Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) | RFA FD 25 020

Opportunity Information: Apply for RFA FD 25 020

This funding opportunity is a Food and Drug Administration (FDA) grant announcement that supports clinical trials of orphan medical products intended to address unmet needs in rare diseases or rare conditions. The central goal is practical and regulatory-facing: to generate clinical evidence on efficacy and/or safety that can be used to support either a new indication for an existing product or a change in product labeling. In other words, the FDA is looking to fund trials that produce data with a clear path to informing regulatory decisions and ultimately improving available treatment options for patients with rare diseases, where therapies are often limited or nonexistent.

The NOFO emphasizes not only running clinical trials, but doing so in ways that strengthen the rare disease development ecosystem. FDA specifically signals interest in trials that are collaborative, efficient, and/or innovative. That typically means study designs and operational approaches that can work well in rare disease settings, where patient populations are small, sites may be geographically dispersed, and natural history data can be limited. The broader intended impact is to increase the number of approved treatments for rare diseases and to create ripple effects that make future rare disease trials easier, faster, and more informative.

Mechanically, this is an R01-style clinical trial grant (clinical trials required), issued as a discretionary grant under the FDA. The opportunity is listed under CFDA 93.103, and it is categorized within Agriculture, Consumer Protection, Food and Nutrition. The funding opportunity number is RFA-FD-25-020, and it is described as a reissue of an earlier announcement (RFA-FD-23-001), signaling continuity in FDA priorities and an ongoing commitment to this type of clinical development support.

A wide range of applicant types are eligible. Eligibility extends across higher education institutions (public and private), nonprofit organizations (including those with and without 501(c)(3) status), for-profit organizations (including small businesses and larger companies), and multiple levels of government (local, county, state, tribal, territorial, and federal). The eligible applicant list also includes independent school districts, public housing authorities/Indian housing authorities, faith-based and community-based organizations, and regional organizations. Importantly for global rare disease research networks, non-U.S. entities are eligible to apply, non-U.S. components of U.S. organizations are eligible, and foreign components are allowed as defined in the NIH Grants Policy Statement. This broad eligibility is consistent with how rare disease research is often conducted, since expertise and patients may be distributed internationally.

Key administrative details provided in the source include an award ceiling of $900,000 and an original closing date of May 16, 2028, indicating a multi-year window in which applications may be accepted according to the announcement’s schedule. While the expected number of awards is not specified in the provided text, the ceiling gives a sense of the maximum funding level per award under this notice. The opportunity was created on July 11, 2025, which helps place it in the timeline of FDA’s orphan product development efforts.

In practical terms, this NOFO is best suited for applicants who already have a product and a credible clinical development plan aimed at a specific rare disease unmet need, and who can justify how the proposed trial will produce interpretable safety and efficacy data relevant to labeling or indication changes. Competitive projects are likely to be those with a realistic recruitment strategy for small populations, strong clinical endpoints (or well-justified surrogate endpoints), and an operational plan that shows the trial can be executed efficiently despite common rare disease constraints. The FDA’s framing makes clear that they are not simply funding exploratory research; they are investing in clinical studies that can meaningfully advance a product toward regulatory outcomes and patient access.

• The Food and Drug Administration in the agriculture, consumer protection, food and nutrition sector is offering a public funding opportunity titled "Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.103.
• This funding opportunity was created on 2025-07-11.
• Applicants must submit their applications by 2028-05-16.
• Each selected applicant is eligible to receive up to $900,000.00 in funding.
• Eligible applicants include: Others.

Apply for RFA FD 25 020

[Watch] Creating a grant proposal using the step-by-step wizard inside the applicant portal: