, recognises that: “The experience of the recent crisis the willingness of local people to step forward and collaborate, the flexibility shown by public services and the social commitment of businesses shows what is possible. Add the extraordinary new dynamics of data and digital innovation, and a wholly new paradigm is possible in which community power replaces the dominance of remote public and private sector bureaucracies.”

“Digital is just another means of providing patients with choice, and it must not become the default position”

Commenting on the implication of Kruger’s report on UK pharma, Paul Naish, director of UK policy at AstraZeneca said: “The Kruger report flags the importance of digital – where our industry is already highly active – but also issues that should be a clarion call for pharma to advance community collaboration within, and alongside our deep commitment to established patient organisations. This way we can support people to be healthy in addition to supporting patients to be well.”

Creating health alongside prevention and treatment

COVID-19 has demonstrated the value of resilient and connected communities, while reinforcing that creating health within communities must sit alongside prevention of ill health and treating illness through medicines and services. Creating health is not new, but it is effective, and it is cost effective. It is also an area that pharma should be actively involved in.

Commenting on the potential role of pharma in creating health, Lord Nigel Crisp, former chief executive of NHS England, and author of the recently launched, Health in made at home, hospitals are for repairs, said, “The NHS can’t by itself deal with many of today’s major health problems such as loneliness, stress, obesity, diseases of poverty and addictions. It can only react, doing the repairs but not dealing with the underlying causes.

“The pharmaceutical industry has a great track record in working with the NHS in treating and preventing illness through medicines, vaccines and support for services. But, since healthcare provided by the NHS accounts for only 10-20 percent of a population’s health, the industry needs to think about collaborating more widely with the health and care voluntary sector to support the creation of health in schools, homes, communities and the workplace. This will have the added benefit of reducing the burden on the NHS.”

The value of digital as a connector and enabler

COVID-19 has had a super booster effect on the adoption of digital technology to support patients. It has also led to a shift in attitude in the healthcare setting, with providers wanting patients to take greater responsibility for their own health and wellbeing with technology. This is turn is matched by a stepping-up of the use of technology by the HCVS in terms of better connecting people and communities, reaching out to communities, taking services and support online, and launching new services such as Zoom exercise classes. In addition, the sector has been actively breaking down barriers to digital exclusion through training, supporting funding of technology and targeted outreach to vulnerable, disadvantaged, and isolated communities who would otherwise not be engaged in digital and digital health.

Commenting on the role of pharma in further supporting advocacy in this digital space, Mindy Daeschner, chief executive of Daeschner Consulting and author of What’s next in digital healthcare transformation post COVID-19, said, “We know that industry has made a significant contribution to digital health and to supporting organisations up their digital capacity and capabilities. However, digital is just another means of providing patients with choice, and it must not become the default position. COVID-19 has made it clear that digital exclusion is and will remain a challenge for some, while for many patient’s people interaction is critical. Supporting digital initiatives that enhance choice and people interaction must be a key consideration.”

Building Better Together

As countries focus on Building Back Better, New NHS Alliance has been quick to point out that this means Building Better Together. There is a great opportunity for pharma to revisit and refocus advocacy programmes to support the HCVS to continue building connectivity within communities.

COVID-19 has radically changed the environment within which the VHCS sector operates and this must be understood and reflected by industry. At the heart of this is listening to the voice of organisations, but in today’s word, industry must take this one step further and listen to the voice of people and the communities the VSCS represent.

About the author

Neil McGregor-Paterson is director of connections and collaboration for Trio Health.

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Alnylam’s Oxlumo (lumasiran) Receives the US FDA’s Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients

Shots:

  • The approval is based on P-III ILLUMINATE-A & -B trials. The studies demonstrating reductions in urinary oxalate and encourage safety and tolerability in pediatric and adult patients
  • The ILLUMINATE-A showed that Oxlumo met its 1EP i.e. change in 24hrs. (65% vs 12%) compared to PBO, the study also achieved significant results for all 6 tested 2EPs
  • In ILLUMINATE-B, Oxlumo demonstrated a 72% mean reduction in spot urinary oxalate: creatinine ratio from baseline to 6mos., reduction of oxalate as consistent across all three body wt. categories. Additionally, therapy demonstrated positive results across 2EPs, including additional measures of oxalate

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

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The Art of Listening: Beyond the Chief Complaint

By HANS DUVEFELT

A doctor’s schedule as typical EMR templates see it only has “Visit Types”: New Patient, 15 minute, 30 minute. But as clinicians we like to know more than that.

One patient may have a brand new worrisome problem we must start evaluating from scratch, while another is just coming in for a quick recheck. Those are diametrically opposite tasks that require very different types of effort.

Some visits require that test results or consultant reports are available, or the whole visit would be a waste of time. How could you possibly plan your day or prioritize appointment requests without knowing more specifically why the patient needs to be seen?

So, as doctors, we usually want our daily schedules to have “Chief Complaints” in each appointment slot, like “3 month diabetes followup”, “knee pain” or “possible dementia”. That helps everybody in the office plan their day.

I always bristled at “not feeling well” because that is too nonspecific. After all, that could be something that would have been better handled with a 911 call. But there is also a danger in being too simplistic when classifying what people come in for. We like to pigeon hole clinical concerns a little too quickly sometimes.

I had such a situation recently. It hinged on the patient’s choice of one common word over another.

A middle aged woman wanted to be seen for “throat pain”. It was halfway into a busy afternoon and between the three providers in our office, we had no openings to offer her.

Autumn asked me, “can we fit in a throat pain today? I’ve got Nicole Bamford on hold”.

“What kind of throat pain?” I asked. “You mean just a sore throat?” I was working on refills between patients. Autumn asked the patient to elaborate while I continued to work.

“She says she can swallow all right but for the last few days she gets this pain in her throat every time she does anything heavy.”

“Does she have pain right now?” I asked.

Autumn checked. “No.”

“Have her come right over.”

Nicole had no cold symptoms. She had normal vital signs. She had a two week history of throat and occasionally jaw or ear pain after minor exertion, never more than a few minutes. Sometimes she felt a little short of breath at the same time.

Her exam and her EKG were normal. She was a smoker with a family history of heart disease.

“Call the ambulance, 54 year old woman with new angina, no pain right now. I’m calling the ER”, I told Autumn after I explained my assessment to Nicole. She had seemed to accept my diagnosis of unstable angina without questioning and also my recommendation that we get her to the hospital by ambulance without expressing any sign of surprise or emotion.

When I saw her in followup after her ER visit, transport to the tertiary care center and successful stenting of a 95% blockage of one of her coronary arteries, she told me “I thought you were crazy”.

I thought to myself that this could have played out very differently if the nuance between “throat pain” and “sore throat” had gone unnoticed.

It’s nice to know what a patient is coming in for, but that isn’t necessarily the diagnosis they leave with.

Hans Duvefelt is a Swedish-born rural Family Physician in Maine. This post originally appeared on his blog, A Country Doctor Writes, here.

Healthcare’s COVID-19 backlog: how pharma can help

IQVIA’s Sarah Rickwood explores how pharma can help healthcare systems address treatment backlogs caused by the COVID-19 pandemic.

As lockdowns started in the West during March 2020, there was, inevitably, much punditry on exit scenarios – for economies, populations, and healthcare systems. Considerable time was devoted to the discussion of V- , U- and L-shaped recoveries.

As we cope with second waves of COVID-19 infections, it is probably fair to say most early pundits were over-optimistic on the length of time the crisis would take to resolve. The pandemic’s aftermath is looking to stretch into the first half of the 2020s. One of the most far reaching impacts will be from the backlog of non-COVID-19 patients who have seen diagnosis and treatment delayed, slowed or even missed and cancelled. For many patients the consequences will be devastating.

With the first wave of lockdowns, treatment backlogs were inevitable due to physical distancing requirements and a pivot to focus on COVID-19. The most immediate impact was cancellation of elective surgeries and other procedures, and the movement of healthcare professional/patient consultations to virtual platforms.

A study in the British Journal of Surgery estimated that globally, 28 million procedures would be cancelled or postponed during the peak 12 weeks of COVID-19 disruption. The same study also made an important prediction: even if all countries increased their normal surgical volume by 20% after the pandemic, it would take a median of 45 weeks to clear the backlog of operations resulting from the 12 weeks of the first wave of disruption.

It is pretty clear that healthcare systems are unlikely to be currently at 120% of their pre-COVID-19 capacity for elective surgeries. In all likelihood, as Europe and the US are in the throes of a second wave of COVID-19 infection, the specific backlog of elective surgery will take longer than predicted to address. Even past the second wave, as vaccines become available, healthcare systems will not return rapidly to pre-pandemic capacities, let alone deliver more than that capacity. The conclusion is that backlog clearance will extend into 2021.

“It is pretty clear that healthcare systems are unlikely to be currently at 120% of their pre-COVID-19 capacity for elective surgeries. In all likelihood, as Europe and the US are in the throes of a second wave of COVID-19 infection, the specific backlog of elective surgery will take longer than predicted to address”

Elective surgery, is, of course at one end of the spectrum of medical conditions with respect to disruptability by the pandemic – by definition they are optional (although if surgeries are postponed, they may become less optional over time), and necessitating visits to a health facility, which may not be possible if there is an infection risk, or that facility has been repurposed for COVID patients. Patients with other conditions, especially cancers, cannot wait.

It is clear that delay in referrals, diagnosis and treatment can mean the difference between treating a cancer that is early stage and therefore often curable to one that is late stage and could result in early death. In an August 2020 paper in the Lancet Oncology, the authors noted that referrals via the 2-week-wait urgent pathway for suspected cancer in England were reported to have decreased by 84%.

The authors used data on cancer patients treated in the English NHS to model the impacts of lockdown induced delays in diagnosis and treatment, and concluded, “Delays in presentation via the 2-week-wait pathway over a 3-month lockdown period (with an average presentational delay of 2 months per patient) would result in 181 additional lives and 3,316 life-years lost as a result of a backlog of referrals of 25%, 361 additional lives and 6,632 life-years lost for a 50% backlog of referrals, and 542 additional lives and 9,948 life-years lost for a 75% backlog in referrals.” The 2-week-wait pathway refers to the request from a General Practitioner in England for an urgent referral to a specialist of a patient exhibiting symptoms which could be cancer.

IQVIA has been conducting a survey of oncologists and haematologists on their experience of treating their cancer patients before and during the pandemic. So far, the study has undertaken three waves of research, covering hundreds of oncologists/haematologists across the five major European countries.

The cancer specialists interviewed reported that, on average, they were seeing 77 patients a week prior to the pandemic. This fell to 41 patients a week at the height of the first wave of the pandemic. The June period, which coincided with lows of infection numbers in these countries, was little improved, at 50 patients/week, and as countries faced the second wave of infection in October, the number of patients reported seen per week had fallen back again, to 45. This pattern was seen across the five countries.

Graphic 1

Because of the lockdown, it is not surprising, but very worrying, that cancer specialists reported delays in diagnoses, surgeries and chemotherapy during each of the three waves of interviews. Graphic 2 details the average percentage of specialists reporting each type of delay across the five countries. It is notable that the percentage of specialist reporting delays, which had dropped during the June infection low, rose once again when the second wave was being faced during October.

Graphic 2

In summary, we have evidence that cancer specialists are reporting delays to patient diagnosis and treatment for cancers across the major five European countries, delays that are continuing past the first waves of the pandemic and into the second, and there are models which suggest that significant increases in mortality and years of life lost will be a consequence of such delays. Treatment backlogs caused by the pandemic are real, will have serious consequences for patients, and will take significant time to resolve.

I’ve picked for discussion two areas, elective surgery and cancer treatment, where patients need to visit health facilities, and in the case of cancers, treatment delays have serious consequences. Not all diseases or conditions are so far along these axes – many chronic conditions can be managed to a very large extent remotely, and for some conditions delays to treatment, even quite lengthy ones, may not have an immediate adverse effect.

Many chronic conditions, such as diabetes, hypertension, dyslipidaemia are also highly prevalent. The impacts of the pandemic backlog on treatment outcomes for these diseases will be played out over years to come, and IQVIA’s longitudinal patient data is already showing decreases in treatment changes (new and switch prescriptions) in relation to key chronic conditions such as diabetes.

“Pharmaceutical companies will undoubtedly place more emphasis on developing treatments that can be home administered and self-administered in the future, and payers and health technology assessors may well value those attributes more highly”

The treatment backlog caused by the COVID-19 pandemic matters – to individual patients, to public health systems, and to the pharmaceutical companies developing treatments which patients may now not be receiving as and when they should for optimal treatment. Pharmaceutical companies cannot address this challenge alone. But they can help, and we are starting to see how.

The first step to addressing the backlog is through raising awareness, with health policy makers and with patients. For patients highlighting the importance of seeking treatment, but without generating unnecessary anxiety and health system pressure, is a sensitive task to be done in close collaboration with health systems. Companies need to acknowledge the pressures that healthcare professionals are under and identify where and how they can alleviate pressures and help doctors to help their patients.

Second, as health systems emerge from the pandemic and vaccines are rolled out (and mass vaccinations will also place pressure on health system and some individual doctor’s time), they will need better data and insight on patient journey bottlenecks, on healthcare system capacity stresses and on the services required to remove barriers to effective treatment.

Finally, pharmaceutical companies should play their role in seizing the opportunity to learn and future-proof healthcare delivery against future pandemics. The pandemic highlighted the value of treatments which patients can self-administer, and in many cases there’s been big swings to treatments which can be taken at home – a movement from warfarin, which requires clinic visits, to Direct Oral Anticoagulants (DOACs or NOACs), which do not, for example, in anti-thrombotic treatment. Pharmaceutical companies will undoubtedly place more emphasis on developing treatments that can be home administered and self-administered in the future, and payers and health technology assessors may well value those attributes more highly.

Graphic 3

The pharmaceutical industry has risen magnificently to the challenge of COVID-19, in terms of treatment and vaccine development. As the virus is defeated and infection rates fall during 2021, pharma’s next challenge will become increasingly apparent – to help address the other public health crisis of the treatment backlog precipitated by the pandemic. The solutions that pharmaceutical companies can bring to address this, and the relationships that they can forge consequently, will shape the pharmaceutical industry’s role in healthcare systems for the remainder of the 2020s.

About the Author

Sarah RickwoodSarah Rickwood has 26 years’ experience as a consultant to the pharmaceutical industry, having worked in Accenture’s pharmaceutical strategy practice prior to joining IQVIA. She has wide experience of international pharmaceutical industry issues, having worked for most of the world’s leading pharmaceutical companies on issues in the US, Europe, Japan and leading emerging markets, and is now vice president, European thought leadership at IQVIA, a team she has run for eight years.

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3 steps for comprehensive patient collaboration

Janssen’s Daniel de Schryver tells us why it’s time to give patients a permanent seat at the table and improve health outcomes through patient-pharma dialogue.

I’m struck by just how much we thrive when we share a common purpose; when – as a society, a group or a family unit – we work together to achieve the same goal. It’s no different in healthcare. Pharma companies, healthcare providers and governments share a well-established common purpose: the desire to make disease a thing of the past. But healthcare is often a system of silos, and what can get lost in the gaps is the input of patients themselves. Which rather begs the question: how well are we caring for patients, if we can’t always hear their voices?

I saw a statistic in a Health Europe article, published in September this year, which reported that 81% of patients do not feel listened to by pharma companies. Patient-centricity has, I fear, turned into a buzzword. We must remind ourselves to focus on systematic changes – from the early R&D phases, all the way through to reimbursement discussions – that enable us to develop optimal solutions together with patients. In short, to ensure they have a seat at the table at every stage of the process.

To get back on track and embed a comprehensive collaboration between patients and industry, I believe we need to consider the following three areas:

“To ensure we are making a difference, we need robust KPIs, both short and long-term, to measure patient-industry collaborations. We must understand the value over time of these solutions”

1. Open conversations lead to better patient-industry collaborations

Pharma needs to convey a clear picture of who we are and what we’re trying to achieve. When the public sees patients working directly with us, it can still arouse suspicions, as if there’s some form of collusion going on. Transparency is crucial, therefore, so that any outcomes from our collaborations with patients are shared and accepted.

We have been adapting our processes at Janssen to put patients at the heart of everything we do. It’s a work-in-progress, because this means streamlining the way we work to include the patient voice within acceptable timelines, while still meeting compliance requirements. But the aim is for every function in our value chain to demonstrate how they have involved the patient in their decision-making.

A good example of a productive, open collaboration we’ve experienced with patients, clinicians and regulators is the one that resulted in the Psoriasis Symptoms and Signs Diary (PSSD). Through this outcomes measurement tool, patients keep comprehensive diaries of their condition, documenting what aspects of the disease affect them most.

A major insight revealed once the tool was in use was to not just focus on the percentage of the body covered by psoriasis – a patient with smaller areas of their face or hands affected, for example, may find it more difficult to cope compared to patients with larger areas affected on their backs.

Projects like these are time intensive, of course; this collaboration with psoriasis patients took five years to co-develop. But the important thing was that it worked, and that it has provided a great model of patient interaction for us to replicate.

2. Pharma must support patient empowerment

Patient empowerment is not a catchphrase; it is a genuine drive to enable patients to take ownership of their health. We need more people to see themselves as experts in their own treatment, so they feel able to provide input into key debates and initiatives. It should be our duty, as pharma, to help more people achieve this.

The European Patients’ Academy on Therapeutic Innovation (EUPATI) is a good example of this in action. EUPATI is a public-private partnership run by a multi-stakeholder consortium. It brings together patients, pharma, academia, regulators, non-profit organisations, and health technology assessment bodies, and it allows trained patient experts to input into the R&D process, regulatory deliberations, and other initiatives.

In the UK, my colleagues have created a Patient Advisory Committee, two of whom mentor several members of the UK management committee, helping them to further embed the patient voice in their decisions. A more empowered patient helps improve solutions that are beneficial to their own health and the health of many other patients besides, thereby contributing to a healthier future for all.

3. Measure the success of patient-industry collaborations

To ensure we are making a difference, we need robust KPIs, both short and long-term, to measure patient-industry collaborations. It is rewarding to see the immediate impact these projects can have, but we must also understand the value over time of the solutions we are co-developing with patients.

Patients Active in Research and Dialogues for an Improved Generation of Medicines (PARADIGM) is a public-private partnership paid for by the European Commission and the pharma industry, and co-led by the European Patients’ Forum and The European Federation of Pharmaceutical Industries and Associations (EFPIA).

PARADIGM provides a framework for innovative patient engagement approaches and is able to demonstrate the benefits of these approaches to all stakeholders. Through this partnership, we have been able to support the development of solutions designed to not only improve an individual patient’s experience of care, but also the overall health of populations, and which, ultimately, should reduce the per capita costs of care.

Putting patients at the heart of the process

It’s essential to listen to what patients have to say, to identify solutions that are beneficial individually and collectively, and to provide feedback on how patients’ insights have helped shape better outcomes for all. If we can listen and listen well, and if we can show we genuinely care about patients’ views, then their contributions can make a real difference.

As I said at the start, we thrive when we are united behind one common purpose. We must be united with patients, and that common purpose must be their purpose.

So, to any patients reading this, I urge you to join a patient advisory group and multiply your impact, to study with EUPATI and become an expert, and – put simply – to get involved and be heard. And to everyone with whom we work across the industry, let’s follow the process: ask the patient, include them from the onset, and ensure their insights are at the core of everything we do. Let their voice be the driving force in our shared goal of making disease a thing of the past.

About the author

Daniel De Schryver is patient engagement & advocacy lead, Europe, Middle-East and Africa, at Janssen. He joined Johnson & Johnson in 2001 as Director Corporate Communications. In that function, he initially worked in the field of oncology. In 2006 he joined the Janssen teams working in Infectious Diseases where he helped to maintain and enhance the company’s relationships with the HIV Patient Community. Later, he built the external relationships in the field of Hepatitis C, before becoming the Global Therapeutic Area Communications Leader Infectious Diseases and Vaccines.

