Euro Roundup: MHRA commits to major reform of rare disease therapy regulation
The Medicines and Healthcare products Regulatory Agency (MHRA) has outlined plans for major reforms to streamline the process of testing, developing, and launching drugs for rare diseases in the UK.The overarching goal is to help deliver treatments for the 95% of rare diseases that currently lack approved therapies. In doing so, the UK wants to reduce the number of patients suffering from delayed diagnosis and limited treatment options. The UK estimates that shortcomings of the current system contribute to £4.7 billion ($6.1 billion) in health-related disability costs.
The MHRA has identified the rigidity of conventional regulatory pathways as a barrier to developing drugs for rare diseases. In response, the agency proposes to make evidence requirements more flexible, introduce new licensing structures, and rely on post-market surveillance to address knowledge gaps.
Officials are assessing how real-world data, in-silico trials, and artificial intelligence and machine learning (AI/ML) models could be used to generate evidence for regulatory decision-making. The assessments are part of a plan to develop guidance on how companies can use prior quality, clinical, non-clinical, and statistical knowledge to support regulatory submissions. Platform technologies could benefit from the guidance.
Companies will be able to use prior knowledge to support new types of approvals. MHRA is looking into investigative licensing pathways that would grant preliminary approval “based on appropriate, albeit limited, evidence such as mechanism hypothesis, with iterative reassessment throughout the process.” Companies could use modular data packages and iterative checkpoints to work toward full approval.
Post-market surveillance is central to MHRA’s plans to enable companies to win provisional approval based on limited evidence. Rather than expect companies to have comprehensive data pre-approval, MHRA will rely on national rare disease registries, risk management plans, and international data sharing to ensure the safety and long-term effectiveness of rare disease medicines.
“Key priorities are ensuring registries are fit for purpose, achieving international harmonization, and balancing the data collection burden with the needs of patients and the healthcare system,” MHRA said. “Real-world evidence, patient follow-up, and leveraging existing healthcare systems for long-term monitoring are also identified as priorities.”
MHRA aims to have drafted the framework by Spring 2026 and conduct an external review in the first half of the year. Potential legislative or further development steps may occur during this period. The agency is also planning a public consultation in 2026.
Press Release, Policy Paper
European Commission posts batch of new and updated clinical trial guidelines
The European Commission has published new guidelines on selecting the lead Member State for assessing clinical trial applications. Officials posted the new guidelines alongside updates to three existing documents.
Under the Clinical Trials Regulation, sponsors file a single application for all European Union countries involved in a study and choose one Member State to lead the scientific and technical assessment of the application. However, current data from the Clinical Trials Information System (CTIS) shows that around 30% of proposed lead Member States decline the role.
The guideline explains what happens when a Member State declines. If one Member State volunteers, they will be selected. If multiple Member States volunteer, the country with the lowest “workshare” will be selected. Workshare is based on how often a country takes the lead on assessments. If no Member States volunteer, CTIS will automatically assign the project to the country with the lowest workshare.
The Commission recommends that sponsors contact their preferred national regulatory body before filing a submission to ensure that the agency accepts their reporting Member State's requests. Officials said pre-submission contact is particularly important for complex trials that reference documentation in another ongoing study.
The updated guidelines cover decentralized elements, auxiliary medicinal products, and issues that are frequently identified during the two parts of clinical trial application assessments.
Commission Notice
Trade groups warn PFAS restriction review ‘risks disregarding critical evidence’
Nine healthcare associations have voiced serious concerns about how the European Chemicals Agency (ECHA) plans to assess the impact of PFAS restrictions.
Most PFAS persist in the environment. Concerns about the impact of persistent PFAS has led ECHA to propose restrictions on the class of synthetic chemicals. In August, ECHA said five countries had carried out assessments of eight sectors, including “other medical applications” such as immediate packaging and excipients for pharmaceuticals, that were not named in the initial restriction proposal.
Last week, EFPIA, Medicines for Europe, and other trade groups published a joint statement to express concerns about ECHA’s decision not to individually assess “other medical applications” as committees on risk and socio-economic impacts prepare their final opinions.
“Excluding medicines from individual assessment, while keeping them within the overall scope of the restriction, prevents the European Commission from obtaining a full understanding of the public and animal health and socio-economic impacts,” the trade groups said. “Sector-specific evidence and emission mitigation measures have not been adequately reflected in the current analysis.”
Press Release
MHRA starts winding down tool for seeing and tracking regulatory assessments
MHRA has decided to wind down its RegulatoryConnect program after assessing how much it will cost to complete the system.
The UK regulatory agency pitched RegulatoryConnect as a way to make it easier for industry to see and track their regulatory assessments when it shared guidance on the project last year. MHRA envisaged users signing into the portal to track the progress of applications and see live license details. The agency planned to add the ability to file applications and variations through the service later.
MHRA abandoned plans to develop the system this week. Officials decided to wind RegulatoryConnect down over the coming months after a comprehensive review of the platform, its costs, and the agency’s strategy.
“The program no longer offers value for money for UK taxpayers and the cost to complete the program is too high for a solution which does not now enable us to deliver the aspirations of the agency,” MHRA said. “The functionality to track applications and view live authorization details through the existing portal will remain available.”
MHRA is planning a replacement that aligns with the strategy it intends to publish next year. The agency will revisit its technological needs in light of the strategy, with a particular focus on risk-proportionate regulation, data, and AI. MHRA plans to share further updates in the coming weeks.
MHRA Update
Other news
MedTech Europe has led a call for the European Commission to address digital labels in its revisions of the medical device and in vitro diagnostic regulations. The trade group wants officials to allow medtech companies to provide information about the importer and authorized representative via a digital label. The proposal, which was backed by AESGP, COCIR, and Euromcontact, reflects a belief that physical labels should focus on information needed for the safe use of the device. Press Release
MHRA has appointed Jacob George its first chief medical and scientific officer. George is a professor of cardiovascular medicine and therapeutics at the University of Dundee Medical School and a consultant physician. The agency said it has created the role to “drive scientific excellence and shape the future of regulation through the MHRA science strategy.” George will join MHRA in January. Press Release
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