An orphan drug is a medication developed to treat rare diseases (also called orphan diseases) that affect a small percentage of the population. Because these diseases are rare, developing drugs for them is often not commercially viable without special incentives.
Key Features of Orphan Drugs:
- Target Rare Diseases – Typically for conditions affecting fewer than 5 in 10,000 people in the EU or fewer than 200,000 people in the U.S.
- Regulatory Incentives – Governments provide benefits to encourage orphan drug development, such as:
- Tax credits for clinical trials
- Market exclusivity (10 years in the EU, 7 years in the U.S.)
- Waived or reduced regulatory fees
- Grants and fast-track approval pathways
- Examples of Orphan Diseases – Cystic fibrosis, Duchenne muscular dystrophy, Huntington’s disease, and some rare cancers.
- Approval Process – Orphan drug designation is granted by:
- European Medicines Agency (EMA) – Committee for Orphan Medicinal Products (COMP)
- U.S. FDA – Office of Orphan Products Development (OOPD)
Important of Orphan drugs:
Orphan drugs provide life-saving treatments for patients with rare diseases who might otherwise have no medical options. Incentive programs help make drug development for these conditions financially feasible for pharmaceutical companies.
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