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European Alliance for Personalised Medicine

Clinical trials, HTA, Orphan and paediatric




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Good afternoon, and welcome to the European Alliance for Personalised Medicine (EAPM) update – with a real shake-up in UK politics dominating the headlines, EAPM turns its thoughts as to what this may mean in the health arena as well as developments in EU policy, writes EAPM Executive Director Dr. Denis Horgan.

HTA approval 

Health technology assessment experts are closing in on the methodology to be used for EU-wide assessments. Most recently, they have delivered a position on how to choose endpoints for clinical trials of a new therapy. Morning Health Care caught up with the team at EUCOPE, Europe’s biotech entrepreneurs’ lobby, to find out their thoughts on this latest draft.

Deciding what to measure when testing a new therapy is no small decision. Should it be a measure of symptoms, or the absence of care needs? Should it be a medical biomarker, or a patient survey score? And when should these things be measured? Defining the outcome of a trial not only impacts how a regulator will consider how well a product works versus the risks, but also whether it can demonstrate that it’s adding benefit to the existing armory of care.

The clinical trials outcomes (endpoints) draft guide sets out when to opt for one outcome over another and who should report it. For example, they can be reported by a physician or with help of a diagnostic, or they can be reported by patients themselves.

Parties have until November 1 to comment on the draft. It’s also the final paper to feed into the methodology for EU-wide HTA. EUnetHTA is expected to compile the finalized methodology toward the end of the year.

Commission mulls changes to rules for rare disease drugs

With weeks to go before the Commission publishes its legislative proposals, members of the parliament’s ITRE committee are divided over how to secure the future of Europe’s most research intensive sector


With the legislative proposals due to be published before the end of the year, the European Parliament rapporteur remains at odds with fellow members of the industry and research committee, ITRE, over the direction of the new EU pharmaceutical strategy.

At issue is a fundamental divide between MEPs who believe that given proper incentives, pharma companies will invest in R&D, promoting their global competitiveness and benefitting Europe as a whole, and MEPs who believe bigger profits are more likely to go to the shareholders than into research.

A paper that EAPM published on this issue is available here: Making Sure That Orphan Incentives Tip the Right Way in Europe.

Orphan Drug review

Under the Commission’s current rules, drugs that treat rare diseases benefit from a decade of market exclusivity. That’s two more years than the eight years given to other innovative medicines. The added bonus is meant to incentivize the development of medicines that can only be used to treat a small patient population.

WIth the upcoming revision, it’s important to understand how the terms of classification criteria influence the ongoing development of a drug. In relation to prevalence, it’s mandatory within the EU to base this on the whole community, and not just to use data from a couple of countries. Prevalence is not something that can be ‘fudged’ and is reviewed in collaboration with expert advice that draws on current evidence.

As understanding of the disease and information about the orphan drug becomes available, clinicians are better informed and more likely to provide a diagnosis for the condition. It’s also possible for the condition to be recognised as a subset of a more prevalent disease that removes the orphan status in the EU. Any calculation of prevalence should reference the EMA guidance.

A medical condition might seem like an obvious term but, within the context of eligibility as an orphan drug, it should be distinct with a specific pathophysiological profile and clinical prognosis. An essential consideration for EU orphan drug designation is not being a subset of a more prevalent condition.

Paediatric medical devices at risk

A regulation which came into effect in May last year with the aim of improving the oversight of medical devices may put some surgeries for children and the treatment of rare diseases at risk, new research has revealed.

The study from Trinity College Dublin, published in Pediatric Cardiology, pointed out that medical devices include a great diversity of technologies, which are evaluated and approved in the European Union (EU) according to a revised law that came into effect on May 26, 2021 known as the Medical Device Regulation or MDR (EU 745/2017).

It has a transition period that allows products that were approved under the previous rules to continue to be marketed until May 26, 2024 at the latest.

