Pharmaceutical wholesaler Lexon (UK) has lost its appeal against a £1.2 million fine in a case that accused it of exchanging commercially sensitive information about prices for nortriptyline tablets.
Lexon had appealed against a ruling by the UK’s Competition and Markets Authority (CMA) that it had exchanged information with King Pharmaceuticals and Alissa Healthcare Research in order to keep the price of nortriptyline – an antidepressant – as high as possible in breach of competition law.
“From 2015 to 2017, when the cost of the drug was falling, the three suppliers exchanged information about prices, the volumes they were supplying, and Alissa’s plans to enter the market,” said the CMA.
That period was preceded by several years in which the price of nortriptyline had been rising dramatically, with NHS spending on the drug escalating from just over £6 million in 2011 to £38 million in 2015.
The exchanges consisted of email messages, phone calls and a meeting in a London hotel, according to the tribunal documents, aiming “to maintain, or slow the decline in, the price of nortriptyline”.
King Pharma was fined £75,573 and Alissa £174,912, with both of the companies given reduced fines in return for admitting the infringement. Lexon held out and denied the charges, and so the CMA imposed a £1.2 million fine, which Lexon appealed.
The decision this week by the Competition Appeal Tribunal (CAT) dismisses all of Lexon’s grounds for appeal.
The wholesaler had argued that the CMA had wrongly interpreted the law, misconstrued the content and purpose of the information exchanges and failed properly to take account of their economic context, and had set its fine too high.
One of Lexon’s senior figures – Pritesh Sonpal – now faces a ban on serving as a company director. That application – which could lead to disqualification for up to 15 years – is currently being heard at the High Court of Justice.
Sonpal currently holds 15 active directorships, according to Companies House records, including Lexon (UK), Pure Health Medical, LTI Pharma, and Knights Pharmacy.
“We welcome the decision… to dismiss Lexon’s appeal in its entirety and support the findings of our investigation, including our decision to fine the firm over £1.2 million,” said CMA chief executive Andrea Coscelli.
“Lexon illegally exchanged competitively-sensitive information to try and keep prices up, meaning the NHS – and ultimately the UK taxpayer – could have been paying over the odds for this important drug. Such behaviour is unacceptable,” he added.
“We will continue to crack down on companies that seek to break the law and will be keeping a close eye on this sector.”
The UK drugs regulator has approved Seagen’s Tukysa as a third-line treatment for HER2-positive breast cancer, shortly after it was given a green light by the European Commission.
Tukysa (tucatinib) – an orally-active HER2 inhibitor – can be used in combination with anti-HER2 antibody trastuzumab and chemotherapy capecitabine in patients treated with two prior anti-HER2 regimens, according to the MHRA.
It’s the third oral HER2 drug to be approved for use in the UK and Europe, but could be the first with a chance to generate blockbuster sales, according to analysts.
As a result of Brexit, the MHRA approval applies to England, Scotland and Wales, while the European Commission has cleared use of the drug in Northern Ireland, which remains under its regulatory remit for the remainder of this year.
According to Seagen – known as Seattle Genetics until a name change last year – Tukysa is the first HER2 tyrosine kinase inhibitor shown to improve overall and progression-free survival (PFS) in previously treated HER2-positive breast cancer.
The approval is based on the HER2CLIMB study, which involved patients who had previously been treated with Roche’s trio of HER2 drugs Herceptin (trastuzumab), Perjeta (pertuzumab) and Kadcyla (trastuzumab emtansine).
The Tukysa arm demonstrated an improvement in overall survival, with a 34% reduction in the risk of death compared to trastuzumab and capecitabine alone.
In addition, out of the 47% of patients whose cancer had spread to the brain in the study, Seagen’s drug cut the risk of disease progression or death by 52%, thanks to its ability to cross the blood-brain barrier and penetrate the central nervous system.
Seagen’s chief executive Clay Siegall said the company will now “look forward to further collaborating with the national reimbursement bodies to ensure it is available to adult patients” in the UK.
The first oral HER2 drug to reach the market was Novartis’ Tyverb (lapatinib), which is generally reserved for use after HER2 and chemotherapy-based treatment has failed, and is a minor product in Novartis’ oncology portfolio.
Tyverb – known as Tykerb in the US – was followed a couple of years by Puma Biotech/Pierre Fabre’s Nerlynx (neratinib), which is also approved for a different indication. The drug is given as an adjuvant therapy after surgery for early-stage HER2-positive breast cancer to reduce the risk of the disease coming back, and made around $211 million in revenues for Puma last year.
Tukysa was approved by the FDA almost a year ago, and at that time analysts at Leerink predicted sales of the drug could reach $1.6 billion thanks to wording on the US label that allowed second- or third-line use of the drug.
They reckon that Tukysa’s ability to treat tumours that have spread to the brain will help it capture market share from Kadcyla, a go-to second-line therapy.
Seagen is already running trials to expand the use of the drug into earlier lines of breast cancer therapy – including adjuvant and neoadjuvant use – as well as in other solid tumours including colorectal cancer.
Sometime within the next few weeks, young and healthy volunteers will be deliberately exposed to the COVID-19 coronavirus in the UK in what looks set to be the first study of its type worldwide.
The human challenge study has just secured ethics committee approval and will recruit up to 90 volunteers aged 18-30 years, according to the UK government, which is backing it with £33.6 million ($47 million) in funding.
The volunteers will be infected in a controlled environment where they can be carefully monitored by medical staff 24 hours a day.
The researchers will also inoculate them with the version of the virus that has been circulating in the UK since the start of the pandemic, rather than one of the new, more transmissible variants, as an added safety precaution.
The aim of the study is to find the smallest amount of SARS-CoV-2 that is needed to cause an infection, see how the immune system reacts to the virus, and to assist in the development of new treatments and vaccines.
In particular, the study will aid in the design of other challenge studies that could be used for vaccine efficacy testing, and to determine what type of immune response is required to protect against re-infection. It will also examine how a person who is infected with the COVID-19 virus transmits infectious virus particles into the environment.
Dr Chris Chiu
“Our eventual aim is to establish which vaccines and treatments work best in beating this disease, but we need volunteers to support us in this work,” said the study’s lead investigator Dr Chris Chiu, from Imperial College London.
The study was first announced last year in a media briefing at the Wellcome Trust in London. At that event, Chiu said that the ICL team had been safely running human challenge studies with other respiratory viruses for over 10 years, according to a Lancet Respiratory Medicine report on the proceedings.
There have been questions about the necessity of the challenge studies, particularly as the number of vaccines becoming available for COVID-19 is rising quickly, amid concerns that if a volunteer in the trial was to become severely ill, get ‘long covid’ or die, it could fuel conspiracy theories or anti-vaccine sentiment.
Debate has also surrounded enrolment of young subjects, who are most likely to have a mild infection, as the findings may not be relevant to protecting more vulnerable people against serious disease.
Charles Weijer, a bioethicist and expert on the ethics of randomised controlled trials, wrote in the British Medical Journal last November that with vaccines expected to arrive soon it would be unethical to run challenge studies as it is unclear why they are required for second-generation shots.
He also points out that while challenge studies have been used for decades, with no reported patient deaths, in the past they have focused on well-characterised diseases while SARS-CoV-2 is much less well understood.
Clinical research company hVIVO, which is helping to run the trial, says that some of the research objectives in the challenge study cannot be achieved with traditional field trials which rely on exposure to virus circulating in the general population.
“COVID-19 human challenge studies have the potential to play an important role in providing data and information that will help continue to develop vaccines to control the pandemic,” said the company’s chief scientific officer Dr Andrew Catchpole.
The UK government’s massive spending on the coronavirus pandemic response has been “rife with conflicts of interest”, says the opposition Labour Party, and an independent watchdog should be set up to oversee procurement contracts.
That was the message delivered by shadow Cabinet Office minister Rachel Reeves in a speech yesterday which claimed almost £2 billion of the £23.1 billion public sector spend on the pandemic went on contracts that were awarded to “Conservative friends and donors”.
In the last year, £2bn of taxpayers’ money has been spent on products and services provided by firms with a financial or personal connection to the Conservatives.
Labour will launch the biggest wave of insourcing in a generation – bringing services back under public control.
The allegations of cronyism have been rejected by the government, which said its award of contract for items like personal protective equipment (PPE), hand sanitisers and ventilators has been based on “robust rules in place to make sure there are no conflicts of interest”.
The government has accused Labour of “playing politics” as it concentrates on delivering on its commitments to fight COVID-19.
Reeves’ speech drew heavily on the findings of a National Audit Office (NAO) report published last November, which concluded that companies recommended by MPs, peers and ministerial offices were given priority amid the initial scramble for PPE supplies.
The watchdog concluded that 58% of contract – worth £10.5 billion – were awarded without any competitive tender process, and also that not enough was done to address potential conflicts of interest.
It also concluded that there was no evidence that ministers had been involved in either awarding or managing PPE contracts.
“When a US jeweller received £200 million as a middleman for a PPE contract – did no-one in government sit up and ask what on Earth was going on?” asked Reeves, referring to a case involving Florida-based designer Michael Saiger who brokered PPE contracts for the NHS.
“This is an unforgiveable waste,” she said.
She also pointed to the case of former Conservative minister Owen Paterson, who lobbied for Randox – a company that provided 750,000 defective tests which had to be recalled – while being paid £100,000 on top of his MP’s salary.
Reeves said she had written to the top 10 companies with Conservative party links that have won contracts to ask how much profit they had made from the pandemic
Labour wants the government to introduce tendering rules with improved transparency to scrutinise contracts to prevent cronyism, and has pledged to expand the Freedom of Information act to cover all new public service contracts delivered by private companies.
It has also called for the creation of an “independent anti-corruption and anti-cronyism commissioner to replace the current toothless and far from independent anti-corruption tsar” – an unpaid position appointed by the Prime Minister and currently held by John Penrose MP.