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Regeneron’s Casirivimab and Imdevimab Receive the US FDA’s EUA as the First Combination Therapy for COVID-19

Shots:

  • The US FDA has issued a EUA for casirivimab (1,200mg) + imdevimab (1,200mg) administered together for mild to mod. COVID-19 in adults and pediatric patients aged ≥12yrs. weighing at least 40kg [~88 pounds]) with positive results of direct SARS-CoV-2 viral testing and who are at high risk for progressing to severe COVID-19
  • The EUA is based on a P-II study that showed a reduction in hospitalization or ER visits in patients at high risk for disease progression within 28days after treatment and a reduction in viral loads
  • Initial doses of Ab cocktail will be made available to ~300,000 patients, with no medication out-of-pocket costs, under the US government allocation program

Click here ­to­ read full press release/ article | Ref: Regeneron | Image: Fox Business

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Sanofi’s Supemtek (quadrivalent recombinant influenza vaccine) Receives the EC’s Approval to Prevent Influenza

Shots:

  • The approval is based on two P-III studies assessing the safety and efficacy of Supemtek (quadrivalent recombinant influenza vaccine) in 10,000 patients with influenza aged > 18yrs. Supemtek is 1st and only recombinant influenza vaccine approved in the EU
  • The P-III efficacy study demonstrated improved protection against influenza compared to standard-dose influenza vaccine and reduced the risk of influenza by an additional 30% in adults aged ≥50yrs.
  • Supemtek contains three times more antigen than both egg-based and cell-based standard-dose vaccines. Supemtek is also approved in the US under the tradename Flublok Quadrivalent

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

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Bridging the gap between pharma and the NHS in MS

Janette Curlis is a Multiple Sclerosis Nurse Advisor (MSNA) at Biogen, helping to support the company’s Multiple Sclerosis (MS) field teams and developing patient and Healthcare Professional (HCP) materials.

We spoke to her about being a bridge between pharma and the NHS and the challenges that are facing Multiple Sclerosis Nurses (MSNs) and patients today.

Janette, what does your day-to-day work involve as a MSNA?

I work within the medical department at Biogen covering the UK and Ireland where my role is wide and multifaceted. As well as supporting our field teams, I develop nurse and patient materials, education and projects.

As a MSNA, my role has naturally evolved to accommodate how I support the nurses that I work with. I listen to and identify the gaps in their education and work cross functionally to develop training and education that will support their needs – at the same time being mindful not to increase their workload.

“As the demands of the job have changed, perhaps it is time to review the role and skill mix needed within MS teams. MS support nurses and MS admin coordinators would free the MSNs to concentrate on more complex needs of their patients”

The Disease Modifying Therapy (DMT) landscape alone requires education, information and planning to ensure the patient has the right treatment at the right time and the best experience.

The role of an MSN has changed over the years and has become more complex – what would you say are the main challenges MSNs face today and what are some of the wider challenges in MS care now?

The role is multipurpose, challenging and ever changing along with the National Health Service (NHS) in the UK and the Health Service Executive (HSE) in Ireland.

MSNs are time-poor. As the role has developed there are several new challenges they face. With around 14 disease modifying therapies now available, the MSNs of today require a different skill set to previous years.

There is a lack of consistency when it comes to provision of dedicated admin support to the MSNs. The increasing workload has put pressure onto nurses to fit everything into their working week, whilst maintaining quality time with their patients.

As the demands of the job have changed, perhaps it is time to review the role and skill mix needed within the teams. MS support nurses and MS admin coordinators would free the MSN to concentrate on more complex needs of their patients.

Looking at the wider challenges, it’s difficult to separate them out individually. They are wide ranging, and, not unlike the NHS, a lack of investment in developing services that reflect the change in delivery that’s needed today and for the future is a major factor.

What I find is that there are inefficiencies in the systems causing delays and duplication of work, and with no time to address these issues we see delays on areas such as time to treat and referrals.

How is the NHS addressing these challenges, and how does Biogen support that work?

With limited resource around service improvement in the NHS, Biogen have a team of Neurology Business Managers who work with MS services across the UK and can identify where improvements can be made. I can then support the nurses by assisting where appropriate.

How do you support Biogen in helping to address these challenges?

Moving forward we are building on the programme we delivered in 2019; the nurse advisor team ran numerous bespoke events and individual teaching sessions.

We continue to support the MS Trust Mental Health Campaign and I am working with the team to look at how we can support both MSNs and people with MS.

What are the key areas you’re looking to focus on to help improve value and engagement in MS?

All new MSNs appreciate onboarding is difficult. I have a specific half-day training programme to enable the nurses to have a firm grounding in MS. I also hold follow up sessions for MSNs which include immunology, DMTs, and MRI.

Nurses often ask why I left my hospital position of MSN, and whether I miss the patients. My reply is always that I never left MS nursing; I still work on behalf of the patients, just in a different way!

About the interviewee

Janette CurlisJanette Curlis is a MSNA working at Biogen. She has worked in Neurology since 1991 and specifically as an MSN since 1997. Janette was one of the few MSNs pre risk sharing scheme recruitment. She worked in clinical research in MS studies as well as her role as MSN up until leaving the NHS in 2011. She worked with almost all of the now licensed therapies in trial phase. Janette moved to the pharmaceutical industry to take up a new role as nurse advisor for Novartis in 2011 and joined Biogen in 2014.

Biogen-50773

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Abbott Launches IonicRF Generator for Patients with Chronic Pain in the US

Shots:

  • Abbott has launched the IonicRF Generator, which is used to deliver a non-surgical, minimally invasive treatment for the management of pain in the nervous system
  • Radiofrequency ablation uses an electric current to heat up a small area of nerve tissue to stop it from sending pain signals. Studies showed that pain relief following a single radiofrequency ablation treatment can last from 6-12mos.
  • The IonicRF generator is the first Abbott-developed radiofrequency ablation device that uses heat to target specific nerves & block pain signals from reaching the brain, currently approved in the US & EU

Click here ­to­ read full press release/ article | Ref: Abbott | Image: Abbott Neuromodulation

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AstraZeneca and Amgen Report Results of Tezepelumab in P-III NAVIGATOR for Asthma

Shots:

  • The P-III NAVIGATOR study involves assessing Tezepelumab + SOC vs pbo + SOC in adults (18–80yrs.) & adolescents (12–17yrs.) with severe, uncontrolled asthma, who were receiving treatment with medium/high dose ICS + at least 1 additional controller medication with or without OCS
  • Trial met its 1EPs i.e. reduction in AAER @52wks. in the overall population. The study also met 1EPs in patients with low levels of eosinophils i.e. <300 & 150 cells/microlitre
  • Tezepelumab is mAb that inhibits the action of TSLP and has received US FDA’s BT designation in Sept’2018 for patients with severe asthma, without an eosinophilic phenotype

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

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5 Things Patient Advocates Want Pharma and the Healthcare Industry to Know

The COVID-19 pandemic has presented many obstacles, yet the healthcare industry continues to march forward to meet the needs of patients. With physical distancing, new office protocols and the widespread use of telehealth, the patient experience is changing–particularly for people living with chronic conditions, who may require more regular access to care.

To truly understand the impact of the changing patient experience on attitudes and behaviors, and the industry as a whole, Health Union recently passed the mic directly to the source. Through a virtual content series entitled The COVID-19 Effect: How Pharma Can Adapt to the Evolving Patient Experience, patient advocates living with multiple sclerosis, psoriasis, Crohn’s disease, Parkinson’s disease, migraine and lung cancer shared their personal accounts of life with a chronic condition during the pandemic and beyond. Presented alongside Health Union’s trusted survey data from tens of thousands of respondents, these live conversations shed light on what’s top of mind for patients through unforeseen challenges.

While much of each session focused on treatment journey and COVID-19, advocates were also given the opportunity to share advice for pharma and the healthcare industry–access any or all of the session recordings to hear the full conversations, or read on for five excerpts highlighting what patient advocates want you to know: 

  1. Recognizing and treating the whole person can ultimately impact compliance. 
    PlaquePsoriasis.com advocate Reena Ruparelia feels strongly about holistic health, and would like to see pharma encourage a blend of medication and lifestyle changes, which could help to reinforce that the industry is focusing on caring for the whole person.For Reena, holistic health also has an impact on treatment compliance; when incorporating lifestyle changes, such as diet and mindfulness, she cares for herself more and is less likely to skip medication. Living with psoriasis, or any chronic condition, is often a difficult journey, and finding the right treatment can be really helpful–but addressing more than just prescription medication can go a long way.

For the full article, visit health-union.com.

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Be in the room: Turning patient engagement to patient centricity

After three brushes with cancer, Robert Weker understands the patient journey more than many – and he is determined to put that experience to good use.

The avid blogger and passionate advocate retired from his R&D role four years ago to focus on his own health, and now works full time on making sure the patient voice is “in the room” when pharma companies make decisions.

Winding road

“My journey is a bit complicated, because I am a three-time cancer survivor,” said Robert, 60, who lives just outside of Philadelphia in the United States.

“First, it was testicular cancer, back in 1991, then I was diagnosed with liposarcoma in 2010, and pancreatic cancer in 2014.”

His first two cancers were discovered early, and, while he did experience treatment side effects, things were “manageable”, he said.

“My broad ambition is to get medicines and medical solutions that are more accessible, more affordable, and available in a more timely, more patient friendly way.”

“I don’t want to minimise cancer in any way, shape or form, but the testicular cancer was caught early, and the treatment success rate was well over 90%. It was more of an inconvenience. It was like entering a tunnel, but you could already see the light at the end.

“The liposarcoma was a little but different, but it was still caught pretty early and there was a high survival rate. Neither of the cancers significantly disrupted work.”

The pancreatic cancer, however, was a “completely different ball game”.

“At that time, the five-year survival rate was in the order of 7%. So, when I entered that tunnel, it was pretty dark.”

Robert decided to enrol on a clinical trial of chemotherapy plus high dose vitamin D at the University of Pennsylvania. Afterwards, he had to fight for the proton beam radiation therapy his doctor recommended as his insurance company considered it to be experimental.

“I was told my policy didn’t cover it. Even though my entire medical team was saying ‘we can’t do normal radiation because it’s too much exposure and his system as a whole won’t be able to handle it’.

“I am not a passive patient, so I kept calling and calling and finally they said there was a review panel,” said Robert, adding that he was shocked to find the panel did not include a radiation oncologist. “Even the voice of my doctor, who is probably one of the top three in his field worldwide, was being totally muted.”

After a number of reviews and appeals and countless phone calls, the treatment was finally approved but it had taken six weeks and many headaches.

Being in the room

His experiences have made him realise the importance of the patient voice being front and centre of healthcare decisions.

“I want to be in the room where it happens, where the decisions are made, so that it’s not just me, a passive patient sitting in the chemo suite,” he said.

“That means working with pharma companies to influence early decisions around R&D, and providing patient insights throughout the process.”

We also need to think about how other stakeholders – insurance companies, doctors, and hospitals, for example – fit into that, said Robert.

“My broad ambition is to get medicines and medical solutions that are more accessible, more affordable, and available in a more timely, more patient friendly way.”

Explaining why patients were an essential part of that mission, he said: “Everyone is driven by different goals and different stakeholders will have different drivers of what’s important.”

Levelling up

Robert, who works with several pharma companies on a variety of projects, believes that the industry is going in the right direction, but that there is still some way to go.

He described three levels of patient engagement, saying most organisations were currently “between one and two”.

“The first one is engaging the patient. That might call a patient or group of patients in to review a consent form or a trial protocol. They will ask them if it seems reasonable, feasible, or overly burdensome, then take those comments and say: ‘see you later’.”

The second level is more like a patient partnership, he said, using his position as member of one company’s Oncology Patient Council as an example.

“We meet once a month throughout the development cycle to provide feedback. We engage with them on challenges they might be facing, or on specific topics such as patient diversity. It’s a continuous process – a feedback loop – not a one-off interaction,” said Robert.

The next step is true patient centricity, which Robert described as “putting the patient at the centre of the wheel”.

“What that looks like in practice is ongoing involvement and engagement with the patient throughout,” he said, adding that it included making sure interventions were as easy to access geographically, logistically, and physically, as possible.

“It all goes back to this idea of being in the room. Of course, not all patients can be there, but if I can share the insights I have gained from my own journey, under the caveat that all patients are different, then at least they have heard what I have to say,” Robert concluded.


inspirePatient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 2 million patients and caregivers worldwide.

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Talking Medicines gets funding to expand AI-based “patient voice” platform

Talking Medicines has raised £1.1 million ($1.4 million) in funding that will be used to develop an artificial intelligence (AI) data platform that can be used by pharma companies to gain insights into how patients perceive them.

The Glasgow-based company – which is behind the MedSmart app that helps patients keep a digital records of their medicines and symptoms using barcodes – says it will use the cash injection to launch a new AI data platform which will “translate what patients are saying into actionable pharma grade intelligence.”

The platform could serve as an alternative to traditional patient focus groups, prescriber reports and clinical target patient profiles, and help drugmakers find out who is using their medicines, how they are finding the experience, and what they really think of brands.

The platform will mine information from social media and connected devices to regulated medicine information to capture and analyse the conversations and behaviours of medicine users and get a picture of “patient sentiment.”

The fundraising has been backed by Internet of Things (IoT) investment specialist Tern plc, along with The Scottish Investment Bank, Scottish Enterprise’s investment arm. To date Talking Medicines has raised £2.5 million, including £600,000 in grant funding from Scottish Enterprise last year.

Chief executive Jo Halliday (pictured centre) said the money would allow the company to hire an additional nine staff in its natural language processing (NLP) data tech team, which researches how machines can be made to understand human language accurately.

“Now more than ever we passionately believe that big pharma needs a systematic way to make data driven decisions through accessing high grade social intelligence driven from the patient,” said Halliday.

“This investment will scale our team and the development of…tools to translate what patients are saying into actionable pharma grade intelligence through our global patient confidence score by medicine.”

Tern chief executive Al Sisto is joining the board of the data specialist, and said Talking Medicines’ platform “is solving a critical problem for an industry that spends around $30 billion on marketing annually, whilst lacking systematic data tools that can structure patient sentiment from social channels.”

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Novartis Reports Results of Cosentyx (secukinumab) in P-IIIb ULTIMATE Study for Psoriatic Arthritis

Shots:

  • The P-IIIb ULTIMATE study involves assessing Cosentyx (300/150mg) vs PBO weekly for a mos. with treatment starting @4wks., followed by a once-a-month dose for the next 11mos. in 166 biologic-naïve patients in a ratio (1:1) with active PsA
  • The study reduction of synovitis @12wks. with an early improvement observed as 1wk., ACR20 (68% vs 34%); ACR50 (46% vs 9%,) & enthesitis (change in SPARCC, -2.4 vs -1.7); safety profile was consistent with previous studies @12wks.
  • Cosentyx is the first & only fully-human biologic that directly inhibits IL-17A. Novartis anticipates disclosing full 24-week data from the ongoing ULTIMATE trial at the EULAR in 2021 and final analysis at ACR 2021

Click here to­ read the full press release/ article | Ref: Novartis | Image: GMP News

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Boston Scientific’s Ranger DCB Receives the US FDA’s Approval for Peripheral Artery Disease in the SFA and PPA

Shots:

  • The approval is based on the RANGER II SFA pivotal study assessing the Ranger DCB vs standard PTA for the treatment in patients with PAD in the SFA & PPA
  • The study met its both 1EPs @12mos. i.e. MAE (94.1% vs 83.5%); lesion revascularization rate (5.5% vs 16.5%); Binary primary patency (82.9% vs 66.3%); primary patency by Kaplan-Meier estimate (89.8% vs 74.0%). The DCB has demonstrated 90% primary patency in COMPARE trial
  • The company expects to initiate a registry of the Ranger DCB and the Eluvia stent in the coming months to gather additional RWE and plans to launch the device in the US

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

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BMS’ Deucravacitinib (BMS-986165) Demonstrate Superiority Over Amgen’ Otezla (apremilast) in P-III POETYK PSO-1 Study for Plaque Psoriasis

Shots:

  • The P-III POETYK PSO-1 study involves assessing deucravacitinib (6mg, qd) vs PBO & Otezla (apremilast) in 666 patients with moderate to severe plaque psoriasis
  • The trial met its co-1EPs & 2EPs demonstrating deucravacitinib was superior to Otezla (apremilast) in the patients reaching a PASI 75 and sPGA 0/1 @16wks. The overall safety profile of deucravacitinib was consistent with previously reported P-II results
  • Deucravacitinib (BMS-986165, PO) is the first & only novel selective TYK2 inhibitor, currently being evaluated in psoriasis, PsA, lupus, and IBD

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

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Ring 20: Could the rare disease get left behind by next-generation gene sequencing?

Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet despite huge leaps forward in gene sequencing in recent years, diagnoses are going down instead of up. We spoke to Allison Watson, co-founder of Ring 20 Research and Support, about raising awareness, building the evidence base, and the importance of helping people through a pandemic despite limited funding.

Allison’s son David was just six when he started displaying the symptoms of (R)20, a genetic condition that can cause multiple, uncontrollable seizures, declines in cognition and mobility, and terrifying hallucinations.

As with many cases of (R)20, often called ring 20, David’s seizures appeared to come out of the blue.

“Unlike with many similar conditions, a child will develop normally until the seizures start to kick in,” said Allison.

“The child will often go backwards and lose abilities that they previously had. They may lose the ability to process properly or to speak. They might lose mobility and need a wheelchair. Others need help feeding.”

Ring 20 is a chromosome anomaly. Instead of being a pair that looks like two sticks, one or both arms of the 20th chromosome are joined at the ends, making them look like a ring under the microscope.

“A lack of awareness of the condition, which is known to affect at least 150 people worldwide, contributes to a diagnostic delay, said Allison, adding that children often experienced nocturnal, hallucinogenic seizures that healthcare professionals were simply not familiar with.”

The resulting seizures vary – in length, type, and severity – from person to person, but even mild forms of the disease have a huge impact on quality of life.

Typically, people have impaired awareness seizures that can last anywhere between 20 and 40 minutes. For some, this can go on for days, leaving them in hospital or even in an induced coma.

“My son, who is now 23, will have four to six non-convulsive events every day – that’s just normal.

“It’s like the lights are on but no one is home, so you have to be really careful. They could be doing something like cooking or having a bath. It really impedes their independence,” said Allison.

Non-existent treatments and diagnostic challenges

Despite the huge unmet need, ring 20 diagnosis is slow, and treatments are practically non-existent.

“Ring 20 doesn’t respond to any treatments: nothing seems to work. There are no clinical practice guidelines, meaning doctors treat on a case-by-case basis. They are effectively working blind,” said Allison, who co-founded the charity in 2014.

“Families just have to learn to live with these regular seizures and the associated comorbidities.”

A lack of awareness of the condition, which is known to affect at least 150 people worldwide, contributes to a diagnostic delay, said Allison, adding that children often experienced nocturnal, hallucinogenic seizures that healthcare professionals were simply not familiar with.

“A child may appear to awake from sleep. They may shake or shout out and can have a stiffness in their bodies or their arms. They may have a frightened expression on their face, and some describe horrific events. They see sharks swimming around their head, or fire in the room, or big black holes jumping up in front of them.

“This is often misdiagnosed and disregarded as night terrors, so this obviously denies diagnosis.”

Another, apparently contradictory, point is that the number of ring 20 diagnoses have actually declined with advances in genome sequencing, which cannot detect the presence of ring chromosomes.

Allison explained: “Human genome sequencing, exome sequencing, and even CGH array all look for additions, duplications, or something missing from the DNA. But in the majority of ring 20 cases, there is no misspelling in the chromosome.

“The way to diagnose it is to go back to the old-fashioned carrier type, to physically look at the cells under a microscope to see the rings.”

