Federal opportunity: U.S. Food and Drug Administration Office of Orphan Products Development, Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases, RFA-FD-25-020, R01 Clinical Trials Required.
What FDA is funding
This opportunity supports clinical trials of orphan products evaluating safety and/or efficacy in support of a new indication or change in labeling for rare diseases or conditions with unmet medical needs. Responsive products may include drugs, biologics, medical devices, and medical foods. FDA encourages efficient, collaborative, and innovative clinical trial approaches.
Current deadline and award controls
The current new-application deadline is October 20, 2026 at 11:59 PM Eastern Time. An optional letter of intent is due September 21, 2026. No late applications are accepted. Awards may support up to four years. Applicants may request up to $650,000 in total costs per year. Applications proposing justified innovative or efficient trial approaches may request up to an additional $250,000 in total costs per year, for a maximum of $900,000 per year. Cost sharing is not required.
Eligibility and submission
Eligible applicants include domestic and foreign public and private institutions of higher education, qualifying nonprofit organizations, small businesses and other for-profit organizations, state and local governments, Tribal governments and organizations, independent school districts, faith-based and community-based organizations, and other listed entities. Federal agencies are not eligible. Applications may be submitted through ASSIST, institutional system-to-system solutions, or Grants.gov Workspace and must be accepted error-free through Grants.gov and eRA Commons by the deadline.
Clinical-research requirements
A legitimate application requires an actual qualifying orphan-product clinical trial, a responsive protocol and statistical plan, qualified scientific and clinical leadership, evidence that the investigational product will be available for the proposed study, appropriate clinical sites and patient access, and all applicable human-subjects, single-IRB, good-clinical-practice, FDA regulatory, and training requirements. Applicant-specific regulatory status, IND or IDE requirements, certifications, product rights, clinical data, site commitments, and other mandatory facts must never be assumed or fabricated.
Atlas disposition
This is a high-value national rare-disease clinical-research opportunity with no mandatory match and structural eligibility for small businesses and other for-profit organizations. A direct Dr. Miltie path is structurally possible but is not submission-ready merely because the organization class is eligible. Advancement requires Marc review and independently substantiated evidence of a genuine qualifying orphan-product clinical trial, lawful product access or sponsorship, a qualified PI and clinical team, appropriate trial sites and patient population access, current federal registrations, and all required human-subjects and FDA regulatory infrastructure. If those prerequisites are not present, route as partner-led to qualified rare-disease programs, academic medical centers, clinical research organizations, biotechnology and pharmaceutical developers, and other eligible clinical-trial entities.