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

The Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional), opportunity number FOR-FD-25-017, is a forecasted grant initiative issued by the U.S. Department of Health and Human Services through the Food and Drug Administration (FDA). This funding opportunity is part of the agency’s efforts to support research that can significantly influence the development of medical products for rare diseases or conditions where unmet clinical needs persist. The grant aligns with FDA's ongoing mission to advance public health through rigorous scientific inquiry and regulatory science, particularly in areas that lack sufficient clinical knowledge or therapeutic development. The primary objective of this Notice of Funding Opportunity (NOFO) is to promote efficient and innovative natural history studies that can close existing knowledge gaps in the field of rare diseases. These studies are expected to provide high-quality, interpretable data elements that can inform regulatory and clinical development pathways. The FDA intends for this program to target studies that remove barriers to product development and generate broad, meaningful impact either within a specific rare disease or across multiple rare diseases that share similar pathophysiological features. While the opportunity allows for clinical trials, they are considered optional under this mechanism. As a discretionary grant under CFDA number 93.103, this opportunity is open to a diverse array of applicant types. Eligible applicants include higher education institutions (both public and private), nonprofit organizations (regardless of 501(c)(3) status), for-profit organizations including small businesses, state and local governments, tribal governments (both federally recognized and non-federally recognized), U.S. territories, independent school districts, public housing authorities, tribal organizations, community-based organizations, regional organizations, foreign organizations, and non-domestic components of U.S.-based organizations. Foreign components as defined in the NIH Grants Policy Statement are also permitted, broadening the international scope of the research effort. The total estimated program funding has not yet been disclosed, and while the minimum award amount is unspecified, the maximum ceiling per award is set at $400,000. No matching funds are required from the applicants, removing a common barrier to entry for smaller organizations or early-stage research teams. The number of awards to be issued under this opportunity has not yet been determined. At this stage, the opportunity remains in the forecasted phase, with no concrete dates provided for application submission, award decisions, or project initiation. Additional details regarding application questions, submission components, and evaluation criteria have not been published yet. This grant opportunity is listed under several funding activity categories, including agriculture, consumer protection, and food and nutrition, which may reflect broader interpretations of rare disease impacts across societal domains. Interested applicants are encouraged to monitor Grants.gov for updates regarding the estimated post date and final application due date, neither of which have been announced. The opportunity was last updated on July 11, 2025, and was also initially forecasted on that same date for the Fiscal Year 2025 funding cycle. For inquiries or further information regarding this funding opportunity, applicants may contact Kimberly Pendleton at 240-402-7610. No email address was provided at this time. Given its forecasted status, potential applicants should remain attentive to the official announcement of the full funding opportunity details when the application window formally opens.