Euro Roundup: ABPI calls for UK regulatory actions to improve access to cell and gene therapies

The Association of the British Pharmaceutical Industry (ABPI) has published recommendations on how the UK can improve access to and uptake of cell and gene therapies. The trade group based the advice on its assessment of the systems and pathways that other regions have established to handle advanced therapy medicinal products (ATMPs).

The report said the UK has shown early leadership in ATMP research, manufacture, and patient access but now faces a new set of challenges. ABPI estimates the number of ATMPs approved annually could rise from around two today to 10 to 15 by 2030. The growth could be associated with increased diversity of the therapies, notably an expansion of the targeted indications to include more common diseases.

“Despite the UK’s current strengths in ATMPs, there is much more to do to ensure that patients here will be able to access the number and variety of ATMPs expected to become available in the coming years,” ABPI said. “Based on the learnings from other countries, several important actions are needed for the UK to build on its current momentum to improve the future readiness of the ecosystem.”

The trade group is recommending several UK agencies “work together to establish a single national platform for collecting data on ATMP treatment outcomes that could support innovative access models.” ABPI envisages NHS England, the Medicines and Healthcare products Regulatory Agency (MHRA), and the National Institute for Health and Care Excellence (NICE) collaborating on the project.

The recommendation reflects ABPI’s belief that “improved capabilities for data collection will be essential if the UK is to maintain its position as a priority launch market” in the long term. The nature of ATMPs mean there “will inevitably be questions on whether the long-term data supports the continued value” of the therapies, the trade group said.

“One solution would be the development of a single national platform, analogous to the AIFA registries of Italy and the Spanish Valtermed system, which enable routine use of outcomes-based agreements. This would ensure mandatory long-term data collection into patient registries and may also require appropriate resourcing of healthcare personnel for data management,” ABPI said.

The trade group is also pushing for a “strengthened and refreshed Innovative Licensing and Access Pathway.” ABPI wants the UK to establish “a streamlined end-to-end pathway for ATMPs that helps align all system partners, from development to patient access.” As part of that proposal, ABPI is calling for MHRA to ensure it has the expertise and capacity to support timely scientific advice and approvals.

Another set of recommendations center on NICE. ABPI wants the cost watchdog to use “the existing flexibility to apply the 1.5% discount rate for ATMPs to ensure its appraisal process does not disadvantage future innovative treatments with long-term benefits, including many ATMPs.” The request reflects concerns that “several ATMPs will never be appropriately valued due to a discount rate of 3.5% that does not reflect the long-term benefits of treatment with these therapy types.”

Other NICE-focused recommendations include calls for the UK to work with industry to “accelerate the implementation of a Rapid Entry to Managed Access process by using ATMPs to pilot new approaches” and to adopt a more flexible approach to evidence requirements.

Press Release

EMA answers questions on impact of mutual recognition agreement with the US

The European Medicines Agency (EMA) has published a question-and-answer document about the impact of the mutual recognition agreement between the European Union and United States.

An agreement on the mutual recognition of good manufacturing practice inspections took effect in 2017. The pact was initially limited to human medicines, but the EU began recognizing the US as equivalent for veterinary medicines in 2023. The Q&A lists the dates on which the US confirmed the capability of EU countries for inspections of both types of medicines. Confirmation of veterinary capabilities is ongoing.

The agreements provide opportunities for further expansion. The EU and US are initially focused on the acceptance of inspections conducted within their respective territories, but they have the option to rely on GMP certificates issued for overseas manufacturing facilities. EMA said the provision to accept the outcome of inspections conducted outside the US or EU is not yet operational.

An expansion of the products covered by the agreements is possible too. The current agreement covers human drugs, biological products and veterinary medicines. Human blood, plasma, tissues, and organs are excluded, as are veterinary immunological products.

“At a later stage, and after thorough assessment, the Joint Sectoral Committee has the option to extend the operational scope gradually to human vaccines and plasma-derived products and investigational medicinal products,” EMA said.

EMA Q&A

EMA updates CTIS training program documents on transitioning, managing trials

EMA has answered additional questions about managing clinical trials and transitioning studies to its Clinical Trial Information System (CTIS). The agency added the responses to CTIS training materials.

Officials added three questions to a training module on transitional trials, EMA’s term for studies that were authorized under the Clinical Trials Directive through EudraCT and need to be moved to CTIS. One new response explains sponsors that submit a study without selecting the “transition trial” checkbox will need to withdraw and resubmit the filing.

Another response clarifies that users can create organizations in CTIS, but they will not be validated by EMA. Organizations registered through OMS, the recommended but not mandatory approach, adhere to a data quality standard, “reducing the likelihood of receiving RFIs from assessing authorities regarding inaccurate data,” EMA said. The third response states investigational medicinal product registration is mandatory. Sponsors will be unable to populate the form before registering the product in XEVMPD.

The agency also added one question to a training module on managing clinical trials. EMA’s response explains that users can populate the “anticipated summary of results” date when the status of a clinical trial changes to “revoked” in response to the application of a corrective measure.

EMA FAQ, More

MHRA calls for contributors to study of direct-acting oral anticoagulant side effects

MHRA is seeking contributors to a pilot study that is exploring whether genetics influence certain side effects associated with direct-acting oral anticoagulants (DOACs) and the gout drug allopurinol.

The pilot study is part of the Yellow Card Biobank, a collaboration that MHRA set up with Genomics England. Through the collaboration, MHRA aims to collect samples on people who have experienced side effects to understand if there is a genetic basis for the adverse events. The work is underpinned by data suggesting genetic testing could prevent almost one-third of adverse reactions.

MHRA and Genomics England are initially focusing on links between DOACs and severe bleeding and between allopurinol and severe skin reactions. The latest update on the project is a call for people to contribute to the study. MHRA made the request for contributors in its monthly drug safety update.

The UK regulatory agency may ask physicians who submit adverse event reports about the side effects to check if their patients are willing to participate in the study. Patients who consent will be asked to provide blood samples for genetic analysis. MHRA may also contact physicians who have submitted side effect reports in the past to discuss the cases. The agency will contact patients who file reports directly.

Press Release

Euro Roundup: ABPI calls for UK regulatory actions to improve access to cell and gene therapies

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