MHRA publishes plans to reform medicines regulatory systems for rare diseases

Existing medicines regulatory processes do not consider the challenges that accompany treatment development for rare diseases. The regulatory body for medicines in the UK, the MHRA, has recognised this and is planning a major change by proposing a new framework for rare diseases. We welcome their report, published on 2nd November 2025, which outlines promising plans to reform the rare disease regulatory system by the end of 2026. 

Their ultimate aim is faster access to treatments for people with rare diseases. The report highlights the need for a patient-focused, pragmatic, and flexible regulatory framework tailored to rare disease treatment development. 

To support scientific development, the MHRA acknowledges the difficulties that come with undertaking research and clinical trials for rare diseases, such as small patient cohorts and less background knowledge. To adapt to this fact the MHRA will allow more flexibility in the way that evidence is gathered, such as through N-of-1 trials; natural history and real-world data could be acceptable clinical trial controls; and innovative trial methodologies will be considered, such as with artificial intelligence (AI) and machine learning models. To balance this, they describe robust plans to make sure that once a treatment is approved, sponsors must follow up and collect data to ensure that effects and safety of treatments are continuously monitored. 

The report acknowledges that the current regulatory process is long and multi-stage, which presents various opportunities for delay along the way. The MHRA suggests running regulatory and health technology assessment processes simultaneously, which they hope would help to remove one of the significant barriers to accessing treatments. The new model may allow earlier access to treatment through conditional and stepwise approvals providing there are iterative reviews in place and ongoing collection of data on safety and efficacy throughout the process. 

Ataxia UK is very supportive of the MHRA’s plans to include patients and patient representatives throughout the lifecycle of rare disease therapy development, such as in clinical trial design and regulatory processes.

There are also ambitious plans to develop National and Global Rare Disease Registries which could provide a resource for collection of valuable and scarce real-time data which can be shared internationally to boost evidence bases. This is especially important for rare diseases where data is limited due to small patient numbers. 

Ataxia UK will be contributing to the consultation phase of this project set to begin in early 2026, by representing the views of people with ataxia.  

To read the report please click here.