The Medicines and Healthcare products Regulatory Agency (MHRA) published on 2 November 2025, a Policy Paper outlining the Agency’s intentions to improve regulatory pathways for rare disease therapies to be tested, manufactured and approved in the UK.
Our insight on the changes can be found below. If you require more detailed insight on the MHRA and how these changes will impact your business, please contact Brevia Health (+44 (0)20 7091 1655 or email contact@breviahealth.com).
Rare diseases in the UK
Around 3.5 million people in the UK live with a rare disease, yet fewer than 5 per cent of rare diseases currently have an approved treatment. The average diagnostic journey takes 5.6 years, and 30 per cent of affected children die before the age of five.
Currently, each new rare therapy must follow a separate regulatory pathway, requiring approval to initiate clinical trials and subsequent authorisation for marketing in the UK.
In the Policy Paper, the MHRA recognise that this approach is costly and inefficient, making it unviable to develop multiple highly targeted treatments.
What will be in the new rare disease rulebook?
A new rulebook for rare therapies is being written and is due to be published next year.
A key focus of the new regulatory framework will be to address the unique barriers that currently prevent life-changing rare disease therapies from reaching patients. Key barriers include small patient populations, limited understanding of the underlying conditions, and challenges in patient recruitment and clinical trial delivery.
The MHRA has also announced the newly formed Rare Disease Consortium, which includes patients and their representatives, academics and industry.
Key features of the proposed new framework include:
- Defining scope and entry criteria for a new regulatory pathway for rare diseases.
- Leveraging prior knowledge to meet the evidence requirement for new treatments.
- Developing flexible, proportionate licensing and registration model tailored to the unique challenges of rare diseases.
- Support safety and long-term effectiveness of new therapies by appropriate post-market surveillance.
- Embedding patient expertise and experience into regulatory development sessions and licensing deliberations.
- Work with global regulatory partners to support scientific development in collaboration with academia, NHS providers and industry to accelerate development.
- Work with UK system partners to build on the UK Government’s 10 Year Health Plan for cross-system collaboration.
When will the new framework be published?
A draft framework is anticipated to be available by Spring 2026, followed by external review in the first half of 2026.
According to MHRA, a public consultation will be conducted in 2026 to ensure all views are considered, maintaining patient interests and safety whilst supporting innovation of critical therapies.
Support from Brevia Health
If you require more insight on the work of the MHRA and these changes, please contact Brevia Health (+44 (0)20 7091 1655 or email contact@breviahealth.com).