Labour has challenged ministers to publish “all outstanding contracts” by the end of this month in order for them to be open to public scrutiny.
The UK has agreed a 50 million-dose order for a COVID-19 vaccine with German biotech CureVac to support development of a shot that specifically targets emerging variants of the virus.
The agreement will see the UK work with CureVac develop “new varieties of vaccines based on messenger RNA technology to be developed quickly against new strains of COVID-19,” said the government.
New UK, South African and Brazilian strains of COVID-19 have been ringing alarm bells because they are more likely to be transmitted than the original, although there’s no solid data yet to suggest that they are more likely to cause severe disease.
The current Pfizer/BioNTech and Oxford/AstraZeneca vaccines seem to protect against the variants currently dominant in the UK, according to the government, but as a precaution the aim of the new partnership is to use UK expertise on virus DNA sequencing to allow new mRNA shots to be developed quickly.
An expert advisory group will be set up to identify the variants that could pose a threat to the UK population, with the doses expected to be ready later this year if they are needed.
On Friday, AZ said its vaccine offered people good protection against the UK variant, but a new study has shown it is less effective against the South African variant, according to the Financial Times.
AZ has said it is confident however that it could quickly update its vaccine and have a new version ready for the autumn.
Targeted testing for the South African strain – which includes a mutation known as E484K that seems to help it evade immunity from vaccines and earlier coronavirus infection – is now underway in various parts of the UK.
Prof Jonathan Van-Tam, the deputy chief medical officer for England, said it is “likely that our vaccines will have to adapt to continue to offer the best possible protection.”
He added: “Being able to create these new vaccines at speed will allow our scientists to keep ahead of the virus as they do every year with the influenza vaccine.”
CureVac’s first-generation COVID-19 vaccine CVnCoV is in phase 2b/3 testing with results expected in the coming weeks, and last week the biotech teamed up with UK drugmaker GlaxoSmithKline in a €150 million alliance to develop a candidate that would target multiple new variants in a single jab.
It also has a partnership in place with Germany’s Bayer to produce several hundred million doses of the shot, and is also relying on contract manufacturers Wacker and Fareva to produce supplies.
The UK deal also includes a provision for setting manufacturing of the vaccines within the UK, which the government said would strengthen domestic capabilities.
Production capacity was at the heart of a row that erupted between the EU and AZ over supplies of the company’s vaccine a couple of weeks ago, with the European Commission slamming the company for not delivering as many shots as contracted.
The 50 million dose order comes on top of 407 million doses already booked in by the UK from AZ, Pfizer/BioNTech, Valneva, GSK, Novavax, Janssen and Moderna. So far only the Pfizer/BioNTech and AZ vaccines are being given to the public.
Shortly after the announcement of the CureVac partnership, Health Secretary Matt Hancock announced a new UK target of vaccinating 15 million people by 15 February, and all over-50s in the UK by May.
New figures have confirmed that the UK’s biotech sector received a massive boost from the COVID-19 pandemic last year, with investment levels soaring to £2.8 billion ($3.8 billion) from a level of £1.3 billion in 2019.
That makes 2020 a new record year for the sector, exceeding the previous investment high of £2.2 billion in 2018, according to the latest BioIndustry Association (BIA) and Clarivate report, and represents a 1,000% increase on 2012.
The data show that 2020 was a “watershed year” for UK biotech, according to BIA chief executive Steve Bates, who said the uplift was seen across the life sciences category, not just organisations that were directly involved in the COVID-19 response.
“The successful year means many UK biotechs are well capitalised as we head into 2021 with continuing high levels of global economic uncertainty,” says the report.
Breaking down the numbers, almost £1.4 billion of the total came from venture capital – almost twice the level last year – with seed and early-stage VC financing coming back strongly in the latter half of the year after a dip in the second quarter as the pandemic took hold. Five companies raised more than £50 million in VC funding last year.
The largest venture deals included Oxford Nanopore’s two-part £163 million fundraising, which could be followed by an initial public offering (IPO) in the coming months, according to the biotech. It came to the fore in 2020 thanks to its hand-held MinION DNA sequencing device that is being used to track the emergence of COVID-19 variants.
Cancer therapy company Immunocore secured the largest individual VC deal of just over £100 million, and has just set out its stall for a $200 million IPO to support its phase 3 T cell therapy tebentafusp, scheduled for filing in uveal melanoma late this year.
There were three IPOs in 2020 which collectively brought in £244 million, according to the report. That’s a lower number on previous years but reflects “a growing trend for companies to remain private for longer, made possible by the increased availability of venture capital”.
Two of those were £100 million-plus Nasdaq launches – cell therapy specialist Freeline Therapeutics and magic mushroom drug developer Compass Pathways – which reinforces the appetite of US investors for UK biotechs.
The third was diagnostics company Verici Dx’s listing on the AIM which raised £14.5 million to support its next-generation sequencing platform to guide kidney transplants.
Follow-on financing by public companies was also buoyant, bringing in almost £1.2 billion, with London’s AIM seeing the highest levels of investment (£515 million) since 2015.
The biggest rounds came from Adaptimmune Therapeutics, which raised £168 million on the Nasdaq, and Verona Pharma which netted £160 million from its dual Nasdaq and AIM listings.
Fewer companies founded
Not all the data points are positive. BIA points to a sharp dip in the number of new biotechs formed in 2020 – just eight compared to 30 in 2019 and 47 in 2018 – suggesting that the pandemic had a dramatic impact on entrepreneurship.
There has been a decline in new companies being formed over the last five years, however, and the BIA says it will continue to monitor this trend. On the plus side, the number of companies receiving their first investment has been “relatively sustained” in recent years, but dropped from 70 to 49 last year.
“In 2020, BIA member companies demonstrated their value and impact; they developed a vaccine for COVID-19 currently being distributed to millions of people around the world, while others have provided ground-breaking diagnostics or have promising life-saving therapies in the pipeline,” said Bates.
“In a difficult year, UK life science has been a beacon of economic and health success.”
REGEN-COV neutralizes the SARS-CoV-2 B.1.1.7 variants, identified in the UK & SA. The data was included in a bioRxiv paper and submitted for peer-reviewed publication on the changing resistance of SARS-CoV-2 variants to Ab neutralization
REGEN-COV retained its neutralizing capability against the B.1.1.7 variant. The Ab cocktails also retained its potency against the B.1.351 variant; imdevimab retained its potency against it while casirivimab potency was reduced
Additionally, the Brazil variant (1.1.248) have the same receptor binding domain mutations as the B.1.351, thus REGEN-COV is expected to remain similarly potent. Regeneron is conducting additional research against this strain
Click here to read full press release/ article | Ref: PRNewswire | Image: Barron’s
NHS patients in England with newly-diagnosed multiple myeloma can be treated with Celgene’s Revlimid as maintenance therapy after a stem cell transplant, after new guidance from NICE.
The cost-effectiveness agency has backed interim funding for Revlimid (lenalidomide) in this setting via the Cancer Drugs Fund (CDF), which provides temporary reimbursement for medicines until further data can be gathered that can help NICE with its cost-effectiveness calculations.
Last September, NICE turned down NHS funding of Revlimid for this use, saying that the cost-effectiveness estimates were “uncertain” because of limitations in its modelling that meant it “might not reflect what happens in the NHS in England”.
Now, Revlimid is the first treatment to be made available for newly-diagnosed, post-transplant patients on the NHS, providing an alternative to the standard ‘watch-and-wait’ approach. That means that patients can receive active treatment to keep their cancer in remission for the first time.
Across the UK, around 1,500 newly diagnosed myeloma patients undergo a transplant each year, and most of them eventually relapse. The first remission is a critical period for patients, as it can be an indicator of the overall survival from the disease.
Celgene – a subsidiary of Bristol-Myers Squibb – said there are around 1,150 people in England who could now be eligible for treatment with Revlimid. The drug has been approved for this use by the Scottish Medicines Consortium (SMC) since last September.
In two phase 3 trials – CALGB 100104 and IFM 2005-02 – Revlimid given as a daily oral dose after a stem cell transplant was able to extend the time patients survived without disease progression compared to placebo.
There was also a trend towards improved overall survival, although the trials weren’t statistically powerful enough for that to be a reliable result.
“We are delighted with this outcome,” commented Laura Kerby, chief executive of Myeloma UK.
“Patients who receive lenalidomide maintenance after high-dose therapy and stem cell transplant have a significant increase in overall survival, so the decision to make this available through the NHS is fantastic news,” she added.
The latest decision comes two years after Revlimid was cleared by NICE for NHS use as a treatment for newly-diagnosed, transplant-ineligible myeloma patients. It can also be used as a second- or third-line option in combination with other drugs for relapsed myeloma.
Revlimid is BMS’ biggest product, bringing in almost $9 billion in worldwide revenues in the first nine months of 2020. In addition to myeloma it is also used for a wide range of blood cancers, including myelodysplastic syndromes (MDS) and various types of lymphoma.
More than 100,000 people have died within 28 days of a positive COVID-19 test in the UK, the first European country to meet that grim milestone.
The UK is only the fifth country after the US, Brazil, India and Mexico to pass that threshold, after a surge in cases following the holiday period around the turn of the year.
There were another 1,631 deaths recorded yesterday, and while there are signs that the new lockdown is starting to have an impact on new cases, the number of hospitalised COVID-19 patients – currently around 35,000 – has so far only stabilised and hasn’t started to fall.
Prime Minister Boris Johnson said in a televised address that its “hard to compute the sorrow contained in that statistic. I offer my deepest condolences to everyone who has lost a loved one.”
The PM also promised to learn the lessons of the pandemic so that when vaccines “have finally freed us from this virus, and put us on a path to recovery, we will make sure we learn the lessons and reflect and prepare.” He also said 6.8 million people in the UK have now received one of the available COVID-19 vaccines.
