MHRA consults on draft regulatory framework for rare disease therapies

The MHRA has launched a consultation on a new regulatory framework aimed at accelerating the development and approval of therapies for rare diseases. Published on 21st May 2026, the draft framework is designed to speed up patient access to innovative treatments while maintaining safety and effectiveness standards. It forms part of wider UK efforts to improve outcomes for people living with rare conditions.

A central proposal is the introduction of an Investigational Marketing Authorisation (IMA), which would combine clinical trial and market approval processes into a single, streamlined pathway. This is intended to reduce development timelines, typically 10 to 12 years, and lower costs for bringing new therapies to market. The framework also recognises the challenges of researching rare diseases, such as small patient populations, and proposes more flexible evidence requirements. This would be supported by stronger post-market monitoring to ensure treatments remain safe and effective once approved.