2021 – UK market access prospects

With a new year comes the opportunity to think ahead for the market access landscape for the coming year. 2020 was a big year for market access initiatives in the UK, many of which are only just starting, and their impact will come through in 2021 and beyond. Looking back at key market access news from the last year, Leela Barham takes stock of what the next twelve months could bring for UK market access.

Whilst regulatory approval is only a starting step on market access, the UK has made steps to put the UK on the priority list for companies seeking marketing authorisation. The UK joined two initiatives in October 2020 that should bear fruit in the future: Project Orbis and the Access Consortium.

Project Orbis is coordinated by the US Food and Drug Administration and includes Canada, Australia, Switzerland, Singapore and Brazil and focuses upon review and approval of cancer treatments that offer promise. The Access Consortium includes some of the same players – Australia, Canada, Switzerland and Singapore – and looks more broadly at securing patient access to high-quality, safe and effective medicines. The ABPI has seen these as another way to help the UK deliver faster access.

The promise of faster market access 

Announced in December 2020, and beginning from 1 January 2021, the new licensing and access pathway (ILAP) at the Medicines and Healthcare Regulatory products Agency (MHRA) offers the chance – for a fee – for faster access for medicines that meet the criteria for the scheme. Criteria include that the condition is life-threatening or seriously debilitating or there is a significant patient or public health need.

“The UK has sought to provide incentives for development of new antibiotics by offering a volume neutral deal, also termed a Netflix approach”

Additional criteria include being an innovative medicine, such as an advanced therapy medicinal product (ATMP), significant new indication or a treatment for a rare disease or other special populations such as neonates, children, elderly and pregnant women and aligning with priorities of the UK, be that from the chief medical officer, the Department of Health and Social Care (DHSC) or those from the UK’s Life Sciences Sector Deal.

What is probably most exciting about ILAP is the bringing together of expertise from the MHRA with that of key HTA agencies NICE and the Scottish Medicines Consortium (SMC), as well as NHS England and NHS Improvement (NHSE&I), and, of course, patients too. It’s the first time that all these agencies are working together (much else draws on what has been available for some time; early advice and in parallel between NICE and MHRA).

2021 will reveal just how the ILPA will be operationalised and whichever product will be the first to go through the Innovation Passport stage – a new designation as part of ILAP – will help everyone understand how the criteria for the passport will apply in practice. It’ll also reveal just what the Target Development Profile (TDP) living document will cover and how it can evolve over time. This rolling review features is also another new element on offer under ILAP.

The Netflix approach

Antibiotics are vital to modern health care, yet there have been warnings for years that the economics at play simply don’t incentivise the development of new antibiotics; new antibiotics should be kept in reserve to limit antibiotic resistance and aren’t likely to be used in large volumes. The lack of new antibiotics is a problem when the existing antibiotics stop working.

The UK has sought to provide incentives for development of new antibiotics by offering a volume neutral deal, also termed a Netflix approach. In June 2017, NICE and NHSE&I launched a novel payment approach; offering up to £100 million to companies developing and marketing novel antibiotics based on the value of their products, regardless of volume sold.

2021 will see NICE trialling their adapted HTA approach for assessing the value of new antimicrobials. This will not only interest companies researching and developing new antibiotics, but other countries who will want to see whether the UK approach could work for them too. Where NICE leads, others will want to learn and adapt from.

Key access commitment due to report in VPAS 

The UK has a unique approach to managing the pricing of branded medicines through the 2019 Voluntary Scheme for Branded Medicines Pricing and Access (VPAS). The deal balances affordability commitments that benefit the UK government – where NHS spend on branded medicines cannot go above a pre-agreed level – with access commitments to industry.

A key commitment in the deal is to reach the upper quartile of uptake in relation to comparator countries for the five highest health gain categories of treatments. A timeline is set for hitting this too; it should be met during the first half of the scheme. That makes it a target for June 2021. Whilst there isn’t a timeline against it, there is also a commitment for the Department of Health and Social Care, NHSE&I and the industry association, the Association of the British Pharmaceutical Industry (ABPI) to better understand national and international variation on uptake and where that is unwarranted.