Opposition leader Sir Keir Starmer said the news represented a “national tragedy and a terrible reminder of all that we have lost as a country.”
The British Medical Association (BMA) is marking the landmark with an installation on its London headquarters this evening (pictured), commenting that “we must not and will not forget this day.”
BMA chair Dr Chaand Nagpaul said: “we must learn the lessons of this tragedy. We must understand why so many excess lives have been lost in our nation so that we can prevent this scale of death from coming to pass in any future pandemic.”
He added that “many health and care workers…lost their lives in the course of doing their jobs, caring for others in the most challenging of circumstances as we have seen NHS services tested to their limits.”
England’s chief medical officer Professor Chris Witty wouldn’t be drawn on the range of fatalities expected before the pandemic could be brought under control, but did say that “unfortunately we are going to see quite a lot more deaths over the next few weeks before the effects of the vaccines begin to be felt.”
During the early stages of the pandemic, the UK government had said it hoped to contain the number of deaths to around 20,000.
On the positive side, NHS England chief executive Sir Simon Stevens pointed out that the death rate for patients hospitalised with COVID-19 has improved roughly from a third to a fifth – thanks to improvements in care and new drug therapies – and additional drugs are likely to emerge that could improve that even further.
“I think we can see a world in which coronavirus may be more treatable,” he told the briefing, but for now, the emphasis has to be the combination of measures to reduce infections and vaccinations.
Johnson also responded to questions at the briefing about the EU’s decision to require vaccine manufacturers to register exports from the bloc in the midst of a dispute with AstraZeneca over small-than-expected supplies of its shot.
The EU has said it has no plans to impose an export ban, and the PM said he had “total confidence” that the supply of vaccine doses into the UK won’t be disrupted.
“The creation of these vaccines has been a wonderful example of multinational cooperation and I think that one of the lessons the world has had to learn is the need to…fight the pandemic together,” he said.
“I don’t want to see restrictions on the supply of [PPE, drugs or vaccines] across borders,” he continued, adding: “I’m sure that will be widely supported across the EU as well.”
It’s evident that relying on Google for a health diagnosis isn’t a good idea, but it seems a majority of UK people still do – and even buy medicines based on what they find.
A new UK survey finds that overall 59% of 1,000 respondents Google their health symptom before consulting a doctor, with 16% reporting that as a consequence a condition has gone undiagnosed by a doctor for some time.
Meanwhile, almost half (45%) of the 16-24 age bracket have bought medicines after a diagnosis from “Dr Google”, along with around a third each of 25-34 and 35-44 year olds, according to the poll by vision care company Lenstore.
That’s quite a worrying finding, particularly in light of research published last year that found the accuracy of online symptom-checking websites and apps varied considerably, and only reached the correct diagnosis 36% of the time.
Sometimes, self-diagnosing on the internet can cause more harm than good, according to Dr Chun Tang, a GP at UK private healthcare company Pall Mall Medical.
Some sites are simply unreliable, providing inaccurate or incomplete information that can lead people to “wrong conclusion and wrong directions,” he says.
Meanwhile, websites serving healthcare professionals use terminology that may “cause…confusion and fear in the non-medically trained,” according to Dr Tang, who recommends people only use recognised and reliable websites such as NHS, Cancer Research UK, NICE and the BNF.
That’s backed up by the survey, which found that around 30% of people admitted that Googling a health symptom actually made them feel more anxious, while almost one in four said it had a negative effect on their mental health.
Londoners were the most likely to self-diagnose using a Google search, with 23% always checking symptoms online, whilst at the other end of the spectrum the same proportion of Liverpudlians said they never Googled symptoms.
The reasons for turning to the internet for self-diagnosis are varied, but the survey finds that the most common reason – cited by 40% of people – was simply to be aware of potential problems before seeing a doctor. Another 30% did so to avoid putting pressure on the NHS.
However, an alarming 37% of Brits use their own online self-diagnosis to determine whether they need to see a medical expert or not, which runs counter to medical advice.
Dr Tang advises people who are worried enough to carry out online research make sure they consult a medical professional as well, “whether a pharmacist for minor symptoms or a nurse or doctor for persisting and serious symptoms.”
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.”
Health Canada expedite the review of AstraZeneca’s COVID-19 vaccine after the vaccine received the UK’s MHRA approval for emergency use
Following an agreement to supply 20M of doses for the Government of Canada, AstraZeneca seek out Health Canada’s clearance in Oct’2020, leading to data submission done on a rolling basis for accelerating the review process
Health Canada is looking to give Canadians access to COVID-19 vaccines asap without compromising its safety, efficacy, and quality standards
Click here to read full press release/ article | Ref: Newswire Canada | Image: Money Control
The post-Brexit trade deal agreed on Christmas Eve has been overwhelmingly backed by the UK parliament, providing some degree of stability – but also plenty of disruption – for the coming months and years.
The UK is leaving the EU’s single market and customs union, but the deal means tariffs on goods won’t be imposed when the transition period ends at 23:00 GMT tonight.
In a marked divergence from the wrangling and acrimony that surrounded votes on the withdrawal agreement a couple of years ago, the EU (Future Relationship) Bill was backed by 521 votes to 73 last night.
Prime Minister Boris Johnson said the deal – which comes four-and-a-half years after the UK voted to leave the EU in a referendum – will “fulfil the sovereign wish of the British people to live under their own laws, made by their own elected Parliament.”
The new regime has been welcomed by pharma organisations on both sides of the English channel, although there are still a lot of unanswered questions about what it will mean in practice for the regulation and trade in medicinal products.
With the text of the 1,200-page document now available, it has been confirmed that there will be mutual recognition between the two parties, which means that inspections and certification of good manufacturing practice (GMP) facilities by the UK regulatory authority will be recognised by the EU, and vice versa.
What appears to be missing at first glance from the document however is mutual recognition of safety and quality testing, which might mean some duplicate tests are needed.
The appendix on medicines does however refer to “the exchange and acceptance of official GMP documents between the parties,” and includes an article covering “regulatory cooperation” on changes to technical regulations or inspection procedures.
To make that easier, a Working Group on Medicinal Products will be set up to monitor the impact of the deal on medicines in the UK and EU, for example to respond if there is a threat to medicines supply or public health, and organise future cooperation in areas like scientific or technical guidelines.
BioIndustry Association (BIA) chief executive Steve Bates said after the deal was agreed that it provides “much desired certainty for our sector” after four years of negotiation, although the trade body is still working through the details of the text.
BIA said that traders can self-certify the origin of goods sold and enjoy “full cumulation”, making it easier to comply with requirements and obtain zero-tariff access, adding that there will be specific facilitation arrangements for pharmaceuticals and chemicals.
We are pleased no deal has been avoided between the UK and EU and look forward to examining the trade deal announced today in detail. We will focus on what continued co-operation in Research and Innovation means for our sector and provide full analysis as soon as possible.
The appendix on medicinal products is far from detailed – in fact, Labour Leader Keir Starmer has described the entire document as “thin”, whilst also instructing his party to back it in the parliamentary vote to prevent no deal.
There are other encouraging points for the life sciences, according to the BIA, including the UK continuing to have access to the Horizon Europe Research and Innovation programme as a paying third country.
“We look forward to working with the UK government on this positive agenda, mindful of the fact that our sector seeks, and benefits from, innovative global standards and regulatory collaboration and co-operation,” said Bates.
A joint statement released by the Association of the British Pharmaceutical Industry (ABPI) and European Federation of Pharmaceutical Industries and Associations (EFPIA) – representing the UK and EUK pharma industries – also said it would take time to examine the details of the agreement.
“We have always said that a deal is in the best interest of patients in the UK and the EU,” they said, adding: “regardless…the end of the transition means there will be a significant change in how border and customs arrangements work come Jan 1st and companies have been working on contingency plans to mitigate any disruption.”
Flurry of MHRA guidance
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has already started publishing guidance on how things will change from Friday.
This week, for example, it confirmed it will set a 150-day assessment timeline for new medicines, whilst also providing details of its equivalent to the EMA’s “rolling review” designed process to speed up access to novel medicines.
It has also provided updates on the processes needed to submit changes to marketing dossiers for medicinal products, as well as for amendments to clinical trials.
The MHRA has provided authorization for an emergency supply of AZD1222, for the active immunization of individuals aged ≥18yrs. The approval recommends 2 doses administered with an interval of between 4 & 12wks
The authorization is based on independent advice from its CHM following a rolling review of trial data that included an interim analysis of the P-III program led by the University of Oxford
AstraZeneca aims to supply millions of doses in Q1 as part of an agreement with the government to supply ~100M doses in total. The company will continue the regulatory interactions across the globe for the next approvals
Click here to read full press release/ article | Ref: AstraZeneca | Image: Express Pharma
In the last few years, biopharma companies focusing on psychedelic medicines have been springing up like mushrooms – magic or otherwise – and venture capital money is starting to follow.
Today sees the launch of the first investment fund in the UK devoted to psychedelic healthcare, with plans to invest in “revolutionary mind-altering medicines to treat illnesses including depression, addiction, anxiety and inflammation.”
The fund has been set up by London-based VC Neo Kuma Ventures, a new group formed by Sean McLintock, Clara Burtenshaw and Nick David in 2019. The co-founders say it has already attracted “millions of pounds” in investment, and will continue to draw funds through the first half of next year.
Last year Neo Kuma’s founders backed ATAI Life Sciences AG, a part owner of Compass Pathways, which is a UK-based company trying to develop medicines based on a synthetic version of psilocybin, the main psychoactive constituent in magic mushrooms.
In September, Compass became the first psychedelic medicine company to float on the Nasdaq, raising $127 million, and is now trading at a market cap of $1.98 billion.
