Orphan Products Clinical Trials Grants Program

The Orphan Products Clinical Trials Grants Program sits under the FDA Office of Orphan Products Development and supports clinical investigators testing drugs, biologics, medical devices, and foods for medical purposes in rare diseases and conditions. It is one of the FDA's main grant streams for orphan-product development and is tied to the Orphan Drug Act's effort to make rare-disease trials commercially and scientifically viable. The program runs annually and is funded at roughly $17 million a year, with about $12 million reserved for noncompeting continuation awards and about $5 million for new awards. FDA typically supports five to 12 new grants each year and keeps 60 to 85 projects active at a time. Eligible applicants include foreign or domestic, public or private, for-profit or nonprofit entities, including state and local government units; federal agencies may not apply. The rare-disease threshold is fewer than 200,000 people in the United States, and diagnostics or vaccines qualify only when the intended U.S. population is also below that level. This program fits investigators with a defined clinical question and a trial design that can support a new indication or a label change. FDA emphasizes efficacy and safety studies, including adaptive and seamless designs, modeling and simulation, and basket or umbrella trials, so the strongest applications are usually those with a clear path from protocol to actionable regulatory evidence. Because the route is annual and highly specialized, applicants do best when the trial is tightly built around an unmet rare-disease need rather than a broad exploratory research agenda.

Each grant below is a distinct funding opportunity with its own eligibility, scope, and deliverables.