As a result of a series of unforeseen factors, there is a possibility that the MDR may result in products becoming unavailable, with the consequent risk of a loss of some interventions that are reliant upon those

Tom Melvin, associate professor of medical device regulatory affairs at Trinity College’s school of medicine, said: “The Medical Device Regulation came into effect in May 2021 with the aim of replacing the previous rules in place since the 1990’s and improving the safety of medical devices, in addition to supporting the introduction of innovative technologies.”

EU Parliament and Council sign Digital Services Act

The vote approving the DSA by the Council of Ministers on Tuesday follows the earlier approval of the legislation by MEPs. The new laws (312-page / 686 KB PDF so a lot of reading..) set out extensive requirements for online intermediaries over the way they moderate content posted, and police the goods and services traded, on their platforms. The DSA will only come into force 20 days after its publication in the Official Journal of the EU. 

No date has yet been set for publication. The date of 19 October was set for the president of the European Parliament and the president of the Council to sign the DSA. Publication in the OJEU would follow on a subsequent date.

Out-Law anticipates that the DSA will come into force sometime in mid-November. 

The precise date will be of particular interest to online platforms because, while most of the provisions of the DSA will not take effect until the DSA has been in force for 15 months, some of the new rules will have effect immediately – including reporting obligations that will have a bearing on whether those platforms are considered ‘very large online platforms’ and therefore subject to the most stringent requirements of the DSA or not. There is a three-month deadline set for compliance with the disclosure duties.

COVID vaccine prices forecast to rise

In the pandemic's second year, Pfizer and Moderna's mRNA vaccines largely reign supreme. In new European purchase agreements, the companies are charging more for their doses.

Pfizer and Moderna have both raised the price of their mRNA-based COVID-19 vaccines in Europe, The Financial Times first reported. Pfizer’s shot will cost €19.50 ($23.15) per dose under a new supply deal, while Moderna will charge $25.50 per dose in its own agreement, according to documents seen by the newspaper.

UK clinical trials collapse

Patient care, the NHS, and economic growth are all missing out as a result of a collapse in the number of UK industry clinical trials, according to the latest annual report on clinical research from the Association of the British Pharmaceutical Industry (ABPI). 

The COVID-19 pandemic has accelerated the decline in late-stage industry clinical research in the UK, compared to its global peers. This should ring alarm bells in the NHS and in Whitehall as health leaders and policymakers look to improve patient care and deliver long-term economic growth. 

The report ‘Rescuing Patient Access to Industry Clinical Trials in the UK’ shows that the number of industry clinical trials initiated in the UK per year fell by 41% between 2017 and 2021, with cancer trials falling by the same margin.

The report also shows that between 2017 – 2021:

  • The number of Phase III industry trials initiated in the UK – those with medicines closest to market – fell by 48% between 2017 and 2021
  • The UK has fallen down the global rankings for late-stage clinical research, dropping from 2nd to 6th in Phase II trials and 4th to 10th in Phase III trials between 2017 and 2021 
  • Patient access to industry clinical trials on the National Institute for Health and Care Research Clinical Research Network (NIHR CRN) fell from 50,112 to 28,193 between 2017/18 and 2021/22 – a 44% drop.

These findings point to a clear and serious threat to the long-term future of industry clinical research in the UK – and the benefits it brings to patients, the NHS, and the UK economy.

Health and care sectors in crisis in UK

Political turmoil has dominated the U.K. news these past weeks. A gridlocked health and care system is leading to a deterioration in people’s access to and experience of care according to the Care Quality Commission’s (CQC’s) annual assessment of the state of health and social care in England over the past year.

This year – based on CQC’s inspection activity, information received from the public and those who deliver care alongside other evidence – the assessment is that the health and care system is gridlocked and unable to operate effectively.

Without action now, staff retention will continue to decline across health and care, increasing pressure across the system and leading to worse outcomes for people. Services will be further stretched, and people will be at greater risk of harm as staff try to deal with the consequences of a lack of access to community services, including adult social care. This will be especially visible in areas of higher economic deprivation where access to care outside hospitals is most under pressure. In addition to the increased risk of harm to people, more people will be forced out of the labour market either through ill health or because they are supporting family members who need care.

And that is all for now from EAPM – stay safe and well, and enjoy your weekend.

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