Shortly after, US biotech Mind Medicine – already trading publicly on Canada’s Neo exchange – applied for a Nasdaq up-listing as it advances a suite of psychedelic medicines based on MDMA, LSD and ibogaine derivative 18-MC. It is going after disorders like anxiety, opioid addiction and adult attention-deficit hyperactivity disorder.
Around the same time, Toronto-based Field Trip Psychedelics went public on Canada’s CSE after it completed a reverse takeover of oil and gas company Newton Energy Corp, which followed an CAD 12 million private placement deal.
As well as offering ketamine-assisted treatment clinics, the company is also working on FT104, a novel synthetic hallucinogen for mental health disorders. Meanwhile, other players in the sector include Cybin – which has just acquired rival Adelia Therapeutics for just under $16 million – as well as Numinus Wellness and Verrian Ontario.
Data Bridge Market Research published report earlier this year suggesting that the psychedelic drugs market is projected to grow at around 16% per year over the next eight years to reach $6.85 billion in 2027, spearheaded by new therapies like Johnson & Johnson’s Spravato (esketamine) for treatment-resistant depression.
NeoKuma draws parallels with the medicinal cannabis market, citing research which suggests that in the US it has surged from around $2 billion in 2014 to an estimated $35 billion this year.
“As the medical benefits of psychedelics become more well-known and regulators steadily increase their embrace of these types of drugs, the industry is set for a boom,” says McLintock.
“While much of the conversation on psychedelics is taking place in the US, Europe is the true hub of the burgeoning psychedelic healthcare sector. We look forward to investing in the most exciting, high quality and scientifically-sound European players in the industry to facilitate their ground-breaking research.”
A new network of patient recruitment centres is set to shape the future of commercial clinical trials in the UK by offering new approaches to conduct late-phase, large-scale research projects. In a recent pharmaphorum webinar leaders from the NIHR’s five National Patient Recruitment Centres (NPRCs) outlined how they could bring cutting-edge therapies to greater numbers of UK patients.
The NPRCs are one of the Sector Deal 2 commitments from the government’s Life Sciences Industrial Strategy and are the first centres to be funded by the NIHR that are 100% dedicated to delivering commercial research.
The regional centres have been distributed across the country to provide opportunities for patients in areas of England who may not previously have been able to take part in the latest clinical studies.
They have also been designed to improve the speed and consistency through which commercial research is delivered in the NHS.
The goal is to attract life sciences R&D projects by making it easier and quicker to deliver commercial research, in turn improving the UK’s competitiveness in the global market.
pharmaphorum’s webinar, held in association with the NIHR, featured contributions from the directors of the NPRCs, the medical leaders who are leading the programme.
Dr William van’t Hoff, chief executive officer of the NIHR’s Clinical Research Network, began proceedings by highlighting some of the successes that have already been achieved.
“Our aim is to establish a relationship and build trust with our local community, to develop understanding, reduce the fear of research and increase engagement in that way” Yan Yiannakou
These include the delivery of COVID-19 vaccine trials and one of the UK’s first fully virtual interventional commercial clinical trials.
These studies have been made possible by the franchise-like operating model underpinning the network, which allows them to work in a harmonised manner, he said.
Franchise like operating model and collaborative working
The NIHR has provided £1.3 million in funding to each centre over three years to support their activities. Their collaborative model means the NPRCs will operate in an identical manner, offering a number of advantages to study sponsors.
As pointed out by Dr van’t Hoff, there will be a consistency of approach that could boost the UK’s competitiveness in a global market for late-stage clinical trial research.
The centres will benefit from shared standard operating procedures and will collaborate with each other as they host trials, the webinar heard.
Van’t Hoff said: “Each centre will provide a dedicated space and facilities for commercial clinical research, guaranteed access to NHS services to support research delivery, access to NHS clinicians to work as investigators, and a team of dedicated and highly-skilled research delivery staff to conduct the trials.”
Helen Quinn, director of the Joint Office for Clinical Research (University of Exeter and Royal Devon and Exeter NHS Foundation Trust), added that the centre in Exeter has been able to work with others across the country, costing and contracting for commercial research studies in a swift way.
“We can set trials up quickly for our patients, which is the most important consideration for us,” she said.
Speed, ease and volume
Using a franchise like operating model with standard operating procedures can speed up patient recruitment because of improved efficiency.
This was demonstrated by the swift set-up of a phase 3 COVID-19 vaccine study, which took just two weeks to set up and launch with over 1,900 patients recruited across the three NPRCs involved when it closed.
Dr Gavin Galasko is a consultant interventional cardiologist and director of research, development and innovation at Blackpool Teaching Hospitals NHS Foundation Trust, home to the Blackpool Patient Recruitment Centre.
He said: “We were the first in the world to recruit to this phase 3 study. We want to be there for pharma to show that the UK is a place to offer these latest state of the art late phase commercial studies that we can get to the patients quickly, efficiently, and in high numbers.”
Dinesh Saralaya, director of the NIHR Patient Recruitment Centre in Bradford, added that another factor that can allow trials to be quickly set up is the unified approach to costing across all the centres.
Each centre is also supported by research nurses, pharmacists and other research support staff who are dedicated resources available to the commercial trials run at the sites.
NPRCs are entirely focused on commercial research, further reducing the time to set up trials, the webinar heard.
The ‘recruitment engine’
Each centre is also driven by a ‘recruitment engine’ that uses several proactive strategies to reach out into the local community to empower patients to take part in, and to volunteer for, research.
As well as being linked to large hospitals, each centre will also collaborate with other healthcare organisations across primary, community and social care.
They can interact with primary care networks and integrated care partnerships (ICPs) for example to recruit from the local population.
This can help them find trial participants who are not hospitalised but who live with common chronic conditions and are not presenting in secondary care.
At the same time they benefit from links to large hospitals and their experience and expertise.
The centres can easily link with a wide network of GP surgeries and other local healthcare organisations, using linked NHS data sets to help identify patients to take part in research.
Yan Yiannakou, clinical director of the Newcastle NPRC, said that rather than going to a single GP surgery with 5,000 patients on its books, the centre works with federations of surgeries with 250,000 patients.
He said: “By working with a single point of contact in that federation and by working with the core federations around Newcastle, we can cover a population of 650,000 with a single search.”
Other innovative strategies employed at the NPRCs include virtual consultations, something that many other organisations have tried to implement but not succeeded.
Recruitment is further aided by digital consent, data capture and online eligibility checks, with the model already leading to a ground-breaking virtual trial, possibly the first in Europe.
Yiannakou said that the model has already been used to conduct a virtual trial in irritable bowel syndrome with diarrhoea (Relieve IBS-D).
The trial is currently recruiting 10 patients a week, which is twice as many as was being recruited by 28 sites before the study went virtual.
It’s just one of several innovative approaches to patient recruitment that are being explored at the NPRCs.
Yiannakou said: “Our aim is to establish a relationship with our local community, to build trust with the local community, to develop understanding, reduce the fear of research and just increase engagement in that way.”
Other approaches to recruitment will be explored, aiming to put patients and their needs at the centre of the process.
Melanie Davies, professor of diabetes medicine at the University of Leicester and director of the NPRC in Leicester, said: “This is all about bringing research closer to patients across the patch, across the NHS, but to provide it in a way that is attractive and accessible to patients.”
The cutting-edge of global life sciences research
A theme running all the way through the webinar was the sense of collaboration that each centre will bring to research projects.
The NPRC clinical directors all highlighted the centres’ ability to work together and with the wider healthcare ecosystem, such as primary, community and social care to enable access to a wider cohort of patients.
Yiannakou said: “The key is a willingness to develop and a willingness to change, and we have both the drivers and the substrates for that. The drivers are the fact that we are a novel concept centre, that we specialise in a particular area.
“We want to be the best in that area, we are focused in that area, and we have a remit and an intent to refine process and improve recruitment.”
According to Saralaya, the centres are sending a “strong message” to industry about the UK’s intent to be at the cutting edge of global life sciences research.
He said that the work already under way at the five centres shows they can deliver trials at speed, with quality and with very unified costing.
He concluded: “What better advocate can it be for a sponsor wanting to come to the UK?”
About the interviewees
Helen Quinn is the director of the Joint Office for Clinical Research, a collaboration between the University of Exeter and the Royal Devon and Exeter NHS Foundation Trust.
Dinesh Saralaya is a consultant respiratory physician and honorary senior lecturer at Bradford Teaching Hospitals NHS Foundation Trust. He has served as Director of the National NIHR Patient Recruitment Centre, Bradford since June 2020.
Yan Yiannakou is a consultant gastroenterologist and Clinical Director of the NIHR Patient Recruitment Centre (Newcastle).
Melanie J Davies is Professor of Diabetes Medicine at the University of Leicester and an honorary consultant diabetologist at the University Hospitals of Leicester NHS Trust.
Dr Gavin Galasko is a consultant interventional cardiologist and the director of research, development and innovation at Blackpool Teaching Hospitals NHS Foundation Trust.
Pfizer & BioNTech reports that the MHRA in the UK has granted a temporary authorization for the EU for BNT162b2 against COVID-19. The distribution of vaccine will be prioritized according to the populations identified in guidance from the JCVI
The MHRA’s decision is based on a rolling submission, including data from the P-III study, demonstrating 95% efficacy in participants without & with/ out prior SARS-CoV-2 infection, in each case measured from 7 days after the second dose
This marks the first EUA following a WW P-III trial of a vaccine to combat the pandemic. The companies are anticipating further regulatory decisions across the globe in Dec’2020
Click here to read full press release/ article | Ref: Pfizer | Image: The Jakarta Post
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.”
We’re clearly laying out the prioritisation on groups for early vaccination, starting with prevention of mortality.
If you’re asked to get a vaccine, I urge you to take it to protect yourself, your loved ones & your community.https://t.co/AfXnoOPupx
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.
