Posted by Michael Wonder on 10 May 2023
Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases
9 May 2023 - In Europe, the number of approvals for new drugs targeting rare diseases (orphan drugs) has risen sharply since the introduction of the EU Orphan Regulation in 2000.
The regulation couples orphan drug status with a label of superior therapeutic benefit at approval in the EU without requiring robust evidence even though the real therapeutic benefit of orphan drugs is often unclear.
