Who We Are
The FDA Office of Orphan Products Development (OOPD) supports and advances the development and evaluation of new treatments for rare diseases. OOPD evaluates information from product sponsors to determine if drugs, biologics or medical devices meet the criteria for certain incentives and administers grants to provide funding for research on rare diseases. The office also works on rare disease issues with medical and research communities, professional organizations, academia, government agencies, industry and rare disease patient groups.
What We Do
- Work with sponsors to determine if their products meet the criteria for certain categories (e.g., orphan drug, rare pediatric disease drug or humanitarian use device designations).
- Provide orphan status to drugs and biologics which are intended to treat, diagnose or prevent rare diseases that affect fewer than 200,000 people in the U.S.
- Designate medical devices that intend to benefit patients in treating or diagnosing a disease or condition that affects fewer than 8,000 individuals in the U.S. per year.
- Award priority review vouchers to sponsors of rare pediatric disease products that meet certain criteria. For example, a sponsor who receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
- Award grants to provide funding for clinical trials and natural history studies that advance rare disease medical product development.
- Award grants that provide funding to develop nonprofit consortia to facilitate pediatric medical device development.
Office of Orphan Products Development
Office of Clinical Policy and Programs
U.S. Food and Drug Administration
10903 New Hampshire Ave. WO-32, Room 5295
Silver Spring, MD 20993
- Designating an Orphan Product: Drugs and Biological Products
- Humanitarian use device program
- Rare pediatric disease program
- Orphan products grants program
- Pediatric device consortia grants program