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) grant opportunity is offered by the U.S. Department of Health and Human Services through the Food and Drug Administration (FDA). This initiative is designed to advance medical product development for rare diseases and conditions, particularly those with significant unmet needs. By funding innovative and efficient natural history studies, the FDA aims to fill critical knowledge gaps, reduce major scientific and regulatory hurdles, and promote more effective treatments for individuals affected by rare diseases. This funding opportunity supports projects that propose the design and implementation of high-quality, interpretable natural history studies. The goal is to generate comprehensive and reliable datasets that can accelerate therapeutic development by providing a better understanding of disease progression, variability, and patient subpopulations. This initiative encourages studies that could have a significant and broad impact on one or more rare diseases, especially those with similar pathophysiological characteristics. Studies funded under this grant may or may not include clinical trials. The opportunity falls under the Assistance Listing number 93.103, which supports research initiatives under the purview of the FDA. The program anticipates offering grants with an award ceiling of $400,000. Although the minimum award amount and total program funding have not been specified, there is no cost-sharing or matching requirement for applicants. The anticipated number of awards is not yet defined. Eligible expenses will support the development and execution of natural history studies aligned with FDA goals in rare disease research and product development. Eligibility for this grant is broad. Applicants may include higher education institutions (both public and private), nonprofits (with or without 501(c)(3) IRS status), for-profit organizations (including small businesses), government entities at the federal, state, county, municipal, and tribal levels, as well as public housing authorities and independent school districts. International entities and foreign components of U.S.-based organizations are also eligible. This inclusive scope is intended to encourage a diverse range of institutions and stakeholders to contribute to rare disease research. The opportunity is currently in a forecasted state, with no confirmed application opening, closing, or award dates as of July 11, 2025. The estimated posting date, submission deadline, and project start date have yet to be released. As this is a forecasted grant, it is suitable for early engagement and strategic planning by potential applicants. There is no indication that the program operates on a recurring or rolling basis. Interested applicants are encouraged to monitor the grant's listing for updates and prepare by reviewing prior examples of successful natural history studies in the rare disease space. For more information, applicants may contact Kimberly Pendleton at the FDA by phone at 240-402-7610. No email contact was provided in the forecast. Additional documentation, submission packages, and instructions will be made available once the opportunity is officially posted.