Moderna amended its current supply agreement with the UK govt. for an additional 2M doses of mRNA-1273 against COVID-19. The UK govt. has now secured 7M doses of mRNA-1273
The agreement reflects Moderna’s commitment to making its vaccine available in multiple countries. Moderna ramp up its global manufacturing to be able to deliver ~500M doses/yr & possibly ~1B doses/yr, beginning in 2021
The company is working with its strategic manufacturing partners, Lonza of Switzerland and ROVI of Spain, for manufacturing and fill-finish outside the US. Additionally, Moderna reported that the vaccine showed 94.5% efficacy
Click here to read full press release/ article | Ref: Businesswire | Image: Stat
Researchers at the Wellcome Sanger Institute in the UK are working on software to monitor the genome of SARS-CoV-2 coronavirus in the hope of spotting changes that could affect the fight against COVID-19.
Backed by £12.2 million in UK government funding, Wellcome Sanger and the COVID-19 Genomics UK (COG-UK) Consortium intend to develop a real-time nationwide surveillance system that could identify mutations in the virus.
Wellcome Sanger will use the funding to develop the software to capture genomic information digitally, and pair it up with data from the UK public health agencies and the NHS and Trace programme.
The hope is that monitoring will allow health authorities to quickly identify mutations that might allow the virus to sidestep diagnostics, vaccines and drug therapies, for example, and guide the development of counter measures.
It will also allow linking of viral sequencing patterns with host genomics, immunology, clinical outcomes and risk factors, according to the consortium.
With the second wave of COVID-19 infections now gathering pace, and vaccines on the brink of being made available, genomic data will be critical to determine whether the virus evolves to escape them.
Since the first whole-genome sequence of SARS-CoV-2 was shared online on 11 January, scientists around the world have been sequencing the virus to spot genomic changes that will allow outbreaks to be tracked and controlled.
The effort could also help local authorities respond to outbreaks more quickly, according to researchers, and could become particularly important in allowing international travel to return to some degree of normality.
Specific lineages of virus can be identified from around the world, and that makes it possible to detect lines of transmission by comparing the genetic sequences of virus samples.
Sequencing also allows scientists to monitor key viral characteristics like transmission and disease severity, according to COG-UK director Prof Sharon Peacock from the University of Cambridge.
Since March, COG-UK has published more than 100,000 SARS-CoV-2 genomes, making up over 45% of the global total, an effort that it says has not been achieved previously for any pathogen, anywhere in the world.
The genomic data already generated by the COG-UK network – which includes Wellcome Sanger – has already “provided important scientific insights into the spread and evolution of the virus, at local, regional, national and international scales,” says the consortium.
Earlier this year, Swiss medical data specialist Sophia Genetics launched data mining tools to help researchers examine how the genome of SARS-CoV-2 changes over time, and combine that data with patients’ clinical and genetic information.
Tech company NVIDIA has also made its Parabricks genome-sequencing software available at no charge to researchers sequencing the coronavirus and the genomes of those suffering from COVID-19.
US data analysis company Palantir Technologies could be drafted in to manage the UK government’s troubled COVID-19 Test and Trace programme, according to press reports.
Palantir has been linked with the project for several weeks and the Financial Times is the latest to suggest that the company could get involved with the troubled project.
Palantir was founded in 2003 by a team including paypal co-founder Peter Thiel and the company’s billionaire CEO Alex Karp.
Taking its name from the “seeing stones” in The Lord of the Rings, Palantir is known for counter-terrorism work and fraud investigation with agencies of the US federal government.
The company is reportedly being hired to fix the “technical error” that caused around 16,000 positive test results to go unreported.
There was widespread anger and derision when the issue turned out that the results had been stored on a Microsoft Excel document that had run out of storage space.
According to the FT, officials are in discussions with Palantir over licensing its software to the test and trace programme, with a long-term goal of finding deeper insights into how the virus is spreading.
The aim is to allow the government to respond more quickly to outbreaks, an issue that the government has been struggling with over the last few weeks.
Before the government called a month-long national lockdown, it struggled to keep on top of rising numbers of cases in the north of England with its targeted local approach.
Palantir is already working on another pandemic-related project, after it was drafted in to support work on the COVID-19 data store.
The company’s contract was extended for four months in July although a procurement process is open for a supplier to continue the work on the technology platform.
Since entering the pharmaceutical industry as a graduate trainee in the late 1990s, Asad Mohsin Ali’s career has taken him across the business from Novartis, MSD and Tesaro, before joining global biopharma Ipsen. He tells us how the company has responded to COVID and how he’d like to see the UK industry grow in the future.
As managing director, Ipsen UK & Ireland, Ali heads up one of Ipsen’s three global hubs, as well as chairing a UK & Ireland Site Steering Committee overseeing 900 people.
“My primary goal on joining Ipsen UK&I was to incorporate and elevate the talents of our Specialty Care organisation, but also the 900 colleagues in the UK and Ireland, to ensure we bring maximum value to patients and healthcare systems,” Ali tells pharmaphorum.
Ipsen UK&I’s culture is based on the principle, “One Ipsen” he says.
“Being “One Ipsen” means acting together on what matters most to our patients, customers and our employees.”
As COVID-19 disrupted the world, the pharmaceutical industry was urged to respond quickly – primarily by ensuring continuity of medicines for patients.
“The level of response required by industry, the impact to business and the duration of the crisis have surpassed the extent of a lot of business contingency plans,” Ali says. “Government responses and restrictions put in place to contain COVID-19 have changed over time. So, businesses like Ipsen UK&I have had to stay agile, adapt and re-group to deal with the changes, whilst ensuring a strong crisis management team is in place, and the right subject matter experts advising on our approach.”
“Changes in treatment patterns and patient journeys – companies need to understand how some of the changes will be long-term; not just about how patients can be safely re-introduced to the healthcare system, remote consultation and treatment will be more widely used.”
Ipsen UK&I recognised early on there would be no “one size fits all” way of working, says Ali. “For our manufacturing site in Wrexham, which is classified as critical workers, not much has changed except for the need to have less people on site with more PPE. But for our field-based team, the transition to fully home-based working with limited external interactions it has been a complete change that required a lot more adaptation.”
The company also focused on providing wellbeing resources to employees such as virtual yoga, an online wellbeing hub, a network of Mental Health and Wellbeing Ambassadors and an Employee Assistance Programme.
“We implemented a COVID-19 volunteering policy, which enabled our employees to go back into the NHS or carry out volunteer work for local community groups for one day per week fully paid,” Ali says.
With COVID-19 anticipated to change how pharmaceutical companies engage with healthcare professionals, Ipsen UK&I is preparing for a more remote and virtual healthcare system. Prior to the pandemic, 95% of all the company’s UK customer facing teams’ interactions took place in person at NHS hospitals.
“Overnight this became unfeasible so we invested a significant amount of time on how we could optimise remote interactions. This took many forms, from instant messaging to full video-based interactions with interactive e-materials. We have learned that the way our customers interact with their patients has shifted significantly and maybe permanently, so this in turn will drive how they want to interact with us.”
Indeed, more virtual patient engagement across the board means that services and products allowing remote engagement will thrive.
“Changes in treatment patterns and patient journeys – companies need to understand how some of the changes will be long-term; not just about how patients can be safely re-introduced to the healthcare system, remote consultation and treatment will be more widely used.”
The pharmaceutical industry can also expect to face challenges with innovation and funding, Ali says.
“While the pressure on healthcare systems is at an all-time high, industry innovation is keeping apace, with industry pipelines indicating over 7,000 medicines are currently in development. Products launched by companies will enter a very different launch environment in the mid-term. This will impact on all aspects of launch preparation.
“For HCPs, innovations or technologies that reduce the number of patients that come into hospital for treatment are key; and for patients, innovations (remote consultation, self-care devices or therapies) that empower them to manage their treatment independently are just as important.”
UK industry potential
The UK represents 3% of the global pharmaceutical business, but Ali believes its importance is underplayed.
“With some of the best talent, academic institutions, scientific infrastructure and of course the one-of-a-kind NHS, the UK could play an even greater leadership role than it does currently. Given the UK is home to a large proportion of biotech start-ups across Europe, it has great potential to lead the way in innovation and medicine development with the right investment and recognition.
“What I’d like to see is a clear recognition of what value the pharma industry brings to the UK economy and an understanding of the very high standards we operate with. Without this our public reputation will continue to be mediocre as it has been for the last few decades.”
For Ipsen UK&I it will be focusing efforts on several underserved disease areas across Oncology, Neuroscience and Rare Disease.
“My ambition is to unlock this potential and lead the way in adding value in these areas to our customers and their patients,”Ali says.
About the interviewee
Asad Mohsin Ali joined Ipsen in September 2018 as managing director, UK & Ireland, bringing more than 20 years of experience in the pharmaceutical industry. Since entering the industry as a graduate trainee in the late 1990s Asad has worked in big pharma (with Novartis and MSD) and biotech (with Tesaro), and now mid-sized biopharma with Ipsen UK&I. Over 22 years he has held a range of positions – in sales, marketing, portfolio management, strategy, value and access, and latterly in general management
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.
Lesson for all countries –> build your testing esp. as schools return. And don’t open everything at the same time, but rather step-changes bc if tests are not accessible or results take too long (longer than 24 hours), the entire test/trace/isolate system can’t function. https://t.co/IHhmalXrzP
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.
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.
School Nurses are around to support our schools when they reopen. I can’t wait to start my SCHPN course this September. Even better I’m doing it with my first team@HarrowNursing BBC News – Coronavirus: Missing school is worse than virus for childrenhttps://t.co/Jl0M2DY2e9
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.
I’m sure Prof Whitty is correct, but having worked in acute Medicine for over 20 years, we commonly see a spike in adult respiratory admissions around the 3rd wk of September, after schools go back; hospitals should plan and prepare for this https://t.co/4oAjfrWm1h
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.
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
As 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.
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.