Realistically it’s seems unlikely that the timetable for the upper quartile target can be met. That’s because so far, based on desk research, there doesn’t appear to be a public list of the five highest health gains, and meeting minutes from operational reviews of the scheme suggest discussions have faced difficulties in agreeing them. It’s difficult both from a methods side (which countries, which treatments, etc) as well as data and time; COVID-19 has been a higher priority for many staffers involved. Expect a delay that may even see this reporting in 2022.

More change at NICE

2020 saw NICE consult on a variety of changes and set out plans on cross-cutting market access issues.

NICE started 2020 with the January publication of their principles; essentially a framework for how the agency goes about its work – from working on national priority areas through to publishing their work and updating as necessary.

NICE followed these in March 2020 with their final statement of intent on increasing the use of health and social data in their guidance. The statement covers one of the buzz phrases in market access – ‘real world data’ – and signals a greater willingness for NICE to consider its use in their work. The agency will keep working on how this will be implemented during 2021; this can only help, as much in the statement is aspirational and there’s not a great deal of clarity on exactly what NICE will accept, or not, as the case may be.

In June 2020, NICE let industry know about their changes to the process used in Single Technology Appraisals (STAs), moving from technical reports from Evidence Review Groups (ERGs) – independent academics – to present issues. That allows for easier engagement but also offers companies a right to reply to ERGs. With this change applied from STAs starting in May 2020, 2021 should see more companies seeing the difference. Efficiencies are the gain NICE hopes to see and that ties in with commitments to make NICE faster set out in VPAS. NICE hadn’t yet reached their targets according to metrics set out in July 2020 relating to appraisals up to Q1 of 2020, so 2021 could see NICE really getting to grips with getting faster.

NICE kept up the pace of consulting in 2020, setting out proposals to change the selection of treatments for evaluation in October 2020. The aim is for simplification as well as confirming promises made in VPAS that NICE will appraise all new active substances and significant new indications.

Arguably the most important consultation from NICE in 2020 was a six week consultation on the evidence and considerations for changing their methods, including those used in Technology Appraisals. According to experts at market access consulting firm, Bresmed, the changes being tabled that are likely to have a high potential impact include the removal of End of Life criteria to be replaced with a severity modifier, a change to the discount rate from 3.5% to 1.5% as well as greater emphasis on real world data.

Yet there is still more work for NICE and all stakeholders who will want to shape the final changes, as the agency plans to consult for another six weeks from February to March 2021 as well as consult with stakeholders on the draft programme manual which will encompass the reformed methods in June to July 2021.

The final changes won’t bear fruit for a little more time; implementation will be for treatments assessed from October 2021. This will also be when changes to selection processes will be implemented too. Companies are going to need to keep refining their inputs to NICE as they consult as well as run scenarios and review strategies to make the most of any opportunities for treatments that will be reviewed under the new approach.

Less clear is the ‘what and when’ of changes to the criteria that determine which treatments are reviewed through the NICE Highly Specialised Technologies (HST) programme, another area that NICE is reviewing. This is of interest as it offers a wider set of value components to be considered, as well as more flexibility on the cost-effectiveness threshold, for ultra-orphan treatments.

It’s likely NICE will set out details on this review during 2021. However, anyone hoping that the door will be open for the rarer end of common treatments could be disappointed as NICE say that they want to make the criteria clearer and more specific but not increase, or decrease, the number of HST topics. NICE is funded to do three a year.

NICE, building on their international links, was one of seven agencies who took part in the first ever World Evidence-based Healthcare Day in October 2020. We can expect to hear more on the global initiative in 2021. It’s also likely that their 2020 agreement with Colombia’s IETS will start to bear fruit, shaping how HTA is done in Colombia.

Pulling all this work together will also be a test of NICE’s 2020 appointed chief executive Gillian Leng. She’ll likely bring a steady hand to the consultation and the next steps, reflecting her over 13 years at NICE. 2021 could see big changes that should provide opportunities for faster NICE appraisals but as ever, the devil is in the detail and the full impact won’t be possible to see until a number of treatments have gone through the new methods and processes.

Medicines and Medical Devices Bill and a new Innovative Medicines Fund 

The Medicines and Medical Devices Bill is still going through Parliament and should become law in 2021. It’s part of the legal homework necessitated by the UK leaving the EU and covers a range of regulatory issues.