Additionally, doctors looking for a chromosome abnormality would typically send 30 cells for analysis. But the misshaped chromosomes that characterise ring 20 are only present in a certain proportion of the person’s cells.

Said Allison: “They need to be sending about 100 cells for analysis, otherwise they could miss the ring.

“We suspect that this particular disease is very underdiagnosed in people with genetic epilepsies or an undiagnosed cause for epilepsy, and we also suspect that people that do have a diagnosis may be potentially misdiagnosed.”

Raising awareness, offering support

That’s why Allison is so keen to build the evidence base and raise awareness of the condition.

As co-chair for EpiCARE, the European Reference Network for rare and complex epilepsies, she is working on projects ranging from a patient pathway map to e-learning modules for healthcare professionals.

And members of Ring 20 Research and Support have raised enough money to embark on a two-year natural history and biomarker study, as well as establish a patient registry.

“This is a fundamental building block to future research opportunities so we’re very proud of our families for getting us to this point,” she said, adding that the registry “would have been up and running by now if it wasn’t for COVID”.

The pandemic caused both a surge in demand and a drop-in financial support – a problem that has been seen across the patient support sector.

But with it being more important than ever to ensure people with ring 20 and their families get the support they need, so COVID or no-COVID, Allison and her team have no intention of scaling back their plans. In fact, they have even extended their offering to include online peer networking.

“Because our families are so disparately located, some may never get to meet another family with ring 20 at all. So, we have been having fortnightly Zoom calls. We’re connecting families from the US and Australia, the UK and Europe, even South Africa,” said Allison.

“We’re based in the UK, but we support families worldwide. We’re the only organization doing this,” said Allison.

 


Cambridge Rare Disease Network Exploring Rare Diseases is produced in partnership with Cambridge Rare Disease Network (CRDN). CRDN is building a vibrant network of patients and stakeholders to share knowledge and foster innovation that leads to better diagnosis, treatment and support for those living with a rare disease

The post Ring 20: Could the rare disease get left behind by next-generation gene sequencing? appeared first on .

Fostering Confidence in Newly Diagnosed Autoimmune Patients

When a person is newly diagnosed with an autoimmune condition, such as psoriasis or rheumatoid arthritis, it’s not uncommon for us to hear from them in our online health communities discussing fear or uncertainty, and seeking connection or support. The myriad of difficult decisions they are making around treatment options may foster a sense of self-doubt, and the newly diagnosed person may feel overwhelmed by decision fatigue. How can the healthcare industry better reach newly diagnosed patients and help build confidence in their own decision making?

Recent survey data from Health Union revealed interesting insights into the attitudes and behaviors of 8,289 people diagnosed with various autoimmune conditions.* Since 2010, our syndicated In America surveys have captured patient-reported health data from tens of thousands of people living with chronic health conditions (and their care partners), so we can better support them in our online communities and connect pharma with critical perspectives it may be missing. In this specific analysis, the data showed that patients diagnosed less than two years ago report being less active in treatment decisions, as well as being less confident in their condition management. Additionally, patients diagnosed less than two years ago were more likely to say they generally do what the doctor tells them without asking questions.**

Read the full article on health-union.com.

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Tetra Therapeutics Reports Positive Results of BPN14770 in P-II Study for Fragile X Syndrome

Shots:

  • The P-II study is a two-way crossover study assessing BPN14770 (25mg, bid) vs PBO in 30 adult male patients aged 18-45yrs. with FXS due to >200 CGG repeats in the FMR1 gene
  • The study demonstrated benefits in oral reading recognition (+2.80), picture vocabulary (+5.79), cognition crystallized composite score (+5.29), benefits were maintained up to 12wks. and was well tolerated
  • BPN14770 is a novel therapy that selectively inhibits PDE4D to increase the levels of cAMP. In preclinical trials, it promotes the maturation of connections between neurons, which is impaired in patients with FXS and has received the US FDA’s ODD as well as approved for investigational use in the US

Click here to­ read the full press release/ article | Ref: Businesswire | Image: Tetra Therapeutics

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Regeneron Pauses Dosing of Garetosmab (REGN2477) in P-II LUMINA-1 Study for Fibrodysplasia Ossificans Progressiva

Shots:

  • Regeneron pauses dosing of the garetosmab (REGN2477) in the ongoing P-II LUMINA-1 study in patients with FOP. The pause is due to fatal serious AEs in the study during the open-label extension during which all patients received active treatment
  • Regeneron shared the update with the trial’s IDMC and regulatory authorities and will conduct a review of the study to better understand the benefit/risk profile of garetosmab in people with FOP
  • Garetosmab is a VelocImmune-derived mAb that binds and neutralizes Activin A, which is involved in the development of heterotopic bone in people with FOP

Click here to­ read the full press release/ article | Ref: Regeneron | Image: The Print

The post Regeneron Pauses Dosing of Garetosmab (REGN2477) in P-II LUMINA-1 Study for Fibrodysplasia Ossificans Progressiva first appeared on PharmaShots.

UCB Reports Results of Bimekizumab in P-III BE SURE Study for Moderate-to-Severe Psoriasis

Shots:

  • The P-III BE SURE study involves assessing bimekizumab vs Humira (adalimumab) for 24wks. is followed until 56wks. in 478 adult patients with chronic PsO for at least 6 mos. before screening & with an affected body surface area of at least 10%, PASI of at least 12 & IGA score equal to or > 3 on a 5point scale
  • Results: The study met its 1EPs & 2EPs, @16wks. PASI 90 (86.2% vs 47.2%); IGA 0/1 (85.3 % vs 57.2%); PASI 100 (60.8% vs 23.9%); PASI 100 @24wks. (66.8% vs 29.6%); PASI 90, PASI 100 and IGA 0/1 response rates were maintained through 1year with both q4w & q8w; skin clearance rates increased in patients who switched from adalimumab to bimekizumab @24wks., with response rates @56wks. comparable to patients treated with bimekizumab throughout the study
  • Bimekizumab is an investigational humanized IgG1 mAb that selectively inhibits both IL-17A & IL-17F

Click here to­ read the full press release/ article | Ref: PRNewswire | Image: Pharma Journalist

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AbbVie Reports Results of SKYRIZI (risankizumab) in P-III LIMMitless Study in Patients with Moderate to Severe Plaque Psoriasis

Shots:

  • The P-III LIMMitless study is designed to evaluate the long-term safety & efficacy assessing risankizumab (150 mg q12wks.) continuous risankizumab with a loading dose in adults with moderate to severe plaque psoriasis. The analysis includes integrated data from five P-II & III studies (ultIMMa-1, ultIMMa-2, SustaIMM, IMMvent and NCT03255382) and the LIMMitless study
  • Results: ~63% of patients with moderate to severe plaque psoriasis treated with SKYRIZI achieved completely clear skin for 172 wks., as measured by 100% improvement from baseline in the PASI 100. New results from the P-III LIMMitless study were presented at the 29th EADV Virtual Congress
  • Risankizumab is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. It is part of a collaboration between Boehringer Ingelheim & AbbVie, with AbbVie leading development & commercialisation globally

Click here to­ read the full press release/ article | Ref: Abbvie | Image: Seeking Alpha

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Takeda to partner on patient-centric data with Seqster

Takeda has partnered with patient data firm Seqster in a drive to improve care through better access and understanding of patient-level data.

The partnership follows Takeda’s investment in the US startup earlier this year. The pharma firm said it wants to leverage the company’s technology across its business

San Diego-based Seqster has developed a portal that gathers together a patient’s data – such as electronic health records (EHR), genetic information, fitness results from wearables etc – and keeps it in a secure format that gives control over its collection, ownership and sharing.

Takeda hopes Seqster can help expand its external data and digital collaboration ecosystem, with better access to real-world evidence, and generate powerful data and insights for research and patient services. 

The company says it wants to activate 12 distinct use cases across our business “in a matter of weeks”.

One use of Seqster’s decision support system and research platform is to reduce the time for consenting and onboarding patient data during clinical trials. The aim is to enhance patient engagement and compliance through a single-entry point for EHRs and integrates with partners enterprise data backbones.    

At the time of Takeda’s investment in Seqster, Bruce Meadows, head of investments at Takeda Digital Ventures, said it was “a cornerstone of our digital health strategy,” and that the key element is “interoperability” for health data sharing.

Seqster’s platform “addresses interoperability on not only a nationwide scale but also globally,” according to Meadows, who notes that interoperability “is one of the biggest barriers to applying precision medicine to clinical trials and patient engagement.”

It is also a key objective for the Centers for Medicare & Medicaid Services (CMS) and Office of the National Coordinator for Health Information Technology (ONC) in the US.

The two agencies have a pair of proposed rules on interoperability and patient access to health information under review at the Office of Management and Budget (OMB) that could come into effect later this year.

A key part of those proposals is to allow patients easy, electronic access to their personal health information at no cost.

The Seqster Research Portal (SRP) can be used to speed up recruitment into clinical trials, as well as the consent process, and can work with a broad range of study types including patient registries used to generate real-world evidence, says its developer.

“Seqster provides clinical trial participants a secure platform to consent and share their data with investigators and study personnel in real-time,” according to the company’s chief executive Ardy Arianpour.

In turn, that creates “a longitudinal health record that facilitates patient clinical trial onboarding, monitoring and post-trial follow-ups,” he adds.

Seqster says the SRP platform currently connects users to more than 3,000 healthcare providers and over 100,000 hospitals and clinics across the US.

Listen to our podcast interview with Ardy Arianpour here.

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Why 72% of patients don’t feel listened to by pharma

Claus Møldrup explores research showing that patients want their voice to be heard more by pharma, and asks what a truly effective feedback loop for medicines might look like.

The democratisation of data opens up improvements to virtually every product and service across virtually every sector — bar one. Pharma remains stubbornly closed to feedback, with potentially damaging consequences for the industry, healthcare systems and patients.

The most obvious iteration of democratisation is the rise of online experience sharing platforms. From highly specific sites such as Trip Advisor to all-encompassing platforms such as Trustpilot, the modern consumer’s voice carries weight. While negative reviews can sink a business, forward-thinking organisations see this accountability as a driver for continuous improvement.

The omission of medicines from the feedback revolution is curious and potentially damaging. Data defines modern therapeutics, but this is almost uniquely confined to the clinical trial stages of drug development. Identifying, recruiting, retaining and monitoring patients during this part of the cycle is estimated to cost pharma US$19 billion a year as companies seek to harvest patient experiences.

Once a drug is licensed and launched, however, there is a deafening silence. Millions of patients are prescribed often complex drugs and are expected to follow equally complicated regimens. While clinical outcomes are of course monitored through healthcare systems, the patient’s experiences, such as the reality of their drug administration and day-to-day effects, are ignored by the very companies that developed — and continue to develop — them.

“As most other sectors have long recognised, if a business isn’t taking part in the conversation then others will fill the void to tell their story instead. That narrative, for many patients, is that pharma companies just don’t care”

Pharma is swimming against the tide and can’t pretend that the democratisation of medicines data doesn’t exist — because it’s already here. As most other sectors have long recognised, if a business isn’t taking part in the conversation then others will fill the void to tell their story instead. It means pharma has lost control of its own narrative – and that narrative, for many patients, is that pharma companies just don’t care.

Research supports this conclusion, showing an alarming lack of patient trust. A 2020 survey from DrugsDisclosed.com of 3,346 users of prescription and over the counter (OTC) medicines from the UK and the Nordics revealed that:

  • more than three-quarters of patients do not trust advice from pharmaceutical companies about their medication;
  • 81% feel the pharmaceutical industry influences prescription decisions; and
  • 72% do not feel listened to by pharmaceutical companies.

These are startling statistics, and this overwhelmingly negative perception must be of concern. Pharma is an industry that should have a hugely positive story to tell as it develops the medicines and treatments that bring benefits to billions of people around the world. Yet it is widely mistrusted.

If pharma companies listened more, they would hear that the users of their medicines often feel confused and alone. In direct opposition to the emerging era of personalised healthcare, medicine information leaflets are one-size-fits-all documents that fail to reflect a patient’s individual circumstances and experiences, and are obligated to list every possible side-effect (which can make them virtually meaningless). In addition, patients have no opportunity whatsoever to feed back or pose questions to the company or — as critically — other users.

As one Crohn’s Disease patient describes in a DrugsDisclosed user case study, “It can be difficult getting useful and reliable information on your medication, and leaflets that come with them can be scary. They have to list all of the possible side effects, even the rarest. It is reassuring to read about other people’s real-life experience… It is important because it is by the people that actually take the medications. It makes me feel less alone.”

Shared experience — the power to improve

There is a better way. Healthcare (as distinct from pharma) has evolved with society and its technologies. Go back just a few decades and patients were passive bystanders to their own healthcare. Medicine took a patrician approach: it knew best and patients should lie still whilst they were poked, prodded and prescribed until they (hopefully) got better. This has changed — and changed for good.

Many healthcare systems are well advanced in listening to patients. In the UK, for example, every NHS hospital has a ‘patient panel’ made up of service users. These panels provide real-time feedback on experiences, from food to hygiene to clinical care, and members often sit on the main hospital Board to ensure that actions are informed by patient insight. This isn’t a PR exercise — it improves hospital environments, focuses decision-making and ultimately benefits clinical outcomes.

We are now seeing this evolve further as a new and distinct discipline emerges, known as Public Engagement (PE). PE isn’t traditional ‘communications’ where organisations talk to try and change patient behaviours, rather it’s about listening to patients to change corporate behaviours.

This evolution is particularly prevalent in emerging medicines, with organisations such as Genomics England and The Wellcome Trust supported by Understanding Patient Data recognising the huge and largely untapped value of patient experience — and that the success of new forms of medicine depends on public understanding, acceptance and consented participation.

The way back

Some of these issues fall out of well-intentioned codes and regulations to protect patients, but this can no longer be cited as an excuse for pharma’s continuing lack of engagement with its ultimate end users.

Technologies — specifically a sophisticated form of ‘Trustpilot for medicines’ — exist to bridge the gap between pharma and patients responsibly. Using this kind of technology will see significant improvements to healthcare generally and medicines specifically.

Research again backs up this conclusion, with a study that reviewed patients who had used an anonymised — regulatory compliant — medicines feedback app. After two months:

  • 79% experienced an improvement in remembering to take their medications;
  • three quarters experienced an improvement in taking their medications as prescribed;
  • close to half felt they better understood their medications;
  • 69% felt more motivated to take their medications; while
  • more than a third felt the effect of their medication actually improved.

In calling for further research, another study concluded that use of these apps improved clinically relevant indicators of adherence and impact and benefits were related to level of app usage.

Asking patients how they feel about medications can also benefit wider healthcare systems. In the UK, for example, more than half the public expressed an unwillingness to take a vaccine unless it had been tested for at least a year. Further, close to three quarters would be unwilling to allow their children to receive such a vaccine. This is priceless public insight during a pandemic as pharma races to find a COVID-19 vaccine — allowing the industry to better support governments as they prepare populations should a vaccine become available.

Beyond improvements to patient well-being, health outcomes and pandemic response, listening to patients can also improve pharma’s business operations. Understanding, for example, the factors that heighten the risk of poor adherence to medication regimens can lead to solutions designed to mitigate them — and reduce patient drop out. Further, with insight direct from users, pharma companies can optimise their Patient Support Programmes and patient support materials, while they also have access to a continuous, near real-time market research resource made up of hundreds of thousands of highly engaged patients willing to share experiences.

From bad medicine to good

The democratisation of data is here, and it’s here to stay. Pharma’s longstanding reluctance to join the conversation simply isn’t sustainable. The choice is whether the industry engages quickly and willingly or continues to resist and is dragged into the 21st century under duress. The former will go a long way to restore patient trust, while the latter will further undermine it.

Pharma needs to capture deep patient insight into its medications, which it can apply from product development to marketing to patient support. At the same time, this will allow users to feel empowered in helping to shape a medicine environment that reflects, recognises and responds to their daily experiences.

The tragedy is that a continuing failure to join the conversation hurts pharma companies, health systems and millions of medicines users around the world. Now is the time for pharma to join the feedback revolution and turn bad medicine to good.

About the author

Claus Møldrup is co-founder of DrugsDisclosed.com and CEO of DrugStars.

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Sanofi Presents Results of Olipudase Alfa in Two Clinical Studies at ASHG 2020

Shots:

  • The P-II/III ASCEND trial involves assessing olipudase alfa 3 mg/kg (IV, q2w) vs PBO in 36 adult patients with ASMD type B to evaluate treatment impact on pulmonary function & spleen & liver volume for 52wks. The study demonstrated improvement in lung function (22% vs 3%), reduction in spleen volume (39.5% vs 0.5%), reduction in liver volume (31.7% vs 1.4%); improvement in platelet counts (16.8% vs 2.5%)
  • The P-II ASCEND-Peds trial involves assessing olipudase alfa 3 mg/kg (IV) q2w for 64 wks, in 20 pediatric patients with ASMD without acute or rapidly progressive neurological abnormalities. The study demonstrated 33% increment in predicted DLCO, reduction in spleen volume & liver volume (49% & 41%); 34% increment in platelet count
  • Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin, currently being investigated to treat non-CNS manifestations of ASMD. The therapy has received FDA’s BT designation, EMA’s PRIME Designation and MHLW’s SAKIGAKE designation

Click here ­to­ read full press release/ article | Ref: Sanofi | Image: Evaluate Pharma

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Samsung Bioepis Reports Results of Renflexis (biosimilar, infliximab) from Two Studies in IBD Patients

Shots:

  • The company reported results from two real-world studies of Renflexis (infliximab-abda) in patients with IBD registered in the US Veteran Affairs Healthcare System database. Data were presented at ACG 2020
  • One study assesses the safety of switching from reference infliximab or infliximab-dyyb to Renflexis in patients with IBD identified from the VAHS database and demonstrated an 83% continuation rate over 1yr. with similar continuation rate in patients who underwent a single & double switch from reference infliximab
  • Second study focused on the real-world utilization pattern of infliximab products for IBD, within the context of VANF policy. The study found that Renflexis became the preferred infliximab product on VANF in Sept’2018 which was faster than the adoption of the previous biosimilar (infliximab-dyyb), listed on VANF in May’2017

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

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Pharma 2030: Will payers fund more preventive care programmes?

Preventive care programmes are key to better health outcomes, but several factors are limiting their uptake. A new analysis looks at these barriers and asks how they could be overcome.

Over the past decade, many healthcare systems have worked to expand their focus on health maintenance and disease prevention as several studies have indicated that preventive care services can play a significant role in improving general health while helping reduce long term healthcare costs.

Strategies include different options focused on health maintenance, such as vaccination programmes, smoking cessation services, support for physical activity, and access to technologies that support early detection of diseases. In addition to representing more efficient and cost-effective options in healthcare coverage, these measures offer the prospect of positive health outcomes and quality of life for patients.

While the benefits of preventive measures are established, integration of many options can be limited when payers do not agree on what are considered adequate or optimal levels of preventive care.

This is especially true when preventive care involves a direct cost to payers. CRA’s Life Sciences Practice recently conducted an analysis of the barriers that currently limit reimbursement for many preventive care services and how the evolution of healthcare systems and preventive care offerings could change the demand and uptake of preventive care by 2030.

Understanding payer views of preventive care

The analysis found that a range of factors including significant upfront costs, limited allocation of healthcare budgets to cover preventive care, and the focus on short-term or quarterly cost savings, rather than longer term strategies in managing care, are limiting adoption of preventive care programmes among payers.

From both a business and a societal perspective, in many cases payers consider preventive care as sub-optimal because it is positioned to deliver clinical and economic benefits over a longer timeframe – often years or decades.

Many other industry stakeholders, including medical organisations and government agencies, consider preventive care to be a good investment. They cite preventive care programmes as critical for improving the general health of a population and addressing many major global health issues effectively. These stakeholders also are in position to benefit from strategies that will deliver net reductions in healthcare costs over the long term.

