Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

The Food and Drug Administration (FDA), a key agency under the U.S. Department of Health and Human Services, is forecasting a funding opportunity titled “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required).” This grant initiative is part of the FDA’s ongoing efforts to advance research and development in rare diseases, with a particular focus on clinical trials that could lead to label expansions or new indications for products that treat these conditions. The funding mechanism falls under discretionary grants, specifically aligned with FDA research initiatives, and supports the broader agency mission of ensuring the safety and efficacy of medical products. This opportunity is intended to provide support for clinical trials of orphan-designated products—those developed specifically to treat rare diseases or conditions. These studies must be designed to assess the efficacy and/or safety of such products, and they should aim to produce data that could support a change in the product’s labeling. By encouraging collaborative, efficient, and innovative approaches to clinical trials, the FDA seeks to increase the number of approved treatments available for rare diseases, many of which currently lack viable therapeutic options. The agency anticipates that the program will also help promote broader advancements in the drug development landscape for rare diseases. The funding scope includes costs associated with conducting clinical trials, which may cover patient enrollment, trial management, data collection and analysis, and other trial-related activities. While the document does not specify a minimum or maximum award size, or total program funding, it does clarify that there is no cost-sharing or matching requirement for applicants. This makes the opportunity more accessible, particularly to organizations with limited financial capacity for matching funds. The number of expected awards has not been determined at this stage of the forecast. The eligibility for this opportunity is broad. Applicants may include institutions of higher education, both public/state-controlled and private; non-profit organizations with or without 501(c)(3) status; for-profit organizations including both small businesses and other business entities; as well as governmental entities at the city, county, state, and tribal levels. Faith-based organizations, community-based organizations, regional and non-domestic entities are also eligible. Notably, both U.S.-based and foreign organizations are permitted to apply, as are components of U.S. institutions located abroad. Since this is a forecasted opportunity, specific application dates including estimated post date, application due date, and project start date have not yet been published. The funding opportunity is expected to open in the future, and stakeholders interested in applying are encouraged to monitor Grants.gov or subscribe for updates to receive notice when the opportunity becomes active. There are no pre-application deadlines or submission-specific milestones indicated at this time. For further information or clarification, the main point of contact listed is Kimberly Pendleton, who can be reached at 240-402-7610. No email address has been provided in the forecast listing. Interested parties should check back on Grants.gov regularly or contact the FDA for updated details as the opportunity progresses from forecast to open status.