Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01)

The purpose of this funding opportunity announcement (FOA) is to support studies that advance medical product development through the characterization of the natural history of rare diseases/conditions with unmet needs. Through the support of efficient and innovative natural history studies, FDA expects to address critical knowledge gaps, to remove major barrier(s) to progress in the field, to exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and to inform current or future product development including the design of clinical trial(s) and to ultimately inform the development of medical products that meet patient needs.