The UK government has said it is vital to adopt telemedicine across the NHS in response to COVID-19 – but is the health service ready to fully embrace this digital tech? Dom Tyer investigates.
The rapid digitalisation of life during COVID-19’s acute phase has had a huge impact on healthcare delivery during the pandemic, part of which has included a major shift to telemedicine across the NHS.
Mirroring the acceleration of digital transformation in pharma, many face-to-face services have quickly been provided online or via telephone to keep patients away from overstretched and potentially infectious hospitals and GP surgeries.
The country is by no means out of the woods yet, and as I write this ‘only’ 77 people who tested positive for COVID-19 died yesterday in the UK (almost all in England). Nevertheless, plans for post-COVID healthcare are underway and digital health looms large within them.
The NHS’ digital future
As I noted in my last article, the signs of digital transformation – at a societal level, as well as across pharma – are everywhere. It’s a picture that also holds true for healthcare in the UK.
Speaking about the future of healthcare at the Royal College of Physicians recently, health and social care secretary Matt Hancock said COVID-19 had provided “a moment of exposure, of stark clarity”.
“From now on, all consultations should be teleconsultations unless there’s a clinical reason not to” Matt Hancock, UK Health Secretary
“Coronavirus has tested every single part of our infrastructure, giving us a new appreciation for what works and what doesn’t.
“And we saw things change. I mean really change. I’ve lost count of the number of times someone said to me: ‘what would have taken months took minutes’.”
COVID-19 has, he said, “catalysed deep structural shifts in healthcare that were already underway”, and he cited as examples data-driven decision-making, working as a system and telemedicine.
Indeed, he noted how telemedicine had enabled care at the peak of the pandemic and that the NHS has been treating half of patients in outpatients and primary care online. The difference between healthcare delivery just a year ago is stark.
“In the four weeks leading up to 12 April this year, 71% of routine GP consultations were delivered remotely, with about 26% face-to-face. In the same period a year ago, this was reversed: 71% face-to-face and 25% remotely,” he said.
Earlier this year, on a single day in March, over 750,000 online assessments were carried out and this trend towards digital is, unsurprisingly given his penchant for technology, something the health and social care secretary wants to see continue.
“From now on, all consultations should be teleconsultations unless there’s a clinical reason not to. Of course, if there’s an emergency, the NHS will be ready and waiting to see you in person – just as it always has been.
“But if they are able to, patients should get in contact first – via the web or by calling in advance,” Hancock said.
Expanding healthcare services
Appropriate use of telemedicine also has the backing of the pharma and biotech sectors, and features in the Life Sciences Recovery Roadmap put together by industry and medical charity bodies such as the ABPI, EMIG and BIA. The publication provides an overview of the critical issues affecting the sector as it moves towards recovery and building new partnerships with the government and NHS.
“Progress on telemedicine must be maintained and enhanced, where appropriate, to reduce pressure on acute care, support patient self-management, improve choice and address health inequalities,” the Roadmap notes.
“However, further research is required to assess where telemedicine is suitable for vulnerable people, such as those with learning difficulties. It is also important that telemedicine is not allowed to create new demand or provide an alternative route for people with self-treatable conditions to access a GP consultation when they should be practicing self-care with support, if required, from a pharmacist.”
In terms of vulnerable groups, an article in The Lancet Digital Health last month concluded telemedicine had promise for prison populations, potentially improving health service access, reducing widening health inequalities and contributing to improved health outcomes.
Meanwhile, Marie Stopes saw the potential of telemedicine early on in the pandemic, launching a service for early medical abortion care in April. Its initiative, which is in-line with NICE’s abortion care guidance, includes a nurse-led telephone consultation for eligible women.
“The government and the NHS must make sure that they bring everyone with them on this journey. In a recent survey, 50% of our members told us that they didn’t have access to a webcam” Andrew Goddard, Royal College of Physicians
The charity’s medical director Jonathan Lord said at the service’s launch: “We know first-hand the stress, anxiety and desperation that many women seeking abortion care are experiencing during the COVID-19 pandemic, and we are delighted that we have been able to implement an effective telemedicine service in response to the crisis.
“This would not have happened without a huge joint effort from both the independent women’s healthcare sector and the NHS, and we look forward to working in continued partnership to deliver the best possible care.”
But questions remain…
However, telemedicine must continue to improve if it is to deliver the best possible healthcare.
There are a range of safeguarding issues to be overcome, for example in sexual health, as the British Association for Sexual Health and HIV has noted. It held a joint webinar with The Faculty of Sexual and Reproductive Healthcare, which heard that telemedicine has been a great success during lockdown – improving access, providing a more patient-centred service and reducing complications and stress for women.
However, the webinar was also told how young people might not be able to plan how they engage with such services, or may be unable to make confidential telephone calls away from their family.
Another potentially problematic issue with telemedicine for healthcare providers is how it can be used to safely offer intimate examinations.
Such a delay is entirely understandable in the fast-moving, high-pressured environment of a pandemic, but it should also be a reminder of the balance to be struck between patient care and safety when applying digital health technology to sensitive areas of healthcare at high speed.
There are potential technological issues too. In 2018, the UK’s Office for National Statistics (ONS) classified 10% of the population as “internet non-users”, meaning they’d either never gone online or hadn’t done so in the previous three months. And there’s a digital divide, or at the very least a digital gap, in healthcare too.
Responding to Hancock’s speech at the Royal College of Physicians, its president, professor Andrew Goddard, said the RCP has been arguing for using technology to transform the way in which services are provided for some time.
“But the government and the NHS must make sure that they bring everyone with them on this journey. In a recent survey, 50% of our members told us that they didn’t have access to a webcam.”
Furthermore, early signs are that the massive spike in the use of remote NHS healthcare services during lockdown can’t continue. One rheumatologist in nearby Ireland ran 90% of his consultations remotely at the height of the pandemic, but by late July telemedicine accounted for just 10% of his workload.
Where next for telemedicine in the UK?
Interest in digital health technology has been following an upward curve for some time, but the demands of COVID-19 have generated a global wave of enthusiasm for its greater use. As a case in point, telehealth in the US is very much enjoying a moment in the sun right now.
Meanwhile, in the UK there’s clearly the political will and healthcare backing for wider use of telemedicine by the NHS, despite some, as-yet not entirely resolved, technological and safety issues.
So, as the country begins the slow process of trying to keep its infection rate curve ever-flatter, while holding on to those pandemic healthcare innovations that really worked, telemedicine in the UK finds itself at a pivotal point.
The question is: just how far will it return to the ‘old normal’ of underuse?
Based on the almost overnight explosion of its use during the pandemic’s acute phase, combined with telemedicine’s advantages for some patients of accessibility and responsiveness, a wider place for it in the post-COVID future of the NHS looks assured.
About the author
Dominic Tyer is a journalist and editor specialising in the pharmaceutical and healthcare industries. He is currently pharmaphorum’s interim managing editor and is also creative and editorial director at the company’s specialist healthcare content consultancy pharmaphorum connect.
After ditching its home-grown attempt at a coronavirus contact-tracing app for smartphones, the UK is ready to start pilot trials of a second version based on a platform developed by Apple and Google.
The new NHS Test and Trace app is intended to be used as an add-on to the controversial national and local contact-tracing service, and was due to start initial trials yesterday on the Isle of Wight and among a group of NHS volunteers.
Residents of COVID-19 hotspot Newham in London will also be offered the chance to participate in the pilot a little later, said the government.
The new app uses the decentralised Apple/Google model of relying on smartphones detecting each other, helping to maintain public privacy, even though the government has previously said the approach wouldn’t allow it to respond effectively to localised COVID-19 outbreaks.
It logs the time and distance a user has spent near anyone else, regardless of whether they are known to each other, and can raise an alert if one of them later tests positive for SARS-CoV-2, the virus that causes COVID-19.
The earlier NHS app would have been based on a centralised database of registered participants, but sparked concerns about infringement of civil liberties before it was abandoned.
The new app generates a random ID for a person’s device, allowing test and trace to function. The IDs “rotate”, according to the government, and that would prevent them from being used to track individual devices.
There are still concerns about how well the new app will work in practice, and the government says it is still working to improve the way phones detect each other using Bluetooth, and reduce the number of false alerts that would direct people to quarantine without due cause.
“The performance of individual devices varies wildly and there are currently variances in how Android and iOS report risk, but the experiments showed that the modelling was broadly correct,” according to the National Cyber Security Centre (NCSC), which is contributing to the app’s development.
“The team continue to work with Apple and Google to improve the contact detection reliability,” it said.
One issue is reportedly that in order to preserve privacy, the Apple/Google model doesn’t allow access to certain data on Bluetooth signal strength, according to a BBC report.
That means it can’t use a technique developed by researchers at the University of Oxford and Turing Institute in the UK that can give a better indication of proximity, it says.
The team behind the new app say they hope to get the accuracy rate high enough for widespread roll-out of the quarantine alert system by the end of the year.
Aside from its main contact-tracing function, the app will also inform users of the level of coronavirus risk in their postcode, and allow people to scan a QR code when they check into public venues like restaurants and pubs. They can then be alerted automatically if an outbreak is linked to that venue.
Other features include a symptom checker, a booking system for diagnostic tests, and a timer for those advised to self-isolate.
News of the revised app was welcomed by the Health Foundation charity, but its director of data – Adam Steventon – said the government must ensure that the trials are properly evaluated and that findings are shared.
“The ultimate test for the app is whether it increases the proportion of contacts that are traced by the Test and Trace service. Currently only 79% of people contacted by NHS Test and Trace are able to give information about their contacts, and only 75% of contacts can be reached,” he said.
The Health Foundation is concerned that false alerts from the app might impact more severely on certain populations, for example those unable to work from home.
It also wants reassurance that it hasbeen designed with the most vulnerable people in society in mind, such as those living in poorer areas, older people and some in minority ethnic groups.
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.
