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Julian Beach, the Interim Executive Director of Healthcare Quality and Access at the Medicines and Healthcare products Regulatory Agency (MHRA), has announced a major overhaul of the regulatory landscape for rare conditions. This news release signals a move toward a bespoke UK system that aims to better support the 3.5 million people in the UK living with a rare disease. The new ‘Rare Disease Pathway’ will provide an end-to-end framework, moving away from the static orphan designations used in the past.
Under the previous system, developers often faced a fragmented journey between scientific advice, orphan status application, and final marketing authorisation. The new pathway will offer ‘enhanced’ scientific advice, allowing the regulator to engage with researchers much earlier in the life of a product. Data are to be treated as part of a continuous dialogue, with the agency aiming to provide more frequent feedback to ensure trial designs meet UK requirements the first time.
The announcement marks a shift toward a more integrated approach. The MHRA plans to work alongside other parts of the health system to ensure that regulatory approval and health technology assessments happen in a more joined-up way. A formal consultation on the specific criteria and mechanics of this pathway will open in spring 2026. This window will allow stakeholders to shape how the agency defines ‘unmet need’ and how it should handle products for very small patient populations.
Source: Major change for rare disease treatments on way signals MHRA
Date: 2 November 2025
