Major Regulatory Overhaul for Rare Disease Treatments Announced by MHRA
The Medicines and Healthcare products Regulatory Agency (MHRA) has signaled a major reform of the UK’s regulatory framework for rare disease therapies, aiming to accelerate the journey from discovery to delivery. A new paper published on 2 November 2025 outlines plans to reform regulatory framework for rare disease therapies, with the full framework expected next year.
Currently, only 5% of rare diseases have approved treatments, despite affecting around 3.5 million people in the UK. The economic cost of delayed diagnosis and limited treatment options is estimated at £33 billion annually.
The proposed changes will tackle barriers such as small patient populations and complex evidence requirements while maintaining safety. Key ideas include:
- Single early approval covering both clinical trials and marketing authorisation, supported by real-world evidence and strict monitoring.
- Flexible pathways for highly individualised therapies, including gene-based treatments like CRISPR and mRNA.
- Improved data sharing and post-market surveillance across UK and global networks.
The reforms are backed by the Rare Disease Consortium, comprising patient groups, academia, industry, and government bodies. MHRA Executive Director Julian Beach said the UK has “the ingredients to be a global leader in rare disease therapies” and pledged bold action to speed access without compromising safety.
This initiative supports the Government’s Rare Disease Action Plan and the broader Life Sciences Strategy, positioning the UK as a hub for innovation in rare disease treatment.
Read the full paper here: Rare therapies and UK regulatory considerations – GOV.UK
Source: Major change for rare disease treatments on way, signals MHRA – GOV.UK
