Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases

Key messagesIn 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 2000The 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 unclearThe orphan drug status has knock-on effects on national policiesEU and national orphan drug regulations need reform: granting superiority labels only on the basis of robust evidence would incentivise evidence generation, lower orphan drug prices and ultimately improve outcomes in patients with rare diseases About 30 million people in the European Union (EU) are affected by over 6000 different rare diseases,1 making the lack of available and accessible treatments for rare diseases a major public health issue. The development of drugs for rare diseases (orphan drugs) used to be commercially…
Read Original Article: Reforming EU and national orphan drug regulations to improve outcomes for patients with rare diseases »