No more excuses, no more bluster – the government must now get the test and trace system sorted out.https://t.co/VgWu7oRsBh
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.
New research looks at the factors that speed up and slow down HTA appraisals for rare disease medicines across Europe.
Rare diseases drugs have always faced challenges when it comes to HTA approvals, even as governments bring in more regulatory policies that make their path through assessment easier.
Several factors make it difficult for HTA bodies often to assess orphan drugs, including a lack of robust trial data due to difficulties in finding patients, the absence of randomised controlled trials, the use of surrogate endpoints, and the lack of active drug comparators.
A new analysis from consulting firm CRA has honed in on the different challenges faced in four EU markets – England, France, Germany and Scotland – and looked at how manufacturers can increase the chances of a successful appraisal.
The research analysed more than 70 EMA-approved rare disease therapies and compared reimbursement recommendations from the regulatory bodies in each country, to see how HTA decisions potentially prolonged time to reimbursement.
The results show that HTAs for orphan drugs can vary widely across Europe, causing inconsistencies in evidence requirements and recommendations.
Rates of approval
The study reviewed all 80 European Medicines Agency (EMA) authorised drugs receiving an orphan designation between 1 January 2013 and 31 December 2019, analysing their HTA outcomes and time to reimbursement across France, Germany, England and Scotland.
A comparative analysis was then conducted on the 71 approved drugs that achieved a negotiated price in at least one of the four markets.
Germany had the highest approval rate of orphan drugs at 98% – however most of these recommendations (73%) were awarded a ‘non-quantifiable benefit’ rating, the automatic rating for an orphan drug, which shows the regulator did not see any benefit compared to comparator products (see graph 1). The authors also note that orphan drug trials with higher p values and surrogate endpoints are often accepted for assessment in the country.
A more favourable outcome from the German regulator took on average 1.4 times longer to achieve a final negotiated price (708 versus 510 days).
Graph 1: Assessment of the HTA outcome in France, Germany, England and Scotland of all orphan drugs that obtained an EMA approval between 2013-2019. N above each bar equals the number of drugs reviewed by the respective HTA body. Source: CRA Analysis
France and England had comparable approval rates (92% and 91%, respectively); however, France reviewed almost twice the number of orphan drugs over the period of analysis (67 versus 35). Only 19% of the orphan drugs in France were awarded an Amelioration du Service Médical Rendu (ASMR) rating of V, which indicates no improvement in medical benefit. Drugs with an ASMR IV-V rating were reimbursed in 427 days, compared to 585 days for products with ASMR I-III (see Graph 2).
Graph 2: Comparison of the time to reimbursement (days) for EMA orphan drugs approved from 2013- 2019. Orphan drugs were reimbursed in at least one of the selected markets (N = 70). N within each bar equals the number of drugs with each outcome reviewed by the respective HTA body. Source: CRA Analysis
In England, two key mechanisms were often used to achieve approval: label restrictions or a patient access scheme (PAS).
Over one third (37%) of orphan drugs appraised by NICE only achieved approval in a positioning or population that was restricted versus the full regulatory approved label. Companies that accepted such restrictions saw faster approval time compared to no drugs with restrictions (407 versus 505 days).
Meanwhile, although introducing a PAS improved the chance of approval, the analysis suggests that it actually delays the overall appraisal time (523 versus 311 days).
Scotland had the highest rate of non-approval for orphan drugs. Thirty-three percent of drugs reviewed were not accepted, despite specific modifiers in place for rare disease products, including the incorporation of the patient voice through the country’s Patient and Clinician Engagement (PACE) meetings – which were included in 74% of orphan drug submissions between 2013-2019.
Improving HTA outcomes
The authors conclude that while the various concessions and modifiers introduced by different governments have a positive impact on minimising rejections and accelerating approval times, there are still challenges in capturing the full value of orphan drugs within the HTA process.
“Achieving more favourable outcome ratings, avoiding restrictions, or addressing uncertainty with a PAS all lead to prolonged appraisal times,” the authors say. “Manufacturers are therefore still required to consider carefully their HTA launch strategy and complement this with additional evidence generation and engagement from a wider stakeholder group.”
The authors outline several approaches companies can take to improve the chances of success in orphan drug approvals and overcome the challenge of having limited data and evidence available.
One approach is to agree methods for ongoing real world data collection post-launch with HTA bodies.
They add that “creative” solutions to real world evidence collection could help, such as developing apps for patients and HCPs.
Meanwhile, it is also important to strive for wider engagement with the rare disease community and other stakeholders.
Listening to views from patients and HCPs can help with processes like Scotland’s PACE meetings – but more indirect forms of stakeholder engagement may also improve HTA outcomes, as the value-added services provided to these stakeholders can be leveraged during negotiations.
“For example, Galafold, an enzyme replacement therapy for Fabry’s disease, is primarily differentiated from existing treatments by providing a reduction in administrative burden,” the authors say. “Despite this, Galafold was able to achieve an ASMR IV in France, recommendation by NICE, and was accepted for restricted use in Scotland.”
They note that the perception of Galafold’s value may have been improved by the additional value-added services the manufacturer, Amicus Therapeutics, offered to a wider stakeholder group
For example, Amicus reimbursed amenability tests for patients with unknown mutations that could be referenced against Galafold’s amenability table via a physician support website. This service was accepted in the NICE evaluation as something which avoided additional resource implications for the NHS.
For more information contact the report’s authors:
The two global companies will supply up to ~60M doses of a vaccine to the UK’s government to combat COVID-19
Sanofi leads the clinical development and registration of the vaccine and expects a P-I/II study to be initiated in Sept’2020, followed by a P-III study by the end of 2020. Additionally, the companies are expecting the approval in H1’21
The vaccine is deploying Sanofi’s S-protein COVID-19 antigen together with GSK’s adjuvant technology. Both the companies are scaling up manufacturing of the antigen and adjuvant to produce up to 1B doses per year
Click here to read full press release/ article | Ref: Sanofi | Image: PharmaShots
The UK government has signed its fourth coronavirus vaccine deal, snapping up 60 million doses of an experimental shot being developed by Sanofi and GlaxoSmithKline.
The order has come in even before the vaccine has started clinical development, with a phase 1/2 trial not due to get underway until September. Sanofi is providing the vaccine candidates itself while GSK is contributing the adjuvant used to boost immune responses to it.
The UK has already claimed 100 million doses of the ChAdOx1 vaccine in development at the University of Oxford and AstraZeneca, as well as 30 million doses of a candidate from BioNTech/Pfizer and 60 million doses of another shot created by Valneva.
There has been speculation that the government was gearing up to place an order for the Sanofi/GSK vaccine over the last few weeks, and it is the first official order to come in since the two companies started collaborating on the programme in April.
The value of the order has not been publicly disclosed but has previously been estimated at up to £500 million.
Orders for the vaccine candidates in development for COVID-19 are coming in thick and fast from western governments, leading to concerns of “vaccine nationalism”, with affluent countries buying up the first available stocks and leaving poorer nations at the back of the queue.
The US and the UK – both among the countries worst affected by the pandemic – have been particularly active in signing coronavirus vaccine access deals.
GSK and Sanofi maintain they are committed to making their vaccine available around the world, and plan to provide a significant portion of total worldwide available supply capacity in 2021/22 to ACT Accelerator, an organisation set up to secure access to COVID-19 tests, treatments and vaccines in lower-income countries.
Our discussions continue with other govts and orgs, and we’re committed to providing a significant portion of worldwide supply to the Access to COVID-19 Tools Accelerator in 2021/22. We don’t expect to profit from #COVID19 vaccines during the pandemic. https://t.co/y2DFtVGEsCpic.twitter.com/ioQA0w8ZiQ
“With today’s announcement, the government has now secured early access to four different types of immunisation and a total of 250 million doses, giving the UK the most likely chance of finding a safe and effective vaccine at the quickest speed,” said the Department of Business, Energy and Industrial Strategy in a statement.
Sanofi’s recombinant vaccine is based on the same baculovirus production technology used in its seasonal influenza shots.
The Oxford/AZ candidate uses a non-replicating adenoviral vector, while BioNTech/Pfizer’s candidate is based on messenger RNA and Valneva’s comprises an inactivated form of SARS-CoV-2, the virus that causes COVID-19.
Having different types of vaccines in line is important, according to Kate Bingham, chair of the UK government’s Vaccines Taskforce, because it’s not yet clear if any of them will be effective.
“Whilst this agreement is very good news, we mustn’t be complacent or over optimistic,” she cautioned.
“The fact remains we may never get a vaccine and if we do get one, we have to be prepared that it may not be a vaccine which prevents getting the virus, but rather one that reduces symptoms.”
We have secured access to 60m doses of another promising #coronavirus vaccine.
Our scientists are working at pace to develop potentially lifesaving vaccines, and these agreements increase our chances of finding a safe and effective vaccine quickly. https://t.co/gG7mb0LPuz
GSK and Sanofi are planning a phase 3 trial of their candidate by the end of 2020, which if positive could lead to regulatory approvals in the first half of 2021. They are scaling up production with the aim of producing a billion doses a year.
At last count there were 25 coronavirus vaccines in clinical development, according to a World Health Organisation update published yesterday, plus 139 candidates in preclinical testing.
A new NHS service has been launched to enable people across the UK to seek information on COVID-19 vaccine trials, and has received 72,000 sign-ups, according to the government.
UK manufacturing exports fell sharply during the second quarter as the COVID-19 pandemic gathered pace, but pharma and healthcare products were much less affected, says a Lloyds Bank report.
The latest quarterly UK International Trade Index reveals that the Chemicals and Plastics sector – which covers pharma and healthcare – was particularly resilient in the initial stages of the crisis as customers stockpiled products in anticipation of longer lead times ahead.
That was already evident in many pharma companies’ first-quarter results, and according to Lloyds seems to have been maintained in the second quarter, with “near panic buying” for some medical equipment.
