The MHRA has published a position paper which outlines the current approach by UK regulators and partners to design a forward-looking framework that meets the needs of patients with rare diseases, while ensuring robust evidence generation
- The UK is advancing a comprehensive framework to ensure rare disease therapies are developed, licensed, and delivered in a way that balances patient needs, regulatory robustness, and system sustainability.
- Rare therapies are defined as medicinal products intended to treat rare diseases – specifically, conditions with a prevalence of no more than 5 in 10,000, where significant barriers exist to conducting standard clinical development programmes or regulatory approvals
- Through international collaboration, strong patient engagement, and close alignment with the NHS, this initiative will position the UK as a leader in rare therapy regulation and patient access.
- A draft of the framework is anticipated to be available by Spring 2026, followed by external review in the first half of 2026.
- Potential legislative or further development steps may also occur during this period.
- 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.
- In developing this new pathway, the MHRA will ensure that safety standards are not weakened; instead, the balance of evidence generation will be adjusted.
- By combining flexible pre-approval requirements with robust post-approval monitoring, the UK can provide patients with rare diseases faster access to therapies without compromising safety or scientific integrity.
Furter detailed information is available at the link below.
Source: MHRA