For example, according to the US Centers for Disease Control and Prevention (CDC), “chronic diseases that are avoidable through preventive care services account for 75% of the nation’s healthcare spending and lower economic output in the US by $260 billion a year. If everyone in the country received recommended clinical care, then the healthcare system could save over 100,000 lives a year.”

CRA’s findings also show that if decisions on the adoption of preventive care services are left to individual stakeholders such as payers who focus mainly on short-term cost effectiveness, use of these measures may be limited.

This limitation is also reflected in the fact that, on average, countries that are part of the Organisation for Economic Co-operation and Development (OECD) did not increase the percentage of their healthcare budgets allocated to preventive care between 2010 and 2017 (2.8%), despite the need for many of these countries to adopt measures to control healthcare spending (Figure 1).

 

Figure 1: OECD average of public spending on health (Source: OECD)

Independent global advisors are advocating that the preventive healthcare model must operate beyond the limitations of short-term cost assessments favored by many political and healthcare decision-makers.

The reason is based on both economic and societal benefits. If preventive measures that can deliver cost benefits and improved health over the longer term are not implemented, healthcare systems risk a continued escalation in demand for care which, in turn, may be more costly than implementation of preventive measures in a timely manner.

The fact that payers must allocate resources based on limited annual budgets is another factor leading them to deprioritise many preventive care measures, especially those that require significant upfront investment. Preventive care typically targets large patient populations and has a considerable impact on budgets, even though the cost per patient will often be relatively low. Payers also often have difficulties forecasting the cost-effectiveness of preventive care and thus can be uncertain about the true budget impact.

Even in cases where payers allocate resources to preventive care services, research shows that many may not be allocating those funds as effectively as possible. Misallocation of funding is often the result of payers trying to meet patients’ expectations on services that will be covered rather than focusing on the most efficient approach. On average, nearly half of all preventive care spending is allocated to patient monitoring (e.g. health or dental check-ups), rather than to support immunisation and early disease detection, which have been shown to be more effective.

There are also unique challenges in adoption of many preventive care services in multi-payer systems. As patients move across payers, the economic benefits from an investment in preventive care will likely be realised by a different payer than the one who originally funded the preventive service or programme.

To address any gaps in healthcare funding, some payers are prioritising preventive care measures that deliver nearer-term cost savings rather than cost effectiveness. For example, offering the seasonal influenza vaccine to healthy working-age adults is a cost-saving preventive measure (generating net savings of $68.96 per person in the US) and is widely adopted.

In contrast, vascular disease health checks are a cost-effective preventive measure for the broad population, but access is often limited to only high-risk patients.

“Payers may better recognise the value of preventive care as services become more targeted. With the increased availability of new technologies, including wearable devices, health apps, and genetic testing, preventive care is rapidly improving”

How could payer views change by 2030?

By 2030, there will likely be more data available demonstrating the longer-term benefits of preventive care, suggesting that payers may have fewer concerns over cost effectiveness. Some payers may also come under increased societal and governmental pressure to allocate more funding to preventive care services. For example, government advisors are proposing that Nordic countries should allocate equal amounts to sick care and preventive care (5% of GDP to each) by 2030.

Currently, 3% of global healthcare budgets are allocated to preventive services but a recent analysis projects that this will increase to 9% by 2030 (Figure 2).

This shift in fund allocation is also supported by the fact that integration of preventive care measures could be considered the more cost-effective option as more innovative, targeted (and potentially curative) treatments are developed and become commercially available. As a result, healthcare decision-makers, including payers, are expected to push for broader adoption of preventive care programmes that could delay or eliminate the onset of disease and the associated need for treatment among high-risk patients.

 

Figure 2: How the allocation of healthcare budgets is expected to change by 2030

Payers may also better recognise the value of preventive care over time as services become more targeted. With the increased availability of new technologies, including wearable devices, health apps, and genetic testing, preventive care is rapidly improving.

In one example, genetic testing would allow patients with a high risk of developing some diseases to receive more targeted preventive care including routine screening and biomarker checks. This is currently the case for women with a variant of the breast cancer (BRCA) gene, which greatly increases their chance of developing breast and ovarian cancers and is readily identifiable through testing.

With the development of more targeted and effective preventive solutions, increasing payer willingness to cover them, and growing demand for these services among patients, many healthcare stakeholders expect that both access and uptake will expand and evolve significantly in future. This shift toward broader adoption of preventive care services can support improved sustainability of healthcare budgets and presents many new and more effective opportunities to improve the health and outcomes of patients around the world.

 

About the authors

Michele Pistollato is an associate principal and Elaine Damato is a consulting associate in the Life Sciences Practice at CRA, based in the London office. Rajini Jayasuriya is a senior associate in the Life Sciences Practice at CRA, based in the Washington, DC office. Views expressed herein are the authors’ and not those of CRA or any of the organisations with which the authors are affiliated. The authors wish to acknowledge the contributions of Lev Gerlovin and Neil Turner to this article.

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Foundation Medicine’s FoundationOne CDx Receives the US FDA’s Approval as a CDx for Vitrakvi (larotrectinib)

Shots:

  • The US FDA approved FoundationOneCDx to be used as a CDx for Vitrakvi (larotrectinib) to identify patients with NTRK fusions across all solid tumors. The genomic test is currently approved as a CDx for 20+ therapies
  • The FoundationOne CDx is the tissue-based CGP test approved to detect NTRK1/2/3 fusions across all solid tumor types and identify patients who may be appropriate for treatment with Vitrakvi
  • The approval of Vitrakvi was based on three studies including LOXO-TRK-14001,  SCOUT, and NAVIGATE studies and is indicated for the treatment of adult and pediatric patients with solid tumors that have NTRK gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment

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

The post Foundation Medicine’s FoundationOne CDx Receives the US FDA’s Approval as a CDx for Vitrakvi (larotrectinib) first appeared on PharmaShots.

Toxic positivity and grief: The reality of living through cancer

Cancer doesn’t end when treatment stops – it’s a lifelong journey and people need support throughout, says patient advocate Megan-Claire Chase.

No one gets through cancer unscathed. People are usually left with a mixture of treatment aftereffects, toxic positivity, and lingering anger to deal with.

Megan-Claire Chase, a fierce patient advocate who has been blogging about her experiences since she was diagnosed with breast cancer in 2015, said the disease never went away.

“Sometimes, people feel that once you finish your treatment, you are done, and you should stop talking about it. But really, it has only just begun. It is a lifelong journey.

“We’re going to need to have scans forever and we’ve been traumatised by the whole experience, so it’s hurtful when your family members and your friends think you should just move past it.”

Megan-Claire underwent 16 rounds of chemotherapy, nine operations, and 33 radiation treatments after developing invasive lobular breast cancer in her  30s.

As a result of the treatment, she is infertile, has chemo-induced fibromyalgia and neuropathy in her hands and feet. She goes to the cancer center for a diagnostic mammogram and rotates between getting an MRI and ultrasound every six months for 10 years.

“You know that people have good intentions and they are coming from a good place, but when your family and friends just want to see you smiling all the time, it can feel like you’re putting on an act”

Quality of life after cancer

There are a lot of quality of life issues, many of which were not understood outside of the cancer community, says Megan-Claire.

She says: “Sometimes I have to walk with a cane. Because of the neuropathy on my feet, I can’t walk very long distances because the numbness goes up my leg and I fall over and hurt myself.

“That’s what I want people to understand. Some people are on active treatment forever. For others, that treatment has a stopping point, but even then, there are all these other issues that can stem from the poison that goes into your body.”

News stories about cancer survivors going on to achieve great feats of endurance do nothing to help the public perception of what it means to live with the long-term effects of the disease and its treatments.

“Some people are able to climb mountains and do walks and all of that physical stuff. But for others that ability was taken away and it feels like yet another loss,” says Megan-Claire:

“We’re constantly grieving – grieving the loss of the body parts that are missing, the physical abilities we had, and, for many of us, grieving the jobs we used to have before we had to stop working.

“We also have to grieve the friends we have made and lost along the way. We’re constantly reminded of our mortality and have to live with the fear that it could come back at any time.”

Releasing the negative

Through her blog, Life on the Cancer Train, and her advocacy work, Megan-Claire meets many young people in a similar position to her. She believes it helps to give them the space to own their feelings and share their experiences with others who truly understand the nuances of life after cancer.

“When I write, I write what is on my mind, but I always find that resonates with people. A lot of people are hurting, and I think it helps that I post about the elephants in the room: the mental health issues, the anger, the post-traumatic stress disorder,” says Megan-Claire. “People need a place to release all that.”

‘Toxic positivity’ is another issue people who have been through cancer treatment commonly face and dealing with it head on can be extremely difficult, Megan-Claire explains.

“It’s impossible to be this tower of strength all the time. A lot of us get annoyed when we are told: ‘Oh, you’re so brave’, or ‘I don’t know how you do it’. When that happens, all I’m thinking is ‘Do I want to live or do I want to die. It’s not a fair choice.’

“You know that people have good intentions and they are coming from a good place, but when your family and friends just want to see you smiling all the time, it can feel like you’re putting on an act.”

Peer support

What people need, she went on, was a place where they could “take off the mask and just be vulnerable”. And that’s where peer support comes in.

“Cancer support groups are a safe place where people can meet and talk about their experiences with no judgement.“” says Megan-Claire, who belongs to several young adult cancer groups online.

“My advice to people is always: if you’re angry, be angry. If you’re sad, be sad. Just don’t wallow in it forever. We need to feel these emotions so we can move through them and then do what it is we need to do to move forward with our lives.”

To read Megan-Claire’s blog, click here. See our last interview with Megan-Claire here.


inspirePatient Insights is a monthly series that appears in partnership with Inspire, a company with an online support community of more than 1.5 million patients and caregivers worldwide.

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Jazz Reports Results of Xywav (calcium, magnesium, potassium, and sodium oxybates) in P-III Study for Cataplexy or EDS in Patients with Narcolepsy

Shots:

  • The P-III study involves assessing Xywav vs PBO in patients aged ≥ 7yrs. with cataplexy or EDS with narcolepsy
  • Results: study met its 1EPs & 2EPs i.e. differences in median change in a weekly number of cataplexy attacks and ESS scores. Results were published at World Sleep 2019
  • Xywav is an oxybate product with a unique composition of cations resulting in 92% less sodium or ~1,000- 1,500 mg/night less than sodium oxybate at the recommended dosage range of 6-9gms. The company expects to launch in Q4’20, following RESM implementation

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

The post Jazz Reports Results of Xywav (calcium, magnesium, potassium, and sodium oxybates) in P-III Study for Cataplexy or EDS in Patients with Narcolepsy first appeared on PharmaShots.

What HCPs Think about schools reopening

In our latest article in the What HCPs Think series, CREATION.co’s Jamie Doggett analyses the social media activity of doctors to determine their views on how schools should operate during COVID-19.

When and how schools should return was a big question for the UK during summer, and one that is continuing to go on.

A modelling study released in the Lancet, concluded that the best strategy for schools to prevent a second wave required the “large-scale, population-wide testing of symptomatic individuals and effective tracing of their contacts, followed by isolation of diagnosed individuals”.

The use of social media has increased for healthcare professionals (HCPs) during the pandemic as they seek to learn, give opinions and share resources and guidelines. By analysing the unprompted conversations of HCPs on public social media, it can give us a clearer understanding on their views about schools reopening.

Differentiating common cold symptoms

When publishing a social media post, many HCPs add links to stories they wish to share or discuss. The most shared link by HCPs related to the reopening of schools was a resource from The Royal College of Paediatrics and Child Health website. The page provided a summary of current policy and other guidance. 134 healthcare professionals shared this summary comparing and contrasting common cold and COVID-19 symptoms. HCPs were seeking to show their peers and the public the advice on when children should get tested.

A key voice of influence in the UK HCP conversation was Devi Lalita Sridhar, a Professor and Chair of Global Public Health at the University of Edinburgh. Her Twitter account was highly retweeted by HCP peers, on topics linked to school openings. In one post she added her thoughts on the importance of testing as she called for a high bar in availability and turnaround time for testing in order for school returns to be viable.

Various emotions discussed by UK HCPs

Being sure that the UK’s testing infrastructure was prepared for the return to school was a common point in HCP conversation. Other concerns emerged leading up to the first day back.

Emotions discussed by HCPs relating to UK schools reopening during COVID-19

HCPs discussed various emotions relating to school returns, from general worries to panic – but there was also hope. Dr Matthew Snape, writing for the Guardian, published an article broadcasting this hope entitled “There is now clear data on Covid-19 and children: it should be safe to reopen English schools”.

HCPs weigh up the pros and cons of reopening schools

Following Dr Snape’s piece supporting the reopening of schools, Professor Chris Whitty, on behalf of the government, said “missing school is worse for children than the virus”. The BBC article with a video of Whitty’s advice was the most shared news story (40 shares) by UK HCPs relating to the schools. The idea that missing lessons “damages children in the long run” being a bigger factor than the “incredibly small… chances of children dying from COVID-19” was met with mixed feelings from HCPs.

Sarah Jane Kipps, a London-based nurse, emphasised the role school nurses will play during the reopening and was extremely positive about starting her specialist community public health nursing course.

A little more caution was demonstrated by Chris Roseveare, a consultant physician in Hampshire, who, while not doubting Witty’s statement speculated on the possibility of a spike of infections and warned hospitals should prepare for such an eventuality.

HCPs have discussed many benefits that opening schools, colleges and universities will bring but have also raised concerns, especially connected to safety. The two safety aspects that were most actively discussed were the wearing of masks and how social distancing would be maintained.

Topics of HCP online conversations relating to UK schools reopening during COVID-19

A Public Health registrar in Birmingham argued that evidence shows there is no harm in wearing face masks so “Why would you not support mask wearing [in schools] if it might save a child’s life”? But others responded they were yet to be “convinced of the benefits of masks”. Key issues such as masks are still dividing HCP and public opinion, but on open social media channels HCPs are sharing what scientific evidence they can with each other globally to address the unknowns.

HCP influencers share educational resources

The most active UK HCP was Sharon White, the CEO of the School & Public Health Nurses Association. She often looked to use her online influence to share resources including infographics, webinars and advice from organisations. Sharing resources to help prepare the general public, especially parents and carers, was a common behaviour among HCPs.

Another key resource that resonated with HCPs (shared 44 times) was the Going back to school guide published on the Children’s Commissioner website. This guide was designed for children to inform them about the possible changes when going back to school and gave advice for those who are worried or nervous.

Most pupils return to school

Initial figures indicated that 88% of pupils returned for the start of term – lower than previous years. However, the evidence is not conclusive as to whether this was due to COVID-19 outbreaks or fears. As children and teachers return to school there has been an increase in demand for tests, COVID-19 cases and the R number. HCPs continue to share their best advice using public social media, playing an important part in supporting the public, and their peers, to see an eventual end to the disease.

About the author

Jamie DoggettAs head of insight with CREATION.co, Jamie Doggett leads a team that discovers what healthcare professionals think by analysing their online social media conversations. Jamie has collaborated with healthcare professionals, marketers and communicators to leverage data for health strategy, and has pioneered new research methodologies using CREATION.co’s global dataset. Research produced by Jamie and his team has informed academic articles, health policy, and product launches all over the world.

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Sanofi’s Sarclisa (isatuximab) Receives NICE Recommendation for Patients with Multiple Myeloma

Shots:

  • The NICE has issued FAD which is based on the P-III ICARIA-MM trial assessing isatuximab + pom-dex vs pom-dex in patients prior treated with 3L treatment and at least 2L therapies including lenalidomide and a proteasome inhibitor with RRMM in 307 patients with RRMM
  • The study demonstrated that the combination regimen demonstrated a reduction in risk of disease progression or death in adults by 40%, mPFS (11.5 vs 6.5), well-tolerated with no increase in treatment discontinuation
  • Isatuximab is a mAb that binds to a specific site on CD38 and is now available to eligible patients through the NHS Cancer Drugs Fund. Isatuximab is the first mAb approved in EU to be used in combination with pom-dex for RRMM

Click here ­to­ read full press release/ article | Ref: Sanofi | Image: Bloomberg

The post Sanofi’s Sarclisa (isatuximab) Receives NICE Recommendation for Patients with Multiple Myeloma first appeared on PharmaShots.

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Relapsed or Refractory Classical Hodgkin Lymphoma

Shots:

  • The approval is based on P-III KEYNOTE-204 study assessing Keytruda (200mg, IV, q3w vs BV (1.8 mg/kg, IV, q3w) in 304 patients in a ratio (1:1) with r/r cHL after at least one multi-agent CT regimen
  • Results: reduction in the risk of disease progression or death by 35%, median PFS (13.2mos. vs 3mos); ORR (66% vs 54%); DOR (20.7 mos. vs 13.8mos.). The approval is reviewed under the FDA’s Project Orbis
  • Ketruda is a humanized mAb that blocks the interaction between PD-1 and its ligands, PD-L1 & PD-L2 thus activating T lymphocytes that affect tumor cells & healthy cells. Additionally, the US FDA has approved an updated pediatric indication for refractory cHL or cHL that has relapsed after two or more lines of therapy

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

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Sorrento to Initiate P-II Study of STI-5656 (abivertinib maleate) for COVID-19 in Brazil

Shots:

  • The P-II study will evaluate STI-5656 in ~400 patients hospitalized due to COVID-19 having mild, moderate, and severe symptoms. The P-II clinical trials of abivertinib now cleared to proceed in both Brazil and the US
  • The dose to be tested is the same as in the US P-II trial but the trial protocol in Brazil includes patients at earlier stages of the disease, with a drug administration regimen of 7 days (versus 14 days for more advanced patients in the US)
  • Both the studies are complementary and address both dose duration and disease stage

    Click here to­ read the full press release/ article | Ref: Sorrento Image: Newsmax

The post Sorrento to Initiate P-II Study of STI-5656 (abivertinib maleate) for COVID-19 in Brazil first appeared on PharmaShots.

Bayer Report Results of Aliqopa (copanlisib) + Rituximab in P-III CHRONOS-3 Study for Relapsed Indolent Non-Hodgkin’s Lymphoma

Shots:

  • The P-III CHRONOS-3 study involves assessing of Aliqopa + rituximab vs PBO + rituximab in 458 patients with relapsed indolent NHL who have received at least one or more lines of prior rituximab-containing treatment
  • The P-II study met its 1EP of prolonged PFS, safety is consistent with previously published data on the individual components of the combination, no new safety signals were identified
  • Aliqopa is a PI3K inhibitor with inhibitory activity predominantly against PI3K-α and PI3K-δ isoforms expressed in malignant B cells and is an approved treatment for patients with relapsed FL prior treated with two systemic therapies

    Click here to­ read the full press release/ article | Ref: Businesswire Image: Business Insider

The post Bayer Report Results of Aliqopa (copanlisib) + Rituximab in P-III CHRONOS-3 Study for Relapsed Indolent Non-Hodgkin’s Lymphoma first appeared on PharmaShots.

NICE changes its mind on Novartis’ progressive MS drug Mayzent

UK cost-effectiveness agency NICE has backed Novartis’ Mayzent for secondary progressive multiple sclerosis (SPMS), after turning it down earlier this year in draft guidance.

The change of heart means Mayzent (siponimod) becomes the first oral disease-modifying therapy to be recommended for NHS use in SPMS patients with active disease, defined as relapses or evidence of active inflammation of neurons on imaging.

Secondary progressive disease can occur after the relapsing/remitting stage of the disease, where patients experience fewer or no relapses but find their disability is increasing.

In June, NICE said it wasn’t able to support the use of Mayzent because there was limited clinical evidence for its benefits in SPMS, and it was not persuaded by the cost-effectiveness modelling submitted by Novartis.

Now, a consultation period and a new commercial agreement with Novartis to supply the drug at a discount on its £1,643.72 monthly list price means that around 38,000 people with SPMS across the UK could get access to the drug – which the MS Society charity says is “a huge step forward” for patients.