“This segment has been less exposed to the cyclical downturn in non-essential spending as lockdowns were put in place across the world in response to public health emergencies,” says the report.
Some drugmakers – including Novartis – have however reported an impact from the pandemic on sales in the second quarter resulting from fewer interactions between patients and prescribers.
All manufacturing sectors have been affected by lockdown measures, particularly the availability of air freight which has squeezed cargo capacity, but Europe became the epicentre of supply delays in the second quarter as the pandemic reached a peak there.
The authors of the report also point to some positive signs across economies around the world as containment measures relax and the focus moves to recovery, including a return to growth in China in the second quarter as well as gains in Turkey and Australia. Stabilisation and signs of recovery were evident in May and June in the UK.
The report comes shortly after the Bank of England’s chief economist Andrew Haldane suggested that while it is too early to call the shape of the UK recovery it is “so far, so V” – referring to the much hoped-for rapid bounceback fuelled by consumer spending.
He also said the economy was on course for a contraction in the second quarter of about 20% compared with the final three months of 2019, and that the greatest risk to the UK was “high and long-duration unemployment rates” as furlough schemes start to taper off.
Lloyds says its figures show that the world economy is growing faster than the UK, which is positive for UK net trade given that exports from the country showed a double-digit fall overall in the second quarter.
“Nevertheless, the net impact of the health crisis remains significantly negative even with the improved outlook since April,” according to Lloyds.
It also says that companies will have to look closely at their supply chain weaknesses – such as a focus on “just-in-time” supply to boost efficiency and reduce costs – which have been thrown into stark relief by COVID-19.
“Export measures hit an all-time low in Q2 although we see small signs of recovery as early as May and into June,” said Gwynne Master, managing director and global head of trade for Lloyds Bank Global Transaction Banking and one of the authors of the report.
“While it is too early to talk about the trajectory of recovery, it is encouraging to see enhanced external demand, signs that China’s economy is stabilising, and some UK consumer goods’ export growth in June.”
She added: “Government schemes and finance options continue to be made readily available, which will help UK exporters continue to trade, to position for a return to normality to international trade, and to prepare now for potential future disruption.”
David Peacock has worked across the world for MSD and other companies, and has now taken the helm of MSD’s UK & Ireland operations. In the latest article in our UK Leaders series, he tells us about the opportunities and challenges he is excited to take on in the region.
Peacock has had a strikingly international career, including jobs in the US, Australia, Singapore, Vietnam, Japan, and Hong Kong – working across many different roles in various healthcare companies and even setting up his own business, hydration tablet company Nuun.
The UK might seem like a small change after all that, but Peacock said he was “tremendously excited” at the opportunity to be heading up MSD’s business in the region.
“The world watches the UK when it comes to market access, whether that’s in terms of HTA or the commercial relationships that are established with Public Health England and the NHS,” he says.
“The health system continues to move as the NHS and our industry continue to adapt. Through that process of adaptation, MSD in the UK has led trends for the company globally. I was very excited about having the opportunity to come in and work with a group that has this reputation for embracing new ideas.”
Peacock says his goal coming into the company is to maintain that reputation and build “true partnerships” with the NHS.
“The NHS is a large organisation to change. Getting people aligned, not just in their understanding, but also in their beliefs, is hard.”
“I recognise that I’m coming into the UK when the country is dealing with affordability challenges,” he says. “One of my goals is to understand how this healthcare system is operating, and what challenges it is facing.
“I’m a true believer in partnership and collaboration. What I’m looking for, as I come into this business, is how we can continue to move past the traditional promotional models – like simply pushing messages out to clinicians that represent the risks and benefits associated with our products.
“Those models worked for many years, but they’re not going to be good enough going forward. We need to move past being suppliers to healthcare systems towards becoming true partners. We have skills, expertise, and assets that we can bring to bear to help solve the NHS’ problems. My goal coming into the UK is really to keep us on the path to achieving that.”
Bringing pharma and the NHS together
Luckily, Peacock says that the foundations of this kind of thinking are there for MSD and the NHS to build upon, and he has seen “bright spots” in the health service where these kinds of partnerships already exist.
“The joint working opportunities in the NHS are fantastic – we can go into a meeting space with the NHS and clearly put on the table that we want to work together to solve a problem. That framework doesn’t really exist in most of the world; there is real intent for partnership here.”
Peacock adds that if the sector is to truly solve issues around achieving better patient outcomes, we will need more forums where we can bring together a multitude of partners.
“We have to bring in not just pharma and the NHS but also other industries and patient groups, and tackle problems in a systematic way. At the moment we tend to focus on a particular capacity constraint.”
He says that the patient voice in particular is central to this – especially when both health systems and pharma share the goal of saving and improving lives.
“I don’t view our industry as majorly competitive because we’re all aiming at that same goal. We may use different words, but we’re completely aligned on it.
“The focus is on the patient, so we can’t look at them in a detached manner – we have to actually engage and understand their circumstances, their wants and needs, and their environments. The only way that we can really do that is to directly engage with patient organisations.
“Ultimately, we want them to guide us. I think the NHS wants the same. We just have to figure out a way to connect all these pieces together.”
Peacock says enthusiasm for change from the NHS’ side can vary depending on where you look – although there are pockets of great progress.
“I have certainly talked to people in the NHS who are passionate, who are frustrated and, equally, are hopeful. The NHS is on its own journey, with its own long-term plan. I’m fully supportive of the efforts they’ve got underway, but it’s a large organisation to change. Getting people aligned, not just in their understanding, but also in their beliefs, is hard.”
And from MSD’s side of things, Peacock says that it is important the company considers how its internal structures and its employees can contribute to better collaboration.
“We need to make sure that we have the people, the mindsets, and the skillsets for collaboration, so that when we engage with the system, we’re able to articulate the value that we bring in a way that is not seen as just being self-promotional. That takes time, though, because the reality is that it hasn’t been the operating model for most of the industry’s lifespan.
“It will involve reviewing how we incentivise our people, how we recruit, and how we allow employees to get a broader perspective. Traditionally, people have focused their careers on one area, e.g. commercial, R&D, medical, etc. We need to look at how we can create opportunities for people to move across those boundaries.
“We’ll still need specialists, of course, but we also want generalists who have familiarity with different types of problems and how to solve them.”
Progress during COVID-19
In some ways, he says, the COVID-19 pandemic has set back this progress – and the industry will need to think about how it engages with the NHS to get them back on track – but in other ways, the crisis has accelerated such thinking.
“We’re certainly seeing more people looking at things from an end-to-end perspective. The crisis has forced the system to actually look holistically and break down some of the silos that exist within the NHS to drive better patient outcomes.
“When we were providing products that were needed in ICUs, the NHS was quickly able to break down barriers, work directly with us to source and supply from global outlets all over the world, and bring in the MHRA to provide regulatory support to enable products to flow directly into the country and straight into the hospitals. The speed at which we did that was astounding – things that would normally take us a year were happening within 48 hours.
“That shows the NHS can move rapidly when there’s purpose. My hope is that we can build on these initial stages and make it real. We just don’t want to go back to those old ways where it’s committee after committee after committee.”
In general, though, Peacock says he remains excited about the future of the UK ecosystem.
“The UK has so many opportunities – a single health system, a single payer environment, and an immense amount of data and scientific publications. All the pieces are in place now – we just need to look at how we can tie them together.
“People should feel positive. What we need to do now is help the people who have these dreams to realise a better future.”
About the interviewee
David Peacock is currently serving as the managing director for MSD in the UK and Ireland, having taken up his position in 2019. Prior to this David served as the chief of staff for the office of the chairman, president and CEO of Merck & Co. Over the course of his career with MSD David has gained a broad set of leadership experiences including serving as the CFO for MSK K.K. in Japan, leading the company’s business in Hong Kong & Macau, and successfully delivering in a variety of other commercial roles based in Japan, Vietnam, Singapore, and the United States.
A new study by Choi et al. (2020) compares the health of individuals aged 55-64 in the US compared to England. They use data from Health and Retirement Study (HRS)–in the US–and the English Longitudinal Study of Ageing (ELSA) for 2008-2016. Health is measured across 16 outcomes: 5 self-assessed outcomes, 3 directly measured outcomes, and the prevalence of 8 key comorbidties. They find:
Cross-country differences in health were in favor of England for all health outcomes except ADL limitations, depression, and measured blood pressure, which were not significantly different between countries.
Of perhaps more interest is that they compare health outcomes among individuals in the top compared to bottom income deciles across countries. In this analysis, they find:
Among individuals in the lowest income group in each country, those in the US group vs the England group had significantly worse outcomes on many health measures (10 of 16 outcomes in the bottom income decile); the significant differences in adjusted prevalence of health problems in the US vs England for the bottom income decile ranged from 7.6% (95% CI, 6.0%-9.3%) vs 3.8% (95% CI, 2.6%-4.9%) for stroke to 75.7% (95% CI, 72.7%-78.8%) vs 59.5% (95% CI, 56.3%-62.7%) for functional limitation. Among individuals in the highest income group, those in the US group vs England group had worse outcomes on fewer health measures (4 of 16 outcomes in the top income decile); the significant differences in adjusted prevalence of health problems in the US vs England for the top income decile ranged from 36.9% (95% CI, 33.4%-40.4%) vs 30.0% (95% CI, 27.2%-32.7%) for hypertension to 35.4% (95% CI, 32.0%-38.7%) vs 22.5% (95% CI, 19.9%-25.1%) for arthritis.
The authors do note that these differences persist even after statistically controlling for demographic factors, educational level, smoking, and body mass index. Note that the outcomes are relatively crude measures and the data cover only individuals aged 55 to 64. Further, the study describe what these differences are, but not they why of why these occur. Nevertheless, these results do provide a helpful snapshot comparing the health of Americans and the English across income strata.
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