UK’s MHRA outlines new patient-focused regulatory approach for rare disease treatments

With around 3.5 million people in the UK affected by rare diseases, the MHRA has unveiled plans to create a patient-focused framework aimed at accelerating access to innovative therapies. The proposals seek to balance rapid development with robust safety and evidence requirements.

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has announced the publication of a new position paper setting out its intentions to reform the regulatory framework for rare disease therapies, signaling that “significant change for rare disease patients is on its way”. The paper, published on November 2, 2025, supports the Government’s life sciences strategy and outlines a “bold new rulebook” for rare therapies expected to be published next year.

Drawing on experience gained through its past involvement with the European Medicines Agency and newer initiatives like the Innovative Licensing and Access Pathway, the MHRA aims to build on this foundation to position the UK at the forefront of rare disease regulation and innovation. The agency says the new framework will balance “robust evidence generation, system sustainability, and international convergence.”

The position paper acknowledges the limitations of existing regulatory pathways and the need to address the distinct challenges associated with developing treatments for rare diseases. These include small patient populations, limited natural history data, and the increasing complexity of advanced therapies such as gene- and cell-based treatments. Traditional frameworks, built around large, randomized trials with validated endpoints and multiple confirmatory phases, are often impractical in these settings. As the paper notes, “Rare disease therapies… need flexibility, adaptable trial designs, real-world evidence, and expedited review – needs that existing frameworks do not adequately support.”

Central to the MHRA’s reform agenda is the creation of a more flexible, lifecycle-focused regulatory model. The agency is developing a proportionate licensing and registration system designed to bridge clinical trials and marketing authorizations. This approach would enable earlier investigative approvals, ongoing evidence reassessment, and structured discussions on endpoints, data sources and lifecycle management from the outset of product development.

Post-market surveillance will also play a central role. The paper highlights plans to make greater use of evolving approaches to data generation, registries and proportional pharmacovigilance for rare products. Priorities include ensuring registries are fit-for-purpose, promoting international harmonization, and balancing the data-collection burden with the needs of patients and the healthcare system. The MHRA also outlines ambitions to align, where possible, post-marketing evidence requirements for both regulatory and health technology assessment (HTA) purposes, including through managed access agreements and biomarker-based approvals.

Speaking to The Times, Lawrence Tallon Chief Executive of the MHRA, highlighted the opportunity to use the NHS’s extensive longitudinal datasets to support these aims. He noted that leveraging existing patient data could reduce the need to start new trials from scratch by helping researchers understand how patients fare without treatment over time. “The real world is your placebo,” Tallon said, adding that this represents a particular advantage of the NHS compared with other countries.

Julian Beach, MHRA Executive Director for Healthcare Quality and Access, said: “The UK has the ingredients to be a global leader in rare disease therapies… the challenge is bringing all these elements together, which our new framework will do.” Beach added: “I hope this paper reassures all those affected by rare disease that we are listening and are prepared to take bold action to speed the path from discovery to delivery, while maintaining strict standards of safety.”

A key feature of the proposed model is the active involvement of patients and patient representatives in regulatory decision-making. The paper emphasizes that patient experience will inform benefit-risk assessments, evidence-generation strategies, and ongoing license management.

The reform effort is being supported by a newly established Rare Disease Consortium, uniting patient groups, academic experts, regulators, and industry partners.

Consortium members welcomed the initiative. Nick Meade, Chief Executive of Genetic Alliance UK, described the program as “a vital step towards changing” prospects for families affected by rare conditions. Dr Jacqueline Barry, Chief Clinical Officer at the Cell and Gene Therapy Catapult, said it is “essential to ensure that patients with the greatest unmet need can access innovative treatments without unnecessary delay.”

A draft of the framework is expected in the first quarter of 2026, with external review and public consultation to follow before final implementation.