UK's New Flexible Licensing for Orphan Medicines: Revolutionizing Rare Disease Treatments

Описание: UK orphan medicines licensing
MHRA rare diseases framework
Discover how the UK's Medicines and Healthcare products Regulatory Agency (MHRA) is leading the way with an innovative adaptable licensing regime for orphan medicines. This pioneering approach aims to speed up access to personalized treatments for patients with rare diseases, affecting fewer than 5 in 10,000 people.
In this video, we break down the key changes: preliminary approvals based on limited evidence, iterative reassessments, real-world data instead of large trials, and global rare disease registries for better data pooling. Learn about the timeline, with a draft framework expected by spring 2026, and how this could position the UK as a global test bed for rare therapies.
We also explore challenges, including alignment with NICE for health technology assessments and pricing issues for the NHS. Experts from Pinsent Masons highlight the need for incentives like extended protection certificates to attract manufacturers.
If you're interested in rare diseases, pharmaceutical innovation, or UK healthcare policy, this video explains it all. Stay tuned for updates on orphan drug development and regulatory reforms.