The NICE judgment for England and Wales follows a positive verdict from the Scottish Medicines Consortium (SMC) a few days ago. In Northern Ireland, the Department of Health reviews NICE guidance before deciding on use of a new drug.

Mayzent provides a treatment option to many people living with MS where once there was none, according to the MS Society, which said that people transitioning from relapsing/remitting MS to SPMS “have faced an immensely difficult challenge – being forced to go from having a range of treatments available to them, to severely limited choices.”

At the same time, for some people living with the secondary progressive form of the disease who are able to take beta interferons – currently given by injection – Mayzent provides a less intrusive choice, according to the charity.

Historically, the diagnosis of SPMS with active disease has often been delayed or avoided due to uncertainty around disease progression, as well as the lack of any effective treatment, says Novartis.

“We are working closely with the NHS to ensure eligible patients can start benefiting from siponimod as soon as possible,” said Chinmay Bhatt, managing director for Novartis Pharma UK, Ireland & Nordics.

Mayzent has the same mechanism of action as Novartis’ older drug Gilenya (fingolimod), which is approved for RRMS but not SPMS.

The drug was approved in Europe in January based on the 779-patient phase 3 EXPAND trial which showed that it significantly reduced the risk of disease progression, including physical disability and cognitive decline.

In a subgroup of Mayzent-treated patients with active disease, the data showed that the risk of three-month and six-month confirmed disability progression was significantly reduced, by 31% and 37% respectively, compared with placebo.

“By slowing down disability progression and improving cognition, siponimod has the potential to allow people to carry on working, remain independent and stay connected with family and friends,”  commented David Martin, chief executive of the MS Trust patient organisation.

“More broadly, we hope that the availability of this new treatment will lead to a greater focus on services for progressive MS which would benefit a much wider group of people,” he added.

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Flying the flag for research in Aarskog syndrome

Delayed diagnosis, lack of awareness, and a limited evidence base – Aarskog syndrome faces all the challenges of rare diseases everywhere. But a new study hopes to kick start a revolution in understanding.

For five years, Michelle Erskine faced a succession of closed doors and disbelief as she struggled to convince someone that all was not well with her son.

Eventually, after undergoing five operations in as many years, he was seen by a Consultant Ophthalmologist with knowledge of the condition and was placed on the road to an Aarskog syndrome diagnosis.

“When I saw it in black and white, I just burst into tears,” says Michelle, founder of the Aarskog Foundation, which recently marked Aarskog Awareness Week from 29 September to 4 October.

“Up until then, I was unable to convince anyone that something was wrong. We kept going to the nurse and the GP, but we were never referred to a paediatrician. No one would listen.”

The rare, X-linked genetic condition is characterised by short stature and facial, limb and genital abnormalities and was first described in 1970.

Michelle, who had recognised similarities between her son and two of her brothers, who had never been diagnosed, set up the Facebook page that eventually evolved into the foundation 12 years ago.

“I wanted to start a conversation and give people a space where they would be listened to. But from that, I wanted to formalise things – if all we do is talk as a group then nothing will ever get done”

Together with two fellow ‘Aarskog mums’ who had started social media pages around the same time, she had soon made contact with around 100 people from four countries around the world.

“I just wanted to start a conversation and give people a space where they would be listened to, so that no other mother would feel alone again. We were talking about the characteristics of the condition, advising people on how to speak to a GP, when to get a referral, etc.,” says Michelle.

“But from that, I wanted to formalise things – if all we do is talk as a group then nothing will ever get done. As my mum used to say: ‘if you do what you’ve always done, you’ll get what you’ve always got’.”

Variable impact

The Aarskog Foundation was established in 2017 and was given charitable status the following year. The objective, Michelle says, was to ensure families like hers received the support they needed.

“I wanted to change the dynamic, I wanted to raise awareness of the condition, and I wanted to help people to get a diagnosis,” says Michelle, who has published a six-step care plan pathway, which includes when to ask for a referral to a geneticist, on the foundation’s website.

As with many rare diseases, part of the problem families come up against when seeking care is a lack of solid evidence.

It is thought that around one in 25,000 people live with the condition, but the characteristics vary from person to person.

Most experience some form of cognitive difficulty, and some develop psychosocial problems related to short stature and feelings of social isolation.

The physical impact of Aarskog is extremely wide ranging.  Michelle’s son, who is now 22, needed operations for undescended testicles and a bilateral hernia before his fifth birthday, for example.

“The challenge we have with Aarskog syndrome is that the medical literature hasn’t extensively addressed the natural history and the spectrum of symptoms,” she says. “Although the advancements in sequencing technologies have enabled the genetic confirmation of this disease, an effort to understand the genetic basis of this multitude of symptoms is still due.”

For instance, all our children are on the autistic spectrum in one form or another. Still, there is a considerable lack of literature discussing the variability and etiopathogenesis of the autism spectrum disorder in Aarskog syndrome.”

Because Aarskog is an X-linked genetic condition affecting predominantly males, women have traditionally been considered asymptomatic carriers. However, Michelle believes that around 80% of the mothers she speaks to have an inflammatory disease such as rheumatoid arthritis or ankylosing spondylitis that should be evaluated for any association with this syndrome.

“Women have traditionally been considered asymptomatic carriers – but Michelle believes that 80% of the mothers she speaks to have an inflammatory disease such that should be evaluated for any association with Aarskog syndrome”

We are not aware of so many aspects of this disease because they have not been researched adequately,” she says.

Because this disease is so rare and there is no cure yet, it is not seen as important by researchers and physicians. This is what I want to change. I want people to have a system that they can rely on and be supportive”

Natural history study

To this end, Michelle’s foundation has just started working with two medical geneticists to identify and delineate the syndrome’s novel characteristics. This human history study project will use data from the foundation’s patient registry.

This study will primarily use the data to understand the natural history of the disease phenotype so that we can help the patients and families with better care of their ailments.

At the moment, there is information about this disease, but it’s neither concise nor comprehensive in nature.”

The human history study, which is being funded by the foundation and its supporters, will feed into gene reviews, an international point-of-source for inherited disorders. The team hopes that this study’s scope will also shed light on the gene’s impact on the women who carry it in addition to an elaborative genotype-phenotype correlation.

The two research projects, Michelle says, will start to build the evidence base.

This condition causes so much pain for families. I hope the research encourages clinicians to look into this further so that they can better inform people about the condition and put people on better management pathways,” she concludes.

To contribute to the cost of the studies, click here


Cambridge Rare Disease Network Exploring Rare Diseases is produced in partnership with Cambridge Rare Disease Network (CRDN). CRDN is building a vibrant network of patients and stakeholders to share knowledge and foster innovation that leads to better diagnosis, treatment and support for those living with a rare disease

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How Can Patients Get Medical Records from a Closed Medical Practice?

By GRACE CORDOVANO, DEVEN McGRAW, and AARON MIRI

The HIPAA Privacy Rule gives patients the right to copies of their medical records, with rare exceptions. When patients need a copy of their medical records, most start the process by calling their doctor’s office and asking for how to get access. The receptionist or office staff point them in the right direction, whether it’s instructing them to write down their request and sending it to the office, pointing them to contact the medical records or radiology department (if the practice is large enough), or assisting them in setting up their patient portal, if the practice is using an electronic health record (EHR). Being able to connect with a person inside the four walls of medicine is often crucial for many patients and their carepartners who may be unsure of exactly how to request their records.

But what happens to those records when a doctor closes or leaves the practice?

Independent practices close for a variety of reasons. Physicians may merge with a large practice or health system, retire, they may sell or close their practice for personal reasons, they may file for bankruptcy, or they may get sick and die. The COVID19 pandemic has had devastating financial consequences on many small, independent, and rural practices, leading to their consequent closure, acquisition, or merger.

What should patients do when their doctor’s office closes, and they need a copy of their medical records? This is especially challenging when a doctor may not have had an EHR, as is the case with many independent practices as well as more rural settings. On September 26, 2020, a tweet from Cait DesRoches, Executive Director of OpenNotes, inquired about how a family member may get access to medical records from her physican’s practice that closed, triggering a robust conversation that led to the realization that patients and families are not well informed in these circumstances.

Prevention is Worth a Pound of Cure

It can be much more difficult to get copies of records after a practice has closed. Patients should get copies of their medical records as they are generated instead of waiting until they’re needed. HIPAA Privacy Rule guidance states that individuals can get digital copies of digital information (or even digital copies of records kept on paper, as long as the practice has a scanner). Companies are developing tools and services that enable individuals and their care partners to collect, use, and store health records. Request digital (or paper, if that is preferred) copies of blood work, imaging, discharge instructions, and corresponding reports before you leave the practice.

What Happens to Medical Records When Offices Close? The Law

The Health Insurance Portability and Accountability Act (HIPAA) does not require a physician to retain medical records for any particular period of time. (HIPAA covered entities – which include physicians who bill health insurers for care – are required to keep records demonstrating compliance with HIPAA for at least six years – but those records are distinct from medical records.) However, if the physician still has those medical records – or has placed them in storage for safekeeping – the HIPAA requirements to produce them when a patient requests still apply.

State laws typically set medical record retention requirements for physicians and may also require the physician to take particular steps (such as notifying a patient) prior to or upon closure of a practice. 

An example of some of these state laws:

  • In California, physicians must notify patients in advance of closure of the practice, and are still responsible for safeguarding records and making sure they are available to patients. The California Medical Association recommends physicians keep records for at least ten years from the last date the patient was seen.
  • New York requires that medical records be retained for six years from the date of the most recent entry in the record, and patients are required to informed when a practice closes.
  • Virginia prohibits the transfer of medical records as part of the closure or sale of a practice until the provider has first attempted to notify by the patient by mail or by publishing notice in a newspaper of general circulation in the area.
  • Texas law requires physicians to keep records for a minimum of seven years after the date of last treatment, and physicians leaving a practice are required to notify patients.

During the record retention period, these records are considered to be still “available” and subject to the HIPAA right of access. Consult the medical board or the state medical society in the state where the physician has practiced for further information about physician requirements in the event of closure of a practice. The Medical Board should also have information about how to file a complaint if the physician’s practice has closed without any notice or information about how to obtain records. 

Irrespective of legal requirements, the American Academy of Family Physicians recommend that patients be notified by a letter that the office is closing, giving them the opportunity to obtain a copy of their medical records or have records forwarded to a physician of their choosing. The office may post an update on their website or social media page(s), if ones exist or run an ad in the local newspaper. Patients should be notified who will be the custodian of the medical records and their contact information.

Sorry! The Office Is Closed

Unfortunately, the reality is that most individuals do not get copies of their medical records throughout their care journey. This leaves patients and carepartners in need of records facing significant uncertainty, stress, and frustration when they unexpectedly find out that their doctor’s office has closed. Here are a number of critical tips to assist patients in gathering their medical records, directly and indirectly, in the event their doctor’s office has closed.

  1. It is helpful to know when the office may have closed: was it recently or many years ago? As noted above, state laws govern how long records must be retained as well as how they must be handled with respect to confidentiality, privacy, and how they may be destroyed, when and if needed. Typically, records that are about 10 years from the last documented encounter, may be candidates to be destroyed and may be more difficult to obtain as a copy.  (As noted above, state laws may allow for them to be destroyed even sooner than 10 years.)
  • Individuals should refer to the letter they may have received notifying them of the office closing and contact the designated records custodian. Updates may also have been posted to the physician or practice’s website or social media page, if available. The local librarian may assist with researching for the office closure notice in archived newspapers or posts in the public domain.
  • Insurance companies, current and previous, should be contacted to request any claims that may have been received from the specific physician or provider’s practice. A supervisor should be requested and relayed specific information about the health information needed and why is it critical for one’s care. In the event individuals are encountering difficulty getting traction over the phone, individuals may turn to social media for help.  If the respective insurance company has a Twitter account, individuals may tweet their request while including the insurance company’s Twitter handle. Social media managers are often very responsive and may be an additional avenue for connecting individuals to the information they need if it is perceived that delays in response may be detrimental to their company’s reputation.
  • Is there another doctor or professional now at the same physical office/facility location? Individuals should address the request in-person or via a call. The new office staff often receive many of the same questions from other previous patients and may have contact information for a point person on hand. They may also have the records in question if the practice was acquired (where applicable).
  • Individuals should contact their local chamber of commerce, borough hall, or local Department of Health. If the office closure was recent, someone may know a way to connect with the doctor or a former staff member for more information.
  • Did the doctor have other doctors on staff? If so, individuals may search for the other doctors who may still be in practice at another location to see if they may have a contact for where records have been retained.
  • Individuals may quickly determine if their doctor is on social media, such as LinkedIn, Twitter, and Facebook, and respectfully direct message them with their request for more information.
  • Individuals may search the internet for any recent press releases that may feature the doctor’s work, activism, or research and contact the respective article’s author or journalist. At minimum, they may be willing to forward the request for records to the doctor.
  • If individuals need specific information on medications, they may contact the pharmacy that was used to fill respective prescriptions so as to request copies of prescription records.
  1. Individuals should contact their primary care doctor, and other members of their  care team, to see if records were forwarded to them for continuity of care purposes.
  1. If an individual’s doctor is deceased, the state medical licensing board may be contacted to determine the care provider’s county of residence. Consequently, the specific state’s county probate court may be contacted to confirm if there is a designated executor of estate that has authority over records retention processes. Alternatively, an obituary may list surviving next of kin which may also be contacted for more information on records retention.
  1. If medical records were available digitally, individuals may look up their state and “health information exchange (HIE)”.  An HIE is a secure network that supports the electronic exchange of patient health information among trusted data entities typically across an entire state. Individuals should research if there is an HIE that may serve their local area. An HIE’s website will have a phone number and email to contact directly with your request.
  1. If imaging was performed, individuals may reach out to the respective imaging center or the location where imaging was done to request copies of images on CDs and the corresponding reports.
  1. If bloodwork was performed, individuals may contact the lab, such as Quest or LabCorp, that processed the tests directly for copies of final lab reports. Individuals may contact their insurance company, current or previous, if they are unsure of the names of the labs that may have been in-network via their plan; individuals can also use their right of access to get copies of claims from their health plan, which may identify the lab that processed the tests.
  1.  If individuals are in need of immunization records they may contact their state Department of Health as they may have an immunization registry. The Immunization Action Coalition also has information on locating immunization records.
  1. If individuals are working within the framework of a specific diagnosis or condition, they may research non-profits that support patients within that specific disease state and reach out for peer health support, where other individuals diagnosed with the same condition may also be able to assist in navigating these barriers to patient access based on their own lived experiences.
  1. A state’s medical board, Office of the Attorney General (AG) and state’s Department of Health are all resources for additional support.

Individuals may also file a complaint with the Office of Civil Rights (OCR) at the Department of Health and Human Services (HHS) if all efforts have been exhausted and the needed medical records have not been obtained.

A closed practice does not need to be a dead end for patient access. Proactively requesting copies of medical records throughout one’s care journey can prevent encountering such patient access barriers. Continuing to share best practices for navigating patient access barriers, from legal, regulatory, and practical standpoints, is in the best interest of all patients.

Grace Cordovano, PhD, BCPA is a board-certified patient advocate specializing in the oncology space, a patient experience enhancer, and information unblocker.

Deven McGraw , JD, MPH, LLM (@healthprivacy) is the Chief Regulatory Officer at Ciitizen (and former official at OCR and ONC). She blogs at ciitizen.com.

Aaron Miri is the Chief Information Officer for The University of Texas at Austin comprising of the Dell Medical School, UT Health Austin clinical enterprise, research, and community impact missions.

Roche working “around the clock” to tackle UK test supply problems

Supplies of swabs for coronavirus and other critical NHS tests for diseases like cancer, diabetes and heart disease have been threatened by a failure at a facility operated by Roche.

The company said the problems – which also include other items like reagents and screening kits – have resulted from a switch to a new warehouse, which ironically is intended to make production quicker and more efficient through the use of automated processes.

The issues have led to a significant drop in processing capacity that could impact testing for two to three weeks – just as daily cases of coronavirus in the UK are continuing to escalate with more than 14,500 new infections reported yesterday.

Roche is one of the major suppliers of testing equipment for the NHS, with most of its materials for the UK market coming from a single distribution facility in Newhaven, East Sussex.

A BBC report says that at least one NHS trust has advised doctors to ration non-urgent tests and prioritise supply of swabs for coronavirus testing, and there are concerns some essential items could be out of supply within a few days.

North Devon Healthcare NHS Trust says it is expecting a delivery of swabs next week, adding if that doesn’t materialise it will have a big impact on its ability to carry out COVID-19 testing.

In an emailed statement, Roche told us that: “We deeply regret that there has been a delay in the dispatch of some products. We are prioritising the dispatch of [coronavirus diagnostics] and antibody tests and doing everything we can to ensure there is no impact on the supply of these to the NHS.”

The company adds that since then it has “worked around the clock to prioritise and manage orders as well as increase this capacity.”

It goes on: “As well as extending working hours, we have recruited extra staff and, where they can, our dedicated teams on the ground are working with customers to distribute products and minimise service disruption.”

Antibody test order

Roche’s issues affect the supply of swab tests used to detect if someone is currently infected with SARS-CoV-2, but there was better news on the supply of antibody tests that detect whether a person has been exposed to the virus in the past.

This week the UK government signed a contract with Abingdon Health for the supply of a million AbC-19 rapid antibody tests, which use a small drop of blood from a finger-prick and deliver results in 20 minutes without the need for a specialised lab.

The government said the testing kits will be used to “help build a picture of how the virus has spread across the country and further develop our understanding of how antibodies work.”

Antibody testing was trumpeted in March as the UK’s ticket to emerge from lockdown, but that view was soon undermined by the proliferation of tests that didn’t meet regulatory guidelines.

There are three main approved blood tests for COVID-19 in the UK – from Roche, Abbott and Ortho Clinical Diagnostics – but these require a full venous blood sample rather than a fingerpick and have been in limited supply, reserved mainly for healthcare workers.

That hasn’t stopped a myriad of companies from offering fingerprick tests – including claiming to be based on these technologies – directly to the public. against Public Health England (PHE) and Medicines and Healthcare products Regulatory Agency (MHRA) guidance.

Abingdon says its test has undergone an independent evaluation commissioned by the UK government that will be published in full in due course by PHE, after peer review.

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Roche working “around the clock” to tackle UK test supply problems

Supplies of swabs for coronavirus and other critical NHS tests for diseases like cancer, diabetes and heart disease have been threatened by a failure at a facility operated by Roche.

The company said the problems – which also include other items like reagents and screening kits – have resulted from a switch to a new warehouse, which ironically is intended to make production quicker and more efficient through the use of automated processes.

The issues have led to a significant drop in processing capacity that could impact testing for two to three weeks – just as daily cases of coronavirus in the UK are continuing to escalate with more than 14,500 new infections reported yesterday.

Roche is one of the major suppliers of testing equipment for the NHS, with most of its materials for the UK market coming from a single distribution facility in Newhaven, East Sussex.

A BBC report says that at least one NHS trust has advised doctors to ration non-urgent tests and prioritise supply of swabs for coronavirus testing, and there are concerns some essential items could be out of supply within a few days.

North Devon Healthcare NHS Trust says it is expecting a delivery of swabs next week, adding if that doesn’t materialise it will have a big impact on its ability to carry out COVID-19 testing.

In an emailed statement, Roche told us that: “We deeply regret that there has been a delay in the dispatch of some products. We are prioritising the dispatch of [coronavirus diagnostics] and antibody tests and doing everything we can to ensure there is no impact on the supply of these to the NHS.”

The company adds that since then it has “worked around the clock to prioritise and manage orders as well as increase this capacity.”

It goes on: “As well as extending working hours, we have recruited extra staff and, where they can, our dedicated teams on the ground are working with customers to distribute products and minimise service disruption.”

