Developing Products for Rare Diseases & Conditions
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. The office also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.
OOPD provides incentives for sponsors to develop products for rare diseases. The program has successfully enabled the development and marketing of over 600 drugs and biologic products for rare diseases since 1983. In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983. The Orphan Grants Program has been used to bring more than 60 products to marketing approval. The Humanitarian Use Device Program has been the first step in approval of 70 Humanitarian Device Exemption approvals.
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.
The Rare Pediatric Disease Priority Review Voucher Program says that 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.
The Humanitarian Use Device (HUD) program designates medical devices that are intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year as eligible for Humanitarian Device Exemption.
The OOPD administers three extramural grant programs: The Orphan Products Clinical Trials Grants Program provides funding for clinical research that tests the safety and efficacy of drugs, biologics, medical devices and medical foods in rare diseases or conditions; The Natural History Grants Program which supports studies that advance rare disease medical products development through characterization of the natural history of rare diseases and conditions; The Pediatric Device Consortia (PDC) Grants Program provides funding to develop nonprofit consortia to facilitate pediatric medical device development.
For more information on any of our programs, please visit the programs’ web pages.
Please note our mailing address, telephone number, FAX number and email address:
Office of Orphan Products Development
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002
Main Telephone Number: 301-796-8660
Fax Number: 301-847-8621
- Guidance for Industry Clarification of Orphan Designation of Drugs and Biologics for Pediatrics
- Rare Diseases: Common Issues in Drug Development Guidance for Industry
- Interpreting Sameness of Monoclonal Antibody Products Under the Orphan Drug Regulations
- Guidance for Industry, Researchers, Patient Groups and FDA Staff on Meetings with OOPD
- Guidance for Industry and FDA Staff - Humanitarian Use Device (HUD) Designations
- Search Orphan Drug Designations and Approvals
- IND Application Information and Procedures
- Searchable Database of Research Grants Funded by OOPD
- Listing of CDRH Humanitarian Device Exemptions
- Meeting with the Office of Orphan Products Development (OOPD)
- Educational Resources for Patients, Advocacy Groups, Research Investigators and Drug Developers
- FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
- FDA awards five grants to advance the development of pediatric medical devices
- FDA Orphan Drug Designation Request Form
- Tutorial: Recommended Tips for Creating an Orphan Drug Designation Application
- Orphan Drug Modernization Plan and Updates