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

This funding opportunity aims to support natural history studies that provide high-quality and interpretable data to advance medical product development for rare diseases with unmet needs. By addressing critical knowledge gaps and overcoming significant barriers, these studies are expected to have a substantial and broad impact on specific rare diseases or groups of rare diseases with similar underlying mechanisms. The initiative is designed to facilitate the development of new treatments by enabling a deeper understanding of disease progression and characteristics. Issued by the Food and Drug Administration under the Department of Health and Human Services, this opportunity encourages innovative and efficient research approaches. The clinical trials component is optional, allowing flexibility in study design. The point of contact for further information is Kimberly Pendleton, who can be reached via email or phone. This opportunity was posted in July 2025 and is intended to foster progress in the rare disease field by supporting studies that can ultimately lead to improved therapeutic options.