Antibody test order

Roche’s issues affect the supply of swab tests used to detect if someone is currently infected with SARS-CoV-2, but there was better news on the supply of antibody tests that detect whether a person has been exposed to the virus in the past.

This week the UK government signed a contract with Abingdon Health for the supply of a million AbC-19 rapid antibody tests, which use a small drop of blood from a finger-prick and deliver results in 20 minutes without the need for a specialised lab.

The government said the testing kits will be used to “help build a picture of how the virus has spread across the country and further develop our understanding of how antibodies work.”

Antibody testing was trumpeted in March as the UK’s ticket to emerge from lockdown, but that view was soon undermined by the proliferation of tests that didn’t meet regulatory guidelines.

There are three main approved blood tests for COVID-19 in the UK – from Roche, Abbott and Ortho Clinical Diagnostics – but these require a full venous blood sample rather than a fingerpick and have been in limited supply, reserved mainly for healthcare workers.

That hasn’t stopped a myriad of companies from offering fingerprick tests – including claiming to be based on these technologies – directly to the public. against Public Health England (PHE) and Medicines and Healthcare products Regulatory Agency (MHRA) guidance.

Abingdon says its test has undergone an independent evaluation commissioned by the UK government that will be published in full in due course by PHE, after peer review.

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Retired NFL Linebacker Overcomes ADHD, Invests in Mental Health Platform Done

October is ADHD Awareness Month, and retired NFL linebacker J.T. Thomas wants people to know that it is okay to get help for the treatment of attention deficit hyperactivity disorder (ADHD). As a person with ADHD, J.T. Thomas knows how important getting help is, and why overcoming obstacles such as stigma, access to care and improper diagnosis are so important.
“Whether competing in the NFL, or in life, many of us feel that we can’t show weakness,” says Thomas. “Unfortunately, this way of thinking has kept millions of adults and children from getting the treatment they need. When I finally sought treatment in 2013, I learned how to turn my ADHD into a strength by better managing it. Getting help was the best thing I ever did.”
J.T. Thomas is advocating for greater awareness regarding diagnosis and treatment options for ADHD because he believes that “knowing and treating” can be life changing. This is what led him and others including David Sacks’ Craft Ventures, Dave Morin’s Offline Ventures and NFL Hall of Famer Joe Montana to invest in ADHD treatment platform Done. Done is the first telehealth platform created specifically for people looking to get help with ADHD.
“I travel a lot, and this makes it very difficult to maintain adherence to traditional, in-person treatment,” added Thomas. “A friend told me about telehealth, and that’s when I found Done. They were focused on ADHD 100%, so I reached out to them and started getting help. I enjoyed the service so much, I asked if I could invest in it.”
Done was founded in 2019 and is led by a 13 person team of former Facebook, Stanford University and Kaiser Permanente professionals. Done, which is making its public debut during ADHD Awareness Month, currently has a team of 25 clinicians available in *11 states.
Done is focused specifically on ADHD because many on the team either have ADHD, or have family and friends with ADHD. As such, Done’s staff fully understands the potential obstacles associated with ADHD.
The primary function for physicians is to enhance health and alleviate suffering. Sadly, in the real world, there are many barriers to the realization of these goals. Medical knowledge alone cannot remove the obstacles, for they are social, political, economic and organizational. This is especially true when it comes to the treatment of ADHD.
“In my career as a psychiatrist, I have been particularly affected by the barriers to care created by the stigma attached to the medical disorders artificially categorized as mental,” states Done Clinical President Dr. David Brody. “The care of all psychiatric disorders is severely affected by this stigma, which takes different forms depending on the specific disorder, but is always destructive. In the case of ADHD, the stigma includes the ideas that it is not real, not severe or serious, and that people seeking treatment for it are drug seeking or looking for an easy way. These stereotypes could not be further from the truth, and that’s why in addition to treatment, at Done we are focused on awareness.”
Done is here to combat these stereotypes, and is working to remove barriers to treatment such as requirements and expectations about frequency of visits, documentation, medication choice and choice of provider. Regulations regarding treatment vary tremendously state-to-state, which is primarily due to the regulatory climate surrounding the medications used for first-line treatment of the disorder. This often forces patients to end treatment, especially if they live far from their provider’s office or have multiple career and family responsibilities.
As a flexible and more affordable platform, Done is well on the way to achieving a more effective and patient-friendly system for ADHD treatment. However, much remains to be “done.”
Priorities for Done include recruitment of additional clinicians for its rapidly expanding organization, improved integration with pharmacies and pharmaceutical manufacturers, improvement of regulations affecting patient care, education of both the general public and the medical community on the reality of ADHD and the falseness of stigmatizing ideas, and streamlining the platform to deal with the roadblocks and inefficiencies that stem from that stigma.
*Done offers services to residents of California, Florida, Hawaii, Indiana, New Jersey, New Mexico, New York, Oregon, Pennsylvania, Texas and Washington.

The post Retired NFL Linebacker Overcomes ADHD, Invests in Mental Health Platform Done appeared first on .

Retired NFL Linebacker Overcomes ADHD, Invests in Mental Health Platform Done

October is ADHD Awareness Month, and retired NFL linebacker J.T. Thomas wants people to know that it is okay to get help for the treatment of attention deficit hyperactivity disorder (ADHD). As a person with ADHD, J.T. Thomas knows how important getting help is, and why overcoming obstacles such as stigma, access to care and improper diagnosis are so important.
“Whether competing in the NFL, or in life, many of us feel that we can’t show weakness,” says Thomas. “Unfortunately, this way of thinking has kept millions of adults and children from getting the treatment they need. When I finally sought treatment in 2013, I learned how to turn my ADHD into a strength by better managing it. Getting help was the best thing I ever did.”
J.T. Thomas is advocating for greater awareness regarding diagnosis and treatment options for ADHD because he believes that “knowing and treating” can be life changing. This is what led him and others including David Sacks’ Craft Ventures, Dave Morin’s Offline Ventures and NFL Hall of Famer Joe Montana to invest in ADHD treatment platform Done. Done is the first telehealth platform created specifically for people looking to get help with ADHD.
“I travel a lot, and this makes it very difficult to maintain adherence to traditional, in-person treatment,” added Thomas. “A friend told me about telehealth, and that’s when I found Done. They were focused on ADHD 100%, so I reached out to them and started getting help. I enjoyed the service so much, I asked if I could invest in it.”
Done was founded in 2019 and is led by a 13 person team of former Facebook, Stanford University and Kaiser Permanente professionals. Done, which is making its public debut during ADHD Awareness Month, currently has a team of 25 clinicians available in *11 states.
Done is focused specifically on ADHD because many on the team either have ADHD, or have family and friends with ADHD. As such, Done’s staff fully understands the potential obstacles associated with ADHD.
The primary function for physicians is to enhance health and alleviate suffering. Sadly, in the real world, there are many barriers to the realization of these goals. Medical knowledge alone cannot remove the obstacles, for they are social, political, economic and organizational. This is especially true when it comes to the treatment of ADHD.
“In my career as a psychiatrist, I have been particularly affected by the barriers to care created by the stigma attached to the medical disorders artificially categorized as mental,” states Done Clinical President Dr. David Brody. “The care of all psychiatric disorders is severely affected by this stigma, which takes different forms depending on the specific disorder, but is always destructive. In the case of ADHD, the stigma includes the ideas that it is not real, not severe or serious, and that people seeking treatment for it are drug seeking or looking for an easy way. These stereotypes could not be further from the truth, and that’s why in addition to treatment, at Done we are focused on awareness.”
Done is here to combat these stereotypes, and is working to remove barriers to treatment such as requirements and expectations about frequency of visits, documentation, medication choice and choice of provider. Regulations regarding treatment vary tremendously state-to-state, which is primarily due to the regulatory climate surrounding the medications used for first-line treatment of the disorder. This often forces patients to end treatment, especially if they live far from their provider’s office or have multiple career and family responsibilities.
As a flexible and more affordable platform, Done is well on the way to achieving a more effective and patient-friendly system for ADHD treatment. However, much remains to be “done.”
Priorities for Done include recruitment of additional clinicians for its rapidly expanding organization, improved integration with pharmacies and pharmaceutical manufacturers, improvement of regulations affecting patient care, education of both the general public and the medical community on the reality of ADHD and the falseness of stigmatizing ideas, and streamlining the platform to deal with the roadblocks and inefficiencies that stem from that stigma.
*Done offers services to residents of California, Florida, Hawaii, Indiana, New Jersey, New Mexico, New York, Oregon, Pennsylvania, Texas and Washington.

The post Retired NFL Linebacker Overcomes ADHD, Invests in Mental Health Platform Done appeared first on .

The COVID-19 Effect: How Pharma Can Adapt to the Evolving Patient Experience

Patient experiences are evolving. How can pharma keep up?
By listening to the people who are navigating this new landscape everyday.

Integrating personal experiences and patient-reported data, this one-day virtual event features real-time conversations between people living with chronic conditions and Health Union industry experts.
Tune in to any or all of the sessions based on your interests and hear from patient advocates living with migraineMSpsoriasis, and lung cancer; plus a lunchtime roundtable focused on telehealth featuring advocates living with Parkinson’s disease and IBD.

Tune in to any or all of the sessions based on your interests:

  • [10:00 – 10:30 a.m.] MS & Social Distancing: Devin’s Resilience
  • [11:00 – 11:30 a.m.] Psoriasis Used to Isolate Reena, Now It Connects Her
  • [12:00 – 12:45 p.m.] Telehealth Perspectives: The Good, The Bad & The Ugly
  • [01:00 – 01:30 p.m.] Healthcare + Self-Care: Kerrie’s Migraine Journey
  • [02:00 – 02:30 p.m.] Listen & Learn from People with Lung Cancer, Like Lisa

Register now

The post The COVID-19 Effect: How Pharma Can Adapt to the Evolving Patient Experience appeared first on .

Pfizer Signs an Agreement with SpringWorks to Evaluate Nirogacestat + PF‐06863135 for R/R Multiple Myeloma

Shots:  

  • Pfizer to sponsor & conduct P-Ib/II study evaluating the safety, tolerability, and preliminary efficacy of dual regimen and will assume costs of study & other expenses related to IP rights.  The companies will form a joint development committee to manage clinical study which is anticipated to commence in H1’21
  • The focus of the collaboration is to evaluate SpringWorks’ Nirogacestat + Pfizer’s PF‐06863135, in patients with r/r MM. Additionally, SpringWorks is currently conducting a global P-III DeFi trial to evaluate nirogacestat in adults with progressing desmoid tumors
  • Nirogacestat is investigational, oral, selective, small-molecule GSI in P-III while PF‐06863135 is anti-BCMA CD3 bispecific Ab (SC), being investigated in P-I study to treat r/r MM

Click here ­to­ read full press release/ article | Ref: PRNewswire | Image: Solar Winds MSP

The post Pfizer Signs an Agreement with SpringWorks to Evaluate Nirogacestat + PF‐06863135 for R/R Multiple Myeloma first appeared on PharmaShots.

The value of good data to patients

Better patient care pathways and models of care can be leveraged from the use of data, and the ability to collate and assess this information will drive more sustainable models of care, whilst unlocking the value of ‘longitudinal, real-world data’ as a resource for healthcare organisations, research and academia, policy advisors and pharma.

There is a societal shift that is required though, to educate patients on the value of this data and how it can be used to improve the outcome of patients globally. Samir Dhalla, head of THIN and Richard Ballerand, co-chair of THIN’s Patient Advisory Committee, explore how ‘good data’ can power this.

The benefits for public health

Data has been essential in the fight against COVID-19, with records used to identify and notify 2.2 million individuals who were at significant risk from the virus, and to advise them to shield. Ongoing tracking and data analysis of the virus has also allowed for data profiles to be built up around the types of conditions that result in COVID-19 posing a greater threat, for instance Type 2 diabetes. The NHS has proven the value of public health data in this way, and globally it has been a powerful tool to assess risks and treatments.

It’s also helped to drive research – for example the European Health Data & Evidence Network’s (EHDEN) took part in the OHDSI COVID-19 Virtual Study-a-thon which rallied 330 researchers with very different backgrounds and from thirty different countries. It also launched its COVID-19 Rapid Collaboration Call in a bid to plug the gaps in fragmented, siloed and poorly interoperable real-world health data in order to characterise patients, manage their care and assess treatment safety.

“The public are generally comfortable with anonymised data from medical records being used for improving health, care and services. However, there is still worry around this health data being accessed by commercial organisations”

These forms of national and international collaboration have shown the power of data in managing the pandemic, as the importance of population health data in identifying and containing the virus has not been overstated. The NHS has created its own COVID-19 Data Store for instance, and on top of this, analysis of 16.2 million anonymised longitudinal patient records revealed a significant increase in the proportion of lower respiratory tract infections diagnosed by late 2019 compared to previous flu seasons, raising the odds of UK COVID-19 cases having appeared earlier than previously thought.

By harnessing the information in electronic health records, such findings clearly open the path to near real-time tracking of large-scale population health events that can support public health bodies to detect, investigate, and respond timely and effectively to them.

Anonymisation

Patients are becoming more content for their data to be used in this way, however it must be anonymised to tackle privacy and trust concerns. The public have reported they are generally comfortable with anonymised data from medical records being used for improving health, care and services, for example through research. However, there is still worry around this health data being accessed by commercial organisations, and so the privacy and broader benefits of patient data must be emphasised.

For instance, data can be used to improve diagnosis speeds and anonymous data can be overlaid onto NICE guidance, as well as within local and national patient pathways to provide better insight into the types of treatments that might work more effectively for certain patients. ‘Cohorts’ of patients can be created as well as effective treatment profiles based on these individual characteristics, to ensure the most effective treatment pathway is selected and faster. These can be based on existing health conditions, for example diabetes, and co-morbidities. Information such as age, weight, sex, faith, ethnicity and lifestyle choices can also be included to help specialists get closer to the best treatment the first time around to improve patient outcomes.

A prime example of the value of this type of data is in the case of pancreatic cancer, a disease that is often diagnosed late and progresses exceptionally quickly. It’s the deadliest common cancer, and by the time a diagnosis is achieved, many individuals are at stage three or stage four, with 75% not surviving a year after diagnosis. Symptoms are vague and often confused with other conditions such as pancreatitis, gallstones, irritable bowel syndrome (IBS) or hepatitis.

For both GPs and patients, embedding detailed symptom information within clinical systems would be hugely beneficial, enabling far earlier diagnosis and hospital referral. The GP would spend far less of the budget on one specific patient and it would ensure that the patient receives the best treatment possible for that diagnosis and faster referral to the right clinician.

Predictive modelling

Data analytics can also be used to better understand patient responses to specific treatment types and surgeries, by using predictive modelling to map out likely outcomes – enabling pharma to work closely with healthcare professionals and services to get closer to ‘the right medicine, to the right patient, first time’.

Modelling based on real-world data allows for tailoring and marketing specific medicines to specific groups of patients with specific predispositions or health conditions, and it reduces the ‘trial and error’ process that clinicians sometimes have to follow to find the appropriate treatment.

For example, analysis of cardiac surgery patients has evaluated the risks for certain types of heart surgery as well as post-surgery, taking into consideration a number of factors, including age and ethnicity to improve recovery. This can be extended to provide individual patients with information about their health conditions and risks to empower and encourage them to make changes to lifestyle or behaviours. This Predictive, Preventive and Personalised Medicine (PPPM) explores a complex mix of personal and population health data to avoid future health deterioration or detect problems before they arise.

Utilising datasets

Ultimately, PPPM will help patients to take control of their own health issues. Retaining individuals within primary care will both save money by reducing the pressure on secondary care and release investment in other areas of high demand healthcare. On a broader level, this detailed predictive model will allow the healthcare system to potentially predict population health issues over the next three to four years, helping to allocate resources to the correct specialties at the right time.

The key to this process is early identification of potential health issues, which requires proactive collection of patient health data – from weight to cholesterol and blood pressure readings. Understanding the trajectory of these readings over the past year, combined with predictive modelling as to the potential outcome if no preventative measures are taken, will help clinicians to present personalised patient advice.

The other important factor is the volume of the datasets used in analysis. The pool of reliable patient data must be detailed and vast in order to provide a complete picture and inform decisions. Globally it’s known that there is significant value to be had, but many researchers are still relying on synthetic datasets which mirror real-world data. This data simply fails to deliver the depth of insight delivered by real-world patient data.

It’s clear that the analysis and use of global health data can drive better patient diagnosis, treatments and ultimately outcomes. There has been a societal shift around the use of personal health data in recent months, as the value of it has been shown in the fight against COVID-19, and this change can continue to leverage benefits.

Primacy Care data is being utilised to inform patient pathways across a range of disease areas and enable better understanding of local health economies, while GPs have a chance to inform life-changing medical research, supporting research crucial to gaining insights and developing policies, and helping to highlight trends in clinical effectiveness within the NHS.

Patient data can achieve significant changes in preventative care and improve global health for the better; but it has to be the power of good data.

About the authors

Samir Dhalla is Head of THIN and Richard Ballerand is co-chair of THIN’s Patient Advisory Committee.

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COVID-19 drives a new era of self-care

New research shows that the COVID-19 pandemic is encouraging people to take their health into their own hands, and experts say this trend towards self-care could be a significant boon for healthcare systems – one the pharma industry should be encouraging.

We often hear how early detection and disease prevention will be key for the future of healthcare – alleviating pressure on already over-stressed health systems by nipping costly and preventable diseases in the bud before they can drain healthcare resources, but a sometimes-overlooked dimension to this is encouraging people to practice self-care and rely less on doctors to treat minor illnesses.

“There’s massive pressure on limited resources in healthcare, while systems like the NHS are still mostly only able to focus on curing diseases and not preventing them,” says Ed Hudson, managing director of Create Health. “People are going to have to start taking ownership of their own health. It’s going to slowly stop being socially acceptable to not take any responsibility for your health, especially if you have several comorbidities.

“Self-care will even up resources and give a better shape to our healthcare system.”

“Greater adoption of self-care helps to create a better future for all, through creating a health society, healthy economy and improving the health of the global population,” adds the Global Self-Care Federation’s (GSCF) director general Judy Stenmark.

“For the pharmaceutical industry, responsible self-care is crucial to ensuring a healthy population that is health literate, understands self-treatment and practices it effectively”
Judy Stenmark, GSCF

“For the pharmaceutical industry, responsible self-care is crucial to ensuring a healthy population that is health literate, understands self-treatment and practices it effectively.”

The GSCF, which is celebrating its 50th anniversary this October, represents associations and manufacturers in the self-care industry, and aims to promote the benefits of self care for sustainable and better global health outcomes.

“Self-care is an essential pillar of sustainable healthcare systems,” Stenmark adds. “At a time when less than half of the global population has access to essential health services, responsible self-care helps to improve health, human rights and social outcomes, reduce health disparities and increasing equity and increase health coverage and access.

“It can also reduce costs and improve efficiency, as well as increase the quality of services.”

Stenmark notes that self-care has become even more important during the COVID-19 pandemic, which has quickly seen healthcare systems become overstretched.

“Self-care plays a key role in relieving the burden on healthcare practitioners and services as coronavirus continues to trigger adverse health consequences across the globe,” she says.

Indeed, new research from GSK Consumer Healthcare and Ipsos MORI has shown that since the pandemic began people in the UK are increasingly self-educating and forming new habits to improve everyday health, from visiting pharmacists instead of the GP to staying at home when ill to avoid transmission.

The survey, which looked at 1,108 participants aged between 16 and 75 years of age, found that the majority of people (77%) consider it important to take their health into their own hands in order not to burden the healthcare system.

As a result, almost half of all Britons (48%) said they intend to use pharmacists more in the future to give them advice about how to treat minor health concerns.

The research also revealed that 39% of Britons thought they took their health for granted before the COVID-19 outbreak, with 56% saying that they now have a better understanding of what impacts their health.

