The FDA Office of Orphan Products Development's (OOPD) mission is to assist and encourage the identification, development, and availability of safe and effective medical products for people with rare diseases and disorders.
In support of that mission, the OOPD facilitates three grant programs:
The Orphan Products Clinical Trials Grants Program (formerly known as the Orphan Products Grants Program) with an annual budget of approximately $15 million, supports clinical development of products including drugs, biologics, medical devices, and medical foods for use in rare diseases and conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. This program provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products.
The Orphan Products Natural History Grants Program with an annual budge of approximately $2 million supports studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics