Hot topic: The UK pivots to rare diseases – why this week’s MHRA move matters
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has just announced plans for a complete regulatory overhaul of how rare disease therapies are to be assessed and approved.
Launched last Sunday (November 2), the proposed framework could shave years off approval timelines.
The stakes are high. One in 17 Britons lives with a rare disease – 3.5 million people across 7,000 conditions. Globally, that translates to more than 300 million people. Currently, there are no approved treatments for 95% of such conditions.
Why is the MHRA taking this action? The strategic context matters. Of course, it’s partly about helping people with rare diseases get quicker access to effective new medicines. But it’s also about helping another patient in need: UK PLC.
According to the Association of the British Pharmaceutical Industry (ABPI), the number of clinical trials initiated in the UK fell by 41% between 2017 and 2021. ABPI data also point to life sciences foreign investment falling 58% between 2017 and 2023, resulting in the UK dropping from second to seventh place globally. The rare disease reforms are a deliberate play to reverse the exodus – and get the UK’s life science sector growing again.
The mechanisms are bold: opening the door for NHS real-world data to replace placebo controlled trials, for digital twins – computer-modelled patients – to replace traditional control groups, for approvals to be based on interim results rather than having to wait for full data, and for single authorisations to cover both the clinical trial and early patient use, so if it shows benefit, full approval can follow quickly.
There’s a critical question though – will this new fast-track approach weaken safety? The MHRA insists it will not. The trade-off is deliberate: drugs can be approved on limited but compelling evidence, but only with mandatory strict safety monitoring and ongoing reviews of real-world data. According to the MHRA’s policy paper, approvals will be “granted with a strict safety monitoring plan” and national registries will track patient outcomes over time. Julian Beach, MHRA Executive Director, has committed the agency to “maintaining strict standards of safety.”
For the many companies around the world developing rare disease therapies, the UK could soon become considerably more interesting. Flexibility around evidence generation could compress timelines and de-risk UK-first development programmes.
Consultation on the new framework is set to begin in early 2026, with detailed guidance due to land later in the year.
The bet: trade traditional, extremely stringent evidence thresholds for speed, but double-down on safety monitoring that starts during the trial and continues after approval. For millions of people with rare diseases, it could be transformative. For biotech and pharma, it signals the UK is open for business.