“You don’t really think of TikTok and B2B marketing together, but why not? We need as many different layers of communication as we can get to make self-care viable in the future”
Ed Hudson, Create Health

The authors say that in the wake of the pandemic, there is a renewed appetite for proactive self-education on the topic of health; some 50% of respondents said they now feel more able to recognise their body’s symptoms of ill health and 62% are more likely to consider their health in day-to-day decision-making.

When it comes to self-care, respondents said they considered it important to alleviate pressures on the NHS – they are avoiding GP and A&E visits as a first port of call in times of illness, and instead turning to pharmacists, digital resources or over-the-counter products.

Only 10% said they would visit their GP or A&E as the first port of call when feeling unwell, and over three-quarters (77%) consider it important to take their health into their own hands in order to reduce the burden on the NHS.

Almost half of Britons plan to consult pharmacists more often about how to treat minor health concerns in the wake of the pandemic, and around three-quarters state that they would trust pharmacists for advice.

Seventeen percent said they would use online resources for guidance in the first instance of minor illness (internet search engine, NHS Choices website, or other medical website or app).

Meanwhile, almost half of those surveyed say they are now more likely to treat illness with over-the-counter medicines instead of going to their GP.

Encouraging further self-care

For now these trends are mostly driven by stay-at-home orders and the desire to relieve pressure on health services, but Stenmark says she expects these trends to continue once the pandemic is over.

In fact, she notes that before COVID there were plenty of other factors driving the adoption of self-care – including a rapidly ageing population with a greater need for chronic disease management and a more active lifestyle, as well as the increasing ubiquity of digital, which leads to consumers wanting convenient, transparent and affordable options and empowers them to seek holistic, personalised solutions.

Beyond this, there are other factors within the control of the healthcare and life sciences industries that will determine how ubiquitous self care actually becomes.

For example, Stenmark says that boosting health literacy is both the top driver and the biggest barrier to further adoption.

“Increased health literacy empowers individuals to play an active role in improving and managing their own health. Access to better information and education on self-care and available self-care options is crucial.”

She adds that there is an important role here for the pharma industry to help improve health literacy by better educating consumers.

Trust is another key factor. GSCF has conducted a trust audit to understand trust in the self-care industry among both consumers and industry stakeholders. The audit shows that product safety, efficacy and regulation are the primary drivers of trust in self-care.

When looking at regions, Europe scores high in trust as a result of its focus on policy, testing and regulation control.

The audit also showed that factors weakening trust include weak regulatory frameworks and a lack of responsible use and promotion of self-care products.

“Improving policy and regulation is crucial to strengthening the perception of the industry and will allow for the correct structures to be put in place to enable people to make the right decisions,” says Stenmark.

Self-care via TikTok

Hudson stresses the importance of good communications from pharma and healthcare to improve literacy and trust – but says that the industry still often lacks the ability to truly connect with its customers.

“Often pharma’s communication is not that compelling or motivating,” he says. “They tend to forget that patients are consumers as well, and go for more B2B style comms rather than B2C.

“The industry is good at communicating to clever doctors, but not that good at doing simple or compelling communications for patients. They have to use language that is accessible.

“If your communications can help a patient’s children understand how to, say, put on a wound dressing, then it will work. If they can’t, then it won’t work. It’s not about patronising people but about recognising that medicine is quite complicated.”

He says that the digitisation of communications is helping to make self-care more achievable for populations.

“Formats like animations and videos are important for this and need to become more prevalent. It can be hard to articulate information about a drug, such as what the molecule does, why people should take it, and what it can do for them – but animations and videos can help enormously with that, and are often a better way of explaining these complex subjects.

“These formats aren’t exactly new to healthcare, but at the moment many of them aren’t high-quality. Often companies will just do an animation for the sake of it and not put any thought or budget into it, resulting in a bad asset that won’t be effective at all.

“When using digital formats, the industry needs to think about what problems they’re actually trying to solve.”

Hudson notes some unusual but creative examples of recent comms that have been effective in disseminating self-care information, such as getting diabetic TikTok influencers to post a dance video about how to properly administer an insulin injection, or using character skins in a video game to educate people on a skincare product.

“You don’t really think of TikTok and B2B marketing together, but why not? We need as many different layers of communication as we can get to make self-care viable in the future.”

The post COVID-19 drives a new era of self-care appeared first on .

Sobi & Selecta Report Results of SEL-212 in P-II COMPARE Study for Chronic Refractory Gout

Shots: 

  • The P-II COMPARE study assessing SEL-212 (once monthly) vs pegloticase (twice monthly) in 170 patients with chronic refractory gout
  • SEL-212 showed a numerically higher response rate on 1EP during 3 & 6 mos., but didn’t meet 1EP of superiority, higher response rate during 3 & 6mos., an overall reduction in mean SUA levels; patients with tophi at baseline showed higher responder rates with a reduction in mean SUA and is well tolerated
  • SEL-212 is a combination product candidate designed to sustain control SUA levels in patients with chronic refractory gout, potentially reducing harmful tissue urate deposits. The companies also initiate the P-III studies (DISSOLVE I& II) of SEL-212 for chronic refractory gout with its expected results in H2’22 and BLA filing in Q3’23

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

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Industry group says FDA botched COVID-19 convalescent plasma guidance

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.”

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Chinese starts vaccinating key workers against COVID-19; report

China has already granted emergency use authorisation to multiple COVID-19 vaccines developed by domestic drugmakers and started dosing key workers, according to local media reports.

Zheng Zhongwei, head of China’s COVID-19 vaccine development task force, made the revelation on Chinese television, saying that the vaccines were being used in people at high-risk of contracting SARS-CoV-2, including front-line medical staff, according to the China Daily news service.

Around 20,000 people have been vaccinated since emergency use was approved in July, focusing on healthcare workers and border control staff, and the plan is to scale up the programme before the winter to include other groups like those working in the transport and food industry, he said.

The identity of the shots cleared for emergency use isn’t yet clear. Several Chinese companies have started clinical trials of coronavirus vaccine candidates, and three shots based on inactivated SARS-CoV-2 from Sinovac and Sinopharm are heading for phase 3 testing, according to the World Health Organisation’s latest update on vaccine progress.

Another adenovirus-based vaccine from CanSino Biologics is also in late-stage development and has been approved for use in military personnel, according to the South China Morning Post.

Meanwhile, candidates from Sinopharm’s China National Biotec Group (CNBG) subsidiary are also heading for phase 3 trials outside China – namely in Peru, Morocco, Bahrain, United Arab Emirates and most recently Argentina – after getting approvals to start testing by regulators.

Faced with dwindling numbers of COVID-19 cases in China, CNBG is starting up studies of its inactivated vaccines overseas where outbreaks of the virus are still in full swing. China has gone several days without a locally-transmitted case of the infection.

One has been developed in collaboration with the Wuhan Institute of Biological Products, while the other is partnered with the Beijing Institute of Biological Products. Both have cleared phase 1/2 trials which according to Sinopharm showed they were safe and generated “high-titre” antibody responses against SARS-CoV-2.

China has said that it will prioritise access for countries from Africa, Latin America and the Mekong region – Myanmar, Laos, Thailand, Cambodia and Vietnam – when the vaccine is ready to be rolled out overseas.

Professor Chris Whitty, the UK’s chief medical officer, said over the weekend that he believes it is unlikely that a coronavirus vaccine will be widely available before winter 2021.

Meanwhile, the Financial Times has reported that the Trump administration is considering fast-tracking emergency-use approval of the ChAdOx1 adenoviral vaccine developed by the University of Oxford and AstraZeneca for use in the US ahead of the upcoming presidential election, after being slammed for his handling of the crisis.

That has been denied by the Department of Health and Human Services, but the pandemic continues to be a focus for political wrangling ahead of the election.

On Saturday, Trump tweeted a bizarre accusation that the FDA was deliberately holding up the development of vaccines and drugs for COVID-19 for political reasons.

FDA Commissioner Steve Hahn didn’t choose to respond to that assertion, which came in the build-up to the emergency use authorisation of convalescent plasma as a treatment for COVID-19 yesterday.

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Sorrento Files IND for STI-1499 (COVI-GUARD) in Patients Hospitalized with COVID-19

Shots:

  • Sorrento has filed an IND for STI-1499 in patients hospitalized with COVID-19. In preclinical studies, STI-1499 has demonstrated 100% neutralizing effect (in vitro) against the highly contagious D614G mutant strain at a low dose
  • Additionally, animal model data confirms the neutralizing profile and high potency of Ab, expected effective dose in human to be at least 5 times lower than current known Abs being assessed in other active trials
  • Sorrento has initiated cGMP manufacturing to produce 50,000 doses, expected to be available by the end of 2020, in anticipation of a potential EUA

Click here ­to­ read full press release/ article | Ref: Sorrento | Image: Blogger

Government under fire over plan to scrap Public Health England

Health Secretary Matt Hancock is planning to dissolve Public Health England and replace it with a new agency dedicated to responding to pandemics, according to reports.

The plan is to implement a new body modelled on the independent Robert Koch Institute in Germany, which has been held up as having done well in weathering COVID-19, in part at least because of a well-organised and large-scale testing programme early on.

PHE has come under fire during the crisis for failing to ramp up testing quickly enough and issues with its methods for counting deaths, for example including everyone who had tested positive even if they recovered or died of another cause.

The Sunday Telegraph suggested that PHE will be merged with the NHS Test and Trace service into a new Institute for Health Protection, with responsibility for other parts of its operations such as responding to rising levels of obesity handed to local authorities and doctors.

The move – which could be officially announced as soon as tomorrow in a speech by Hancock – has been met with anger by critics who say that the government is making PHE a scapegoat for its own failings in handling the coronavirus crisis.

The British Medical Association issued a statement saying that PHE “must not shoulder the blame for wider government decisions.”

Dr Chaand Nagpaul, chair of BMA UK Council said: “With more than 1,000 new UK cases of Covid-19 being recorded for the fifth day in a row, we must seriously question whether now is the right time for undertaking such a seemingly major restructure and detract from the very immediate need to respond to the pandemic.”

PHE was only set up in 2013 by former Health Secretary Jeremy Hunt, and has civil service status so operates under direct ministerial control. In particular, it’s worth noting a controversial decision to halt community coronavirus testing and tracing in March was taken in consultation with ministers and government medical advisors.

Shadow health secretary Jon Ashworth said the problems with PHE are “ministerial failures whatever Tory MPs say”.

At the time PHE was set up, the government said its objective was to streamline public health advice and services and create a powerful single entity akin to the US Centres for Disease Control and Prevention (CDC).

Public health experts – including chief medical officer Prof Chris Whitty – have said that PHE has been affected by underfunding over recent years as a result of the government’s decade-long austerity drive, which has led to a big reduction in headcount.

The BBC says it has seen a leaked memo written by PHE head Duncan Selbie that says the new Institute will boost expertise and attract “much needed new investment.”

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UK medical journals spar over breast cancer screening study

Screening women for breast cancer in their 40s rather than their 50s could save lives, say the authors of a new Lancet Oncology study, although that finding has been challenged by other experts.

Lowering the age that women start to routinely get mammograms was associated with a relative reduction in breast cancer mortality without leading to ‘over-diagnosis’ – in other words detecting minor changes that may not turn into full-blown cancers – according to the scientists.

The UK currently offers mammograms every three years to women aged 50 to 70, but in this trial – led by Prof Stephen Duffy of Queen Mary University in London – 160,000 women aged 40 or 41 were randomised to either once-yearly mammograms or no screening.

The best timing for screening is a hotly-debated topic, and the British Medical Journal ran an article challenging the Lancet Oncology findings on the day of publication, citing experts who dismissed the findings as “misleading rhetoric.”

Cancer Research UK also chimed in, saying that even with the new study it is still unclear if reducing the breast screening age “would give any additional benefit compared to the UK’s existing screening programme.”

It found an initial reduction in breast cancer deaths from screening, but this disappeared over time, according to the charity, which believes more effort should be devoted to restoring access to current screening and other cancer-related NHS services that have been disrupted by the coronavirus pandemic.

The Lancet Oncology paper found that after almost 23 years of follow up, there were 83 breast cancer deaths among the almost 54,000 randomised to the screening group, versus 219 among more than 100,000 in the no-screening control group.

The results are similar to those revealed 15 years into the follow-up period, but over the longer term there was little difference in mortality between the groups. Overall, the number of deaths per 10,000 women was 16 in the screening group and 21 for the controls.

The benefit was seen mostly in the first 10 years, but the reduction in breast cancer mortality was maintained in the long term at about one life saved per 1,000 women screened, according to the researchers.

Vinay Prasad, associate professor at the University of California, San Francisco, told the BMJ: “’Saves lives’ means that women, as a result of doing this, live longer than those who do not do it.”

He went on to say that “did not occur in this dataset” adding that in fact it was “quite the opposite,” given that all-cause mortality data found no difference between the groups.

The study recruited women from the late 1990s, when mammography may not have been as effective as today. On the other hand, the cost of extending the screening programme could be significant and could be hard to justify given the small reductions in breast cancer deaths recorded.

“Compared to the existing screening programme, in the younger age group, six times more women would need to be screened to save one life,” said CRUK.

“Many women received false positive results and some women would have been over-diagnosed with cancers that would never have gone on to cause them harm,” it added.

Image by Elías Alarcón from Pixabay

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Strata Oncology Collaborates with Mirati Therapeutics to Broaden Enrollment in Clinical Trial of MRTX849 for Patients with Advanced Solid Tumors

Shots:

  • The multiple expansion cohort will assess the safety, tolerability, drug levels, molecular effects, and clinical activity of MRTX849 in patients with advanced solid tumors harboring a KRAS G12C mutation
  • Strata Oncology will identify patients to be considered for enrollment into Mirati’s P-I/II study. The Strata Trial provides tumor molecular profiling for patients with advanced cancer paired with a portfolio of biomarker-guided clinical trials
  • Strata Oncology utilizes StrataNGS which is a molecular profiling test optimized for performance on tumor tissue samples as small as 0.5mm2 surface area. MRTX849 is a novel KRAS G12C selective inhibitor

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

Novo Nordisk Resumes P-III Study of Concizumab for Patients with Hemophilia A and B

Shots:

  • The company resumes the P-III program (explorer6, 7, and 8) of concizumab. The clinical trials are evaluating concizumab (SC) prophylaxis treatment in hemophilia A and B patients regardless of their inhibitor status
  • The trials will be resumed as soon as local procedures allow. This follows pausing of the trials in Mar’20 due to the occurrence of non-fatal thrombotic events in 3 patients enrolled in the ongoing P-III program
  • In Oct 2019, Novo Nordisk has initiated the explorer7 study to establish the safety and efficacy of concizumab (SC, qd) delivered in a pen device to reduce the number of bleeds in patients with hemophilia A or B with inhibitors towards FVIII/ FIX. In Nov 2019, the company initiated P-III explorer8 study in hemophilia A or B patients without inhibitors. The trials are to enroll ~293 patients across 32 countries

Click here ­to­ read full press release/ article | Ref: Novo Nordisk  | Image: Scrip Informa

Regeneron Reports the US FDA’s Acceptance of Evinacumab’s BLA for Priority Review as a Treatment for Patients with HoFH

Shots:

  • The BLA is based on P-III study evaluating the efficacy and safety of evinacumab (15 mg/kg, IV, q4w) in 65 patients aged ≥12yrs. with HoFH. The 1EPs of the study is reduction of LDL-C from baseline
  • The expected PDUFA date for the therapy as Feb 11, 2021. The US FDA has granted BT designation to the therapy for the treatment of hypercholesterolemia in patients with HoFH in 2017
  • Evinacumab is an investigational mAb that binds to and blocks the function of ANGPTL3 and is currently being studied in patients with HoFH (ongoing P-III extension trial), refractory hypercholesterolemia (P-II) and severe hypertriglyceridemia (P-II)

Click here to­ read full press release/ article | Ref: Regeneron  | Image: CNN

Roche Collaborates with Celleron Therapeutics for Emactuzumab to Treat Patients with Tenosynovial Giant Cell Tumor

Shots:

  • Celleron will receive an exclusive global right for the clinical development, manufacturing, and commercialization of emactuzumab (formerly RG7155 and RO5509554). The companies expect the closing of an agreement by the end of 2020
  • 86% of TGCT patients treated with emactuzumab responded to the drug, with 68% of people experiencing a PR within 6 weeks of starting the treatment. The license agreement demonstrated Celleron’s dedication to the development of cancer medicine and its transformational potential for patients inflicted with cancer
  • Emactuzumab (RG7155) is an IgG1 mAb, designed to target and deplete macrophages in the tumor tissue.  The therapy targets TAMs by binding to CSF-1R on the cell surface and blocking its activation by CSF-1

Click here ­to­ read full press release/ article | Ref: Celleron Therapeutics | Image: PharmaShots

Roche Report Mixed Results of Etrolizumab in P-III Studies for Patients with Moderately to Severely Active Ulcerative Colitis

Shots:

  • In the HIBISCUS I induction study, in patients without prior anti-TNF treatment, etrolizumab met its 1EPs while in HIBISCUS II study in the same kind of people, it did not meet its 1EPs. In the HICKORY study, in patients with prior anti-TNF treatment, therapy met its 1EPs at induction but not at maintenance
  • In the LAUREL maintenance study in people without prior anti-TNF treatment, etrolizumab failed to meet its 1EPs while the safety profile of therapy was consistent with previous studies and no major safety issues were reported till date in the four P-III studies
  • The therapy is currently being evaluated as an induction/maintenance treatment in people with mod. to sev. active CD with/out prior anti-TNF treatment in a global P-III study (BERGAMOT) and OLE and safety monitoring study (JUNIPER), involving 1,100+ people with CD. In addition, Roche is studying other therapies in IBD and is committed to further understanding this disease

Click here ­to­ read full press release/ article | Ref: Roche | Image: Pantone Canvas Gallery

Bad behaviour: why understanding human behaviour can improve outcomes

Why did I do that? A common question I often ask myself after opening the biscuit packet for the third time in an hour. Only ten minutes before I had a mental argument with myself, which I thought I had won, promising not to eat another one until tomorrow. The process that goes on inside our heads, affecting the good and bad choices we make regarding our health every day, is the focus of health psychology and the growing interest around behavioural change.

Research on the social determinants of health indicates that our behaviour accounts for up to 38% of our health status and that we should be looking to broader aspects of health as medical care represents only 11%. This research also shows the complex system that drives our health outcomes, including the environment, our genetics and our social circumstances.

Our behaviour patterns shape the conscious and unconscious decisions we make about our health and are often an indicator of future health outcomes. Therefore, understanding why we exhibit bad health behaviour is an essential part of planning healthcare interventions, and this is especially true for chronic or long-term diseases.

Human behaviour underpins our actions towards medication adherence, physical activity, tobacco and drug use. It affects our sleep patterns, shopping behaviour, hand washing and the way we monitor ourselves for changes in health or disease indicators.

Outside of direct effects on our health behaviour, it also affects health through other people’s decisions. For health workers, choices change the way we interpret new clinical information and the speed at which we adopt new clinical standards. The behaviour of care workers and their decisions also affects individual care. As the social determinants of health help visualise, behaviours should be viewed as part of a system, and therefore when we attempt to change behaviour our interventions need to address the interplay of factors that underpin current actions.

“Great leaps have been made in patient outcomes – but further growth will require us to use a deeper understanding of behaviour to make these gains more sustainable”

Great leaps have been made in the improvement of patient outcomes – but there’s a growing realisation that further growth will require us to use a deeper understanding of behaviour and how to change it to make these outcome gains more sustainable.

The most recent health strategies aim to build an approach to health based on the Four P’s of disease:

  • Prevention: preventing a disease or reversing illness before it has an irreversible effect on health
  • Prediction: knowing when a disease will have an impact on health and providing interventions beforehand
  • Precision: moving to interventions that target diseases specifically instead of broadly
  • Personalisation: creating interventions based on individuals knowing their genetics, biology and psychology as well as their social and geographical environment.

