The FDA Office of Orphan Products Development (OOPD) was created to advance the development of orphan products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. Rare diseases, as generally defined in the US Orphan Drug Act (ODA), are diseases or conditions with a prevalence of fewer than 200,000 persons in the US. Approximately 30 million Americans are affected by 7,000 known rare diseases but only a few hundred of these rare diseases have approved treatments. The Orphan Products Grants Program is an incentive program that has been supporting clinical trial research since 1983 and has funded clinical trials that have facilitated the marketing approval of 70 products. Lack of natural history data presents a significant unmet need for rare disease product development. To help address this unmet need the program has also committed funding to natural history studies since 2016.
Learn about the background and impact of the Orphan Products Grants Program, including past and currently supported Clinical Trials and Natural History Studies.
See request for applications (RFAs) for grants currently available through OOPD, steps to submit, and resources for applicants and current grantees.
See recent press release, publications, and historical information.
Find contact information for various questions relating to the program.