Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)

The Food and Drug Administration, under the Department of Health and Human Services, has issued a forecast for a funding opportunity titled Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases. This initiative aims to support natural history studies that generate high quality and interpretable data to advance medical product development for rare diseases and conditions with unmet needs. By addressing critical knowledge gaps and removing barriers to progress, the program seeks to create a significant impact on specific rare diseases or those with similar pathophysiology. The opportunity is categorized under NAICS code 541715 and was posted on July 11, 2025. Interested parties can contact Kimberly Pendleton for further information. The primary goal is to facilitate the development of medical products by improving the understanding of rare disease progression and characteristics through innovative research.