While great progress has been made in diseases like diabetes, where HbA1c can be controlled for the majority of patients, we can still improve health outcomes further by providing better monitoring of disease and through more personalised drug delivery.

Advances in digital tools and medical devices have also enabled better outcomes through better decisions. More recently, work on understanding the drivers of positive health determinants like food choice, smoking cessation, and increased physical activity have started to add to overall health improvements for diabetic patients. Our understanding of the basis of behaviour and the choices we make are moving us away from the reliance on information and education as the prime intervention. More systematic approaches to understanding behaviour and the interventions that support change appear in the literature. In part, this change is fueled by recent research but also from advances in digital tools and the ability to access people’s actions through devices like smartphones.

In a previous article I argued that behavioural science capabilities are a new skill that pharma needs to master. This is not the first time that pharma has tried to go ‘Beyond the Pill’. Ten years ago, a wave of digital health departments started focusing on the capabilities of new digital media. mHealth followed, with the advent of connected systems and the rise of the Internet of Medical Things (IoMT). Recently a more considered approach to digital medicine and digital therapeutics has taken over. Prescription Digital Therapeutics (PDTs) are appearing as both a companion and a replacement to some drugs. Companies like Pear, Omada, Wealthy, Noom, Lovingo and Welldoc all provide solutions to help patients improve health outcomes, most of them having a component of behavioural change.

There are many models of health behaviour that attempt to both explain the origin of current behaviour and plan for interventions to support change in behaviour. A review of the literature shows common approaches include the Trans Theoretical Model, the Theory of Planned Behaviour and COM-B. Each of these and the other theories have their place in helping health providers plan for change.

COM-B is an accessible approach to behaviour change developed by Michie et al. at the Centre for Behavioural Change, UCL in London. It draws from 93 other models to create a unified approach to understanding and changing behaviour. COM-B is an acronym for the drivers of behaviour, with people needing the (C) capability to perform the desired behaviours as well as the (O) opportunity and the (M) motivation. This approach has had successful implementation in many settings, such as smoking cessation, hand washing, medication adherence and others.

Remembering that behaviours occur in systems helps us use tools like COM-B to create health interventions that are more holistic and consider the underlying cause of behaviours that may need to be changed to improve the target outcome. COM-B asks if the person has the capability of performing the behaviour. Enablers of this are both physical and psychological. For example, in physical activity, there may be a physical limitation, through skill, strength or stamina. Psychologically there may be a need to inform or educate to enable the behaviour.

Going back to the physical activity example, we may need to explain to patients what we mean by physical activity to ensure they can execute the behaviour. COM-B provides a validated taxonomy of interventions that may support patients’ capability to perform the behaviour more frequently.

Beyond capability, patients also need the opportunity to perform the planned behaviour. Patients’ opportunity can be viewed as having the right resources and environment to perform the behaviour. There are also social opportunities that arise from those around us that influence our thinking and actions, as well as the cultural norms that affect the way we think.

Finally, we must also understand patients’ motivation to perform the desired behaviour. For example, what motivates patients to check blood glucose or to take medication? Our motivation systems are complex, and there is much research in this area. In terms of COM-B, we can think of reflective motivation, which encompasses the plans we make and how we evaluate our choices. How much do we value the task of performing the behaviour? The second system of motivation is our automatic motivation with processes involving emotional reactions, desires (wants and needs), impulses, inhibitions, drive states and reflex responses.

Behavioural change frameworks help teams in pharma, medical device, and digital therapeutic companies create programmes to improve health outcomes. These frameworks create intervention plans that support pharmacotherapy and device use. They are also useful tools for looking at brand plans to understand if the planned tactics can support the type of behavioural change intended.

For many diseases, the next level of health innovation will require advanced chemistry, formulation or delivery (in other words, what pharma companies are good at today) along with insights into behaviour and how to change it, as well as how to deliver behavioural change at scale. Not all our behaviours or choices are bad, but there is room for improvement for all of us, and we need all the behavioural support we can get.

About the author

Mark Lightowler is the CEO of Phorix, a behavioural change design agency. They work with patients, physicians and pharmaceutical companies to improve health outcomes through behaviour change design and are based in Basel and London.

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Roche’s Tecentriq (atezolizumab) + Paclitaxel Fail to Meet the Primary Endpoint in P-III IMpassion131 Study for Patients with Metastatic Triple-Negative Breast Cancer

Shots:

  • The P-III IMpassion131 study involves assessing of Tecentriq + paclitaxel vs PBO + paclitaxel, in 651 people in a ratio (2:1) with previously untreated, inoperable, LA/ m-TNBC
  • The study did not meet its 1EPs of PFS for 1L treatment of people with m-TNBC) in the PD-L1+ population.  The data for 2EPs of OS showed a negative trend, however, the study was not powered for the 2EPs of OS and data were immature at the time of analysis while OS follow-up is planned to continue until the final analysis
  • In the previous IMpassion130 study, Tecentriq + Abraxane demonstrated PFS benefit and, while not formally tested, showed improvements in OS for people with metastatic TNBC whose tumors express PD-L1

Click here ­to­ read full press release/ article | Ref: Roche | Image: Forbes

Related News: Roche’s Tecentriq (atezolizumab) + Abraxane Receive European Commission’s Approval for Patients with PD-L1-Positive Metastatic Triple-Negative Breast Cancer




UCB’s Cimzia (certolizumab pegol) Receives the EMA’s Approval for a Reduced Maintenance Dose in Patients with Axial Spondyloarthritis Spectrum

Shots:

  • The P-IIIb C-OPTIMISE study assessing Cimzia (200mg, q2w with a loading dose of 400mg @ 0, 2 & 4wks.) vs PBO during 48wks. open-label induction period in adults with early active axSpA. At 48wks., patients in sustained remission (ASDAS <1.3 @wks. 32/36 & 48) were randomized to Cimzia 200mg, q2w (full maintenance dose) & 200mg q4w (reduced maintenance dose) or PBO (withdrawal) for an additional 48wks.
  • The EMA label extension is based on the results of the C-OPTIMISE study that demonstrated @48wks. 43.9% of patients achieved sustained remission, @96wks. 84%, 79% & 20% of patients receiving the full maintenance dose, reduced maintenance dose or PBO respectively remained flare-free
  • The approval makes Cimzia the only biologic in EU with a dose reduction option in its label for patients in the broad axSpA population

Click here ­to­ read full press release/ article | Ref: UCB | Image: CHE Manager




Lundbeck Halts P-II Study of Lu AF11167 in Patients with Negative Symptoms of Schizophrenia

Shots:

  • The company discontinues P-II proof POC study of Lu AF11167 in patients with schizophrenia, who are experiencing persistent negative symptoms (NCT03793712). The P-II study evaluated two doses of Lu AF11167 vs PBO as monothx. in patients with schizophrenia and persistent prominent negative symptoms
  • The discontinuation is based on the results of a futility interim analysis, which concluded that trial fails to achieve 1EPs i.e. change from baseline to 12wk on BNSS. The recommendation to halt the trial is not based on safety concerns
  • Lu AF11167 is a potent PDE10Ai inhibitor, modulating dopamine D1 and D2 receptor-mediated intraneuronal signaling without binding to these receptors. The company plans to randomize 240 patients from EU countries

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




Post-viral syndrome: Learning to live with COVID-19 on the NHS

Recent months have exacerbated many of the issues that were facing the NHS before anyone had ever heard the word COVID-19 – and the scars are set to remain long after the country gets “back to normal”.

In the absence of a vaccine or a reliable SARS-CoV-2 test, the UK could be operating a middle-income country health system with all the expectations and regulatory standards of a high-income one.

That was the stark warning of a Nuffield Trust discussion paper, Here to stay? How the NHS will have to learn to live with coronavirus.

“As the number of Covid-19 hospital admissions gradually declines, policy attention is turning to how the NHS can restart some more routine activities, with hospitals beginning to resume elective surgery and cancer treatments,” said the paper’s author, Nuffield Trust chief executive, Nigel Edwards.

“But doing this while living alongside COVID-19 will involve major practical challenges that will need to be overcome.”

Re-starting services and tackling the backlog that has built up in the last few months while maintaining social distancing and minimising risk for staff, all within the context of a health service that was already in crisis, is a mammoth task.

COVID-positive until proven otherwise

The paper, based on conversations with representatives from different parts of the NHS, including a teaching hospital, single and multi-site hospitals, community services and primary care, sets out a litany of challenges.

Among these is that, without a quick or reliable test, health and care staff will have to assume all patients are infected until proven otherwise.

In emergency care, which was already struggling to cope with demand, that means enhanced PPE and extra time for cleaning beds, imaging equipment, and operating theatres between patients.

Patients attending for planned surgery will need to be swabbed, told to self-isolate, then swabbed again, bogging down the burgeoning waiting lists for even the most basic processes and treatments.

Group interventions, from peer support to cardiac rehabilitation, will also need to be redrawn, at the same time as developing new services for those on the long road of COVID recovery.

Said Edwards: “The waiting list for planned treatment was already over 4m going into the COVID-19 outbreak, and at that point approximately 1.6m people a month were starting new care pathways. Most of these people have not had their treatment moved forward since the end of March.

“This combined with the effect of the overall productivity losses… means there will be very large increases in the numbers of patients waiting for treatment once GP referrals return to more usual levels.”

“The NHS has a lack of single patient rooms, narrow corridors and cramped waiting areas that do not allow for distancing or separate organisation flows for infectious patients”

Structural challenges

COVID served to compound many of the issues the NHS was facing before the pandemic hit, such as overburdened A&E services, staff shortages, and buildings that were not fit-for-purpose.

Said the paper: “The aim of good hospital architecture has generally been to create ‘loose fit’ design and generous circulation space to aid the flow of patients through buildings.

“Unfortunately, the NHS has not always followed this and there has been a history of eliminating much of this space in NHS building schemes to reduce costs since the 1970s.”

The result is a lack of single patient rooms, narrow corridors and cramped waiting areas that do not allow for distancing or separate organisation flows for infectious patients.

While reductions in emergency department attendances at the start of the pandemic provided some respite and space for people with COVID-19, it also created a backlog of health problems, and demand will soon begin to rise.

“It will not be safe to return to previous levels of activity due to the physical space constraints… The urgent care system will therefore need to consider other ways of managing overall demand for care and spreading work more evenly across the day,” said the paper.

It’s also worth noting that the NHS went into the crisis with a large number of vacancies and shortages of key staff, it went on.

“Some staff have returned from retirement to assist with the pandemic response, but it is not clear whether they will want to stay once ‘usual’ work returns.

“There is some concern among health leaders we spoke to that, given the recent experience of some staff, there will be increased sickness and that people close to retirement or considering leaving will bring their decision forward.”

The need to protect black, Asian and minority ethnic staff, who have higher levels of mortality, is also of concern to rota planning, particularly in some organisations with a high proportion of BAME frontline staff.

Future policy

There is no doubt that the rapid changes made by the NHS were key to the UK avoiding the devastation seen when healthcare systems in Lombardy and New York became overwhelmed by the crisis.

But as we tentatively get back to normal – whatever that might be – we have to accept that the COVID response will have long-term complications for our healthcare system.

As Edwards said: “This could mean the public having to accept reduced services, health and care staff facing continued and long-term changes to their ways of working, and difficult choices ahead for policymakers in accepting a degree of rationing of healthcare that would previously have been seen as unacceptable.”

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Playing an Active Role in Treatment Yields More Confidence in Patients

When aiming to reach the people who will benefit most from a given medication or therapy, patients who play an active role in treatment decisions are often at the forefront of the conversation. But what does playing an active role in treatment really mean?

While there is no concrete definition, a meta-analysis of Health Union’s 2019 large scale, condition-specific In America surveys uncovers certain attitudes and behaviors associated with this mindset.

Nearly 7 in 10* respondents across 18 online health communities (n=30,183) agreed with the statement I play an active role in deciding about treatment for my condition. Demographically, those who agreed were more likely to be female (86%), more likely to be between the ages of 40 and 64 (57%) and more likely to have group health insurance coverage (44%).

To read the full article and understand how Health Union’s knowledge can help you make smarter, more effective marketing decisions, visit health-union.com.

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Government warned over test and trace as UK schools prep to reopen

The current level of test and trace in the UK is inadequate to prevent a sharp increase in coronavirus infections when schools reopen in September and there is further relaxation of social distancing rules, according to scientists.

A second wave of COVID-19 could be avoided if enough people are tested and traced, according to the team from University College London and the London School of Hygiene & Tropical Medicine.

That would need a testing rate for people showing symptoms of coronavirus infection of between 59% and 87% however – along with effective tracing of contacts and isolation, they write in The Lancet Child and Adolescent Health.

Labour leader Keir Starmer claims in The Guardian however that the government’s much-vaunted test and trace system simply isn’t up to the job, and the country will face a “long bleak winter” if it doesn’t improve within the next month.

According to the UCL and LSHTM scientists, if tracing is 40% effective testing would have to be at 87% to prevent a sharp increase in transmissions in early September.

At the moment the test and trace system is claimed to be 68% effective at finding contacts, and that would require a testing level of 75%, according to the researchers’ model. They also don’t believe that the UK system is working at that level of efficiency.

“We need to scale up current TTI [test-trace-isolate] strategies to avoid COVID-19 resurgence later this year,” according to lead author Dr Jasmina Panovska-Griffiths of UCL and Oxford University.

Otherwise, the UK will face a secondary wave that would peak in December that could be at least twice the size of the original COVID-19 outbreak, the researchers predict.

Starmer says that Labour has been a “constructive opposition” during the crisis, but can no longer ignore that Boris Johnson’s government “has been too slow to act throughout this crisis – too slow into lockdown, too slow on testing and too slow getting PPE to frontline workers.”

What is needed is a rapid improvement in TTI and a ramping up of testing among the 70% to 80% of people who don’t have symptoms, he writes in the Guardian column.

The head of the NHS test and trace scheme – Baroness Dido Harding – insisted to the BBC today that the system is delivering tracing rates that are “well within the bounds” of what the researchers described as necessary in the research paper.

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Tackling multimorbidity through collaboration

As populations age, multimorbidity becomes an increasingly pertinent issue for healthcare systems. A new action group from the ABPI seeks to bring the NHS, academia and industry together to find new ways to address the problem. The Association’s Dr Sheuli Porkess and Professor Janet Lord from the University of Birmingham tell us more.

Multimorbidity is an increasing issue for healthcare and the NHS. In a recent analysis of data from 2016 at the Queen Elizabeth Hospital Birmingham, 75% of unplanned admissions, which account for almost 30,000 admissions per year, were for older adults. 83% of these adults had three or more conditions. There was no dominant condition that triggered admission to hospital – although falls, delirium and breathlessness were the three most frequent.

In the hopes of raising awareness about the challenge of multimorbidity and working collaboratively to address it, the ABPI has established a Multimorbidity Action Group to develop policy and move the discussion forward – which is now open to join.

“Doctors can often see patients with a cocktail of conditions all needing treatment at the same time, with few options but to treat each condition individually with multiple medicines or other interventions, which isn’t ideal if one interacts badly with another,” says Dr Sheuli Porkess, executive director of research, medical and innovation at the ABPI. “You can inadvertently cause side effects which bring an already-ill person yet more discomfort.

“In the ABPI, we want to harness support from industry to dig into the problem of multimorbidity.  It’s a wicked problem, particularly when you consider the research process for new medicines.”

For example, the Association has entered into a new partnership with Birmingham Health Partners (BHP) – a strategic alliance between the University of Birmingham and two NHS Foundation Trusts – to tackle the biggest health challenges faced by the West Midlands’ six million residents, one which is multimorbidity in an ageing population.

“Currently the medical profession and pharma industry are not addressing the issue of multimorbidity with any vigour”
Janet Lord, University of Birmingham

“If we are really going to tackle the issues that we are facing with our ageing population, we have to understand the biological drivers of multimorbidity with advancing age acknowledged as the major risk factor,” says Professor Janet Lord, director of the Institute of Inflammation and Ageing at the University of Birmingham.

Multimorbidity is defined as the presence of two or more conditions in an individual. Importantly, multimorbidity occurs as clusters of diseases, and whilst there are different views on what the clusters are and how many exist, Lord says that most would agree there are three main clusters:

  • Cardiovascular/metabolic – including heart disease, hypertension and Type 2 diabetes
  • Musculoskeletal – including arthritis and osteoporosis
  • Neuropsychiatric – including mental health conditions and dementia

“Currently the medical profession and pharma industry are not addressing the issue of multimorbidity with any vigour,” she adds.  There are many reasons for this. At the moment we treat the component conditions of multimorbidity individually, which leads to multiple clinical consultations resulting in a burden on both the patient and the NHS.

“Secondly, this single-disease focus leads to polypharmacy, and we still don’t understand all of the drug-to-drug interactions that this older multimorbid patient will be experiencing and the impact on their health.

“Thirdly, our current drug development and testing programmes are also single disease focused, with many trials excluding the multimorbid patients they will most likely be prescribed to.”

Porkess adds that there are many other challenges in researching drugs that take into account multiple conditions.

“Researching a new medicine is a highly regulated process,” she says. “It is essential to be able to demonstrate to the regulator that any potential new medicine is safe and works for the condition being targeted.

“That means when you test a potential medicine in clinical trials, the traditional model is that it is tested on people who have the disease you are targeting, and only that disease.

“If there is a change to that person’s condition during the trial, you can be more confident that the potential medicine is making a difference. The presence of multiple conditions makes the evidence less clear.”

The fact that multimorbidity frequently occurs in older people adds extra challenges as it can be more difficult to recruit older people into clinical trials and harder to help them stay in the trial for the whole duration of the study.

“These are structural issues in research that can be addressed,” says Porkess, “and we are determined to think creatively, with our partners in Birmingham and others, on how to do that.”

Lord agrees that the NHS, academia and industry need to work together to have a “completely new approach” to multimorbidity.

“Instead of continuing to develop drugs for a single disease we should be looking at tackling the generic biological drivers of these multimorbidity clusters.

“As age is the single biggest risk factor for multimorbidity, an obvious starting point is to target the biological processes that underlie the ageing process itself1. Until recently this would not have been possible, but we now understand what these processes are2 and animal studies have shown that if you inhibit core ageing processes such as cell senescence you can prevent many age-related conditions including sarcopenia, cognitive decline and cardio-respiratory compromise.

“This field is moving fast and the first trials in humans were reported in 2019 showing that intermittent dosing with repurposed drugs that kill senescent cells reduced physical frailty in patients with Idiopathic Pulmonary Fibrosis3.”

She adds that this new approach would include uniting the comprehensive health data and patients in the NHS; the understanding of the biological drivers of multimorbidity clusters in academia; and the drug discovery capabilities of industry.

“We can accelerate drug discovery and clinical trials aiming to prevent or treat age-related multimorbidity and the associated polypharmacy.”

“By bringing the NHS, academia and industry together we can create a completely new approach to multimorbidity,” adds Porkess, “which could change the lives of millions of people in the UK.”

The ABPI’s Multimorbidity Action Group is open for companies and other organisations to join. Contact Su Jones ([email protected]) for more details.

References

  1. Ermogenous C, Green C, Jackson TA, Ferguson M, Lord JM (2020) Treating age-related multi-morbidity: The drug discovery challenge. Drug Discovery Today. https://doi.org/10.1016/j.drudis.2020.06.016
  2. López-Otín C, Blasco MA, Partridge L, Serrano M, Kroemer G. The hallmarks of aging. Cell 2013; 153(6):1194–1217.
  3. Justice JN, Nambiar AM, Tchkonia T, et al. Senolytics in idiopathic pulmonary fibrosis: Results from a first-in-human, open-label, pilot study. EBioMedicine. 2019; 40:554–563.

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