Orphan Drugs

Orphan drug development has expanded with regulatory incentives across major jurisdictions. The session covers the FDA Orphan Drug Act 40 years on (over 1,200 approvals), EMA orphan designation pathway, rare-disease natural-history studies and registries, accelerated approval in rare disease, the comparison of US-EU-Japanese frameworks, and pricing/access debates. Discussion addresses rare paediatric disease priority review vouchers, the Critical Path Initiative, patient-led registries (CoRDS, NORD), Bermuda Principles for data sharing, equity in rare-disease care, and emerging n-of-1 ASO programmes for ultra-rare diseases.