The Medicines & Healthcare products Regulatory Agency (MHRA) has published a new position paper entitled ‘Rare therapies and UK regulatory considerations’ and has plans to introduce a major reform for the rare therapies landscape.
Also in development is a rulebook that will form a new regulatory framework on how rare therapies should be planned and managed in the UK. This will be published in 2026.
With more than 3.5 million people in the UK living with a rare disease, and 30% of children with a rare disease dying before the age of five, there is an urgent need to meet demand and improve the patient experience. Unfortunately, diagnostic processes take on average 5.6 years and, with delayed diagnostics and limited treatment options costing approximately £340 million per year, the financial impact is enormous.
The MHRA is setting out ways to address these issues in the new reform and the ‘Rare diseases and UK regulatory considerations’ paper discusses the following:
- The importance of stronger post-market surveillance, including pharmacovigilance and data generation
- Aims to improve patient engagement and build ‘trust and transparency’
- International convergence and the alignment of regulatory pathways
- Early UK health system planning between the MHRA, healthcare partners and other stakeholders
With this new reform, the MHRA aims to ‘provide patients with rare diseases faster access to therapies without compromising safety or scientific integrity.’
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