The objective of FDA's Orphan Products Natural History Grants Program is to support 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.
The ultimate goal of these natural history studies is to support clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy.
FDA provides grants for natural history studies that will either assist or substantially contribute to market approval of these products.
Applicants must include in the application's Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States (US) is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the US per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.
Additional information may be required upon request, for example, regarding population estimate and rationale.