The importance of the Bill from a market access perspective is not however just about regulation but has also seen discussion of the establishment of an Innovative Medicines Fund. Whilst a proposed amendment to the Bill on establishing such a fund was withdrawn during December 2020, the discussions in the House of Lords suggested that NHSE&I and NICE will be engaging during the first quarter of 2021 on the fund.

The fund will replace the Cancer Drugs Fund (CDF), and widen the treatments that can be funded on an interim basis. The CDF as it has been run from 2016 helps generate evidence for treatments that have the potential to be cost-effective but face uncertainties that can be addressed through evidence generation. It’s proved to be a key enabler for market access for cancer drugs and many – although not all – drugs funded under the CDF have gone on to go into routine commissioning, although often with a hefty price cut. Just how the Innovative Medicines Fund will work is something to watch as it may provide new access opportunities previously not available.

More change ahead in 2022

There will be yet more change in the future with major shakeups expected in the way NHS England is structured, with the potential to abolish Clinical Commissioning Groups (CCGs) and Integrated Care Systems to be put on a statutory footing in 2022. That means much planning could be on the cards and, as ever, a need for the pharma industry to keep up to date and ensure that they’re engaging with those that matter for access on a local level.

About the author

leela barhamLeela Barham is researcher and writer who has worked with all stakeholders across the health care system, both in the UK and internationally, on the economics of the pharmaceutical industry. Leela worked as an advisor to the Department of Health and Social Care on the 2019 Voluntary Scheme for Branded Medicines Pricing and Access (VPAS).

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Moderna’s COVID-19 vaccine is third approved in UK

The UK has approved the Moderna coronavirus vaccine, hard on the heels of its go-ahead in Europe, although supplies are not expected to arrive for several weeks.

Moderna’s mRNA-1273 is the third COVID-19 vaccine to be approved for use by the Medicines and Healthcare products Regulatory Agency (MHRA) and is the second mRNA vaccine after Pfizer/BioNTech’s Comirnaty, which got the nod in December.

The UK government has ordered 17 million doses of the new vaccine, but none will be available before March, when Moderna is able to bring new production capacity online.

That means for now, the country’s immunisation programme will continue to rely on Comirnaty and the AstraZeneca/University of Oxford shot approved just before the New Year.

Around 1.5 million people in the UK have received at least one dose of either Pfizer/BioNTech or AZ vaccines, and that includes around a quarter of the over-80s age bracket who are particularly vulnerable to COVID-19.

The government’s target is to vaccinate 15 million people – around 22% of the total population – by the middle of next month.

The UK is facing a marked escalation in cases however, with the attest daily figures showing 68,000 new cases and 1,325 coronavirus-related deaths, and with a more transmissible strain of SARS-CoV-2 threatening to overwhelm the NHS.

The latest vaccine approval was welcomed by NHS Confederation chief executive Danny Mortimer, but he also stressed that “it does not mean the COVID-19 crisis today is over, especially as a major incident is declared in London, hospitalisations for coronavirus continue to rocket, and as many as one in 50 people are now infected.”

He went on: “It will…be weeks and months until the NHS feels the benefit of the vaccination programme.”

Moderna’s shot claimed conditional EU approval earlier this week, and the first supplies will start to arrive in Europe next week, according to Moderna. The European Commission has ordered 160 million doses, but Brexit means the UK will not benefit from the EU’s allocation and rollout plans.

Meanwhile, mRNA-1273 was also granted emergency use authorisation by the FDA on 18 December, with the US scheduled to receive 20 million doses by the end of 2020. Moderna has also said it aims to make 100 and 125 million more doses available in the first quarter of 2021, of which 85 to 100 million have been claimed by the US

The approval is based on trials showing mRNA-1273 had 94% efficacy in preventing disease, including in the elderly, roughly the same as the Pfizer/BioNTech shot and a little better than the 70% protection rate seen with AZ’s candidate.

The Oxfam charity welcomed that the UK now has more than enough vaccine on order to protect the entire population during 2021, but called for vaccine developers to share the science and technology behind them worldwide so less well-off countries don’t miss out.

“Nine in 10 people in the poorest countries are set to miss out on a vaccine unless the UK government and companies like Moderna urgently shift position,” said Oxfam’s health policy manager Anna Marriott.

“A failure to act is not just wrong but self-defeating and short-sighted – as long as the virus is allowed to spread in other parts of the world, public health and economic recovery in the UK will continue to be under threat,” she added.

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AZ COVID-19 vaccine cleared in UK, dosing to start next week

The UK has approved AstraZeneca and Oxford University’s COVID-19 vaccine AZD1222 in another significant step forward in the fight against the pandemic, with first doses due to be administered on Monday.

The UK government has already ordered 100 million doses of the adenovirus-based shot, enough to vaccinate 50 million people, adding to the 40 million dose order of the Pfizer/BioNTech shot – now known as Comirnaty – that was approved earlier this month.

The UK is the first country to approved AZD1222, and AZ says it is preparing to provide “millions of doses” in the first quarter of 2021, while building capacity for three billion doses for delivery worldwide by the end of the coming year.

The emergency approval comes as millions more people in the UK are facing tighter lockdown restrictions after another daily record of more than 53,000 confirmed new coronavirus cases yesterday.

Health Secretary Matt Hancock warned that while the rollout of AZD1222 brings forward the end of the pandemic, mass vaccination will take time and people should “hold their nerve” to avoid swamping the NHS in the first few months of 2021.

He told the BBC this morning that he now has “a high degree of confidence that we can be out of this by the spring.”

The Joint Committee on Vaccine and Immunisation (JCVI) has set out priority groups who will receive the vaccine, and as with the Pfizer/BioNTech jab first in line will be the over-80s and health and social care workers. So far, more than 600,000 people have received Comirnaty since dosing started on 9 December.

AZ chief executive Pascal Soriot said that millions of doses of AZD1222 have already been produced and are being filled, ready to ramp up supply as the UK immunisation programme gathers pace.

Soriot confirmed that AZ should be able to provide enough vaccine to meet the UK government’s target of a million doses per week “very rapidly” with the first doses due to be delivered to clinics “today or tomorrow.”

He also said that AZD1222 provides a reasonable level of protection from the coronavirus after a single dose, and as the second dose only needs to be given within 12 weeks, that provides an opportunity to immunise more people, more quickly.

In turn, that should start to reduce mortality and hospitalisation from COVID-19 and ease pressure from the NHS as cases continue to surge.

The AZ vaccine can also be stored, transported and handled at normal refrigerated conditions for at least six months making it more suitable for delivery to parts of the world with less sophisticated healthcare systems than the Pfizer/BioNTech shot, which requires colder storage.

Soriot also reiterated his view that AZD1222 should provide protection against the new, more transmissible strain of the SARS-CoV-2 virus that causes COVID-19.

The first case of that has now been identified in the US, along with dozens of other countries, but new research suggests that while it is easier to transmit it isn’t any more likely to cause severe disease.

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved two full doses of AZD122, which has a top-line protective efficacy of 62%, as it decided there wasn’t enough data on a half dose/full dose combination that seemed to be more effective in trials with 90% protection rate.

The British Medical Association’s council chair Dr Chaand Nagpaul, welcomed the approval, but warned the rollout will require a massive step up in immunisation capacity.

“It is now crucial that supplies of this vaccine are given to as many GP practice sites and hospital hubs as possible and that this happens as quickly as possible so that we can begin vaccination en masse,” he said.

“We need to see a step change in distribution so that doctors can protect their patients and communities, beginning with those most at risk, and crucially this must include health and social care workers as they confront the virus on the front line.”

The BMA has previously said it is concerned about patchy access to the Pfizer/BioNTech vaccine by healthcare workers across the country.

EU orders another 100m doses of Comirnaty

The EMA is still reviewing the AZ vaccine, but yesterday exercised an option to acquire another 100 million doses of Comirnaty for distribution in the EU in 2021, taking the tally to 300 million doses.

Pfizer and BioNTech say they will be able to meet that order, agreed just two days after the first vaccinations against COVID-19 started in EU member states. The companies have previously said they will be able to supply up to 1.3 billion doses worldwide by the end of 2021.

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AstraZeneca’s AZD1222 Receives MHRA’s Emergency Supply Authorization in the UK

Shots:

  • 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

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FDA aims for fast approval of Moderna’s COVID-19 shot after panel vote

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

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

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

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

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

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

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

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

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

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

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

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

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Pfizer & BioNTech Receives MHRA’s EUA for BNT162b2 Against COVID-19

Shots:

  • 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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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MHRA looks to AI to hunt for COVID-19 vaccine side effects

The UK drugs regulator has awarded a £1.5 million tender to a software company for an artificial intelligence tool that will be used to process “the expected high volume of COVID-19 vaccine adverse drug reactions (ADRs).”

The tender awarded to Maidenhead, Berkshire-based GenPact UK aims to “ensure that no details from the ADRs…are missed” as the UK prepares to start rolling out COVID-19 vaccines – assuming their safety and efficacy is supported in late-stage trials.

The company is a subsidiary of US group GenPact, which already offers an AI and machine learning based tool called Cora PharmacoVigilance that can be used to identify patterns in data to foresee potential side effects.

The contract from the Medicines and Healthcare products Regulatory Agency (MHRA) recognises that the timelines for the development of coronavirus vaccines has been accelerated so fast that a complete picture of their safety may not be available when they start to be used in widespread immunisation campaigns.

Former UK Prime Minister Tony Blair said this morning that the AstraZeneca/Oxford University adenovirus-based vaccine AZD1222 should be rolled as soon as possible – ideally in the month of November – as “it is essentially safe.”

He also called for experimental drugs being tested in the UK RECOVERY trial to be used in any hospitalised patients at risk of serious illness from COVID-19, in a foreword to a report on the pandemic published by the Tony Blair Institute for Global Change.

The MHRA told the Financial Times that based on historical vaccination data, it is expecting between 50,000 and 100,000 ADR reports for every 100 million doses delivered to patients over a six to 12 month period

The regulator also stressed that an ADR does not necessarily mean a true side effect has been observed, and AI would help spot any emerging safety signals.

The coronavirus vaccination programme would be larger than any adult campaign carried out to date, and the MHRA is concerned it could be derailed by “anti-vaccine social media activity and lobbying.”

The GenPact UK contract bypasses the usual “call for competition” tender process required by EU regulations, according to the MHRA, because if “reasons of extreme urgency”, and because it would not be feasible to “retrofit the MHRA’s legacy systems to handle the volume of ADRs that will be generated by a Covid-19 vaccine.”

The agency also says in the contract announcement that its proposed SafetyConnect programme,  for pharmacovigilance and medical device safety monitoring would not be ready by the expected coronavirus vaccine launch.

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UK patients get early access to Alnylam’s ultra-rare disease drug lumasiran

The UK’s regulator has granted early access to Alnylam’s ultra-rare disease drug lumasiran, allowing patients with primary hyperoxaluria type 1 to gain access ahead of a regulatory decision by the European Commission. 

The Medicines and Healthcare products Regulatory Agency (MHRA) gave a positive opinion for lumasiran through its Early Access to Medicines Scheme (EAMS). 

Eligible patients in the UK, many of whom are children, will be able to receive the medicine for PH1an ultra-rare disease that causes excessive oxalate production ahead of a decision by European regulators expected later this year. 

This can lead to end-stage kidney disease (ESKD) and other systemic complications. 

Affecting between one and three people per million in Europe and the US, there are around 90 patients diagnosed with PH1 in the UK. 

Current treatment approaches do not prevent oxalate overproduction and aim to lessen damage to the kidneys and delay progression to ESKD. 

PH1 patients with advanced kidney disease require dialysis to help filter waste products from their blood, until they are able and eligible to receive a dual or sequential liver/kidney transplant. 

However, long-term dialysis and post-transplant complications can severely impact patients’ quality of life. 

Lumasiran is a subcutabneously administered RNA-interference drug that blocks production of a protein called hydroxyacid oxidase, which in turn inhibits production of oxalate. 

Regulators from the FDA and European Medicines Agency are reviewing filings based on the double-blind phase 3 ILLUMINATE-A study, a six-month trial testing lumasiran in 39 patients diagnosed with PH1. 

Patients were randomised 2:1 to receive three monthly doses of lumasiran or placebo followed by quarterly maintenance doses. 

The primary endpoint was the percent change in 24-hour urinary oxalate excretion from baseline to the average of months three to six in the patients treated with lumasiran as compared to placebo.  

The MHRA made its decision based on findings of ILLUMINATE-A and other data. 

The FDA has granted a six-month Priority Review and is due to make a decision on or before an action date of 3 December. 

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