Aug 6, 2025, 11:30

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

The FDA’s Office of Orphan Products Development (OOPD) invites applications for RFA-FD-25-017, a reissue of RFA-FD-22-001, to support efficient and innovative natural history studies that fill critical knowledge gaps and advance medical product development for rare diseases. Both prospective and retrospective observational studies are welcome, with optional inclusion of clinical trial components.

Eligibility Criteria:

Applicant Organizations: U.S. and foreign higher-ed institutions, nonprofits (with or without 501(c)(3)), for-profits, small businesses, state/local/federal governments, tribal governments, NGOs, school districts, housing authorities.
PD(s)/PI(s): Any individual(s) with requisite skills; multiple PD/PI applications allowed with a Leadership Plan.
Registrations: SAM.gov (with CAGE/NCAGE & UEI), eRA Commons account(s), Grants.gov.

Funding Details:

Prospective Studies: Up to 400K USD/year for 1–4 years (max 1.6M USD), plus up to 200K USD/year additional for innovative trial designs (max 600K USD/year).
Retrospective Studies: Up to 150K USD/year for 1–2 years (max 300K USD).
Project Periods: Prospective, up to 4 years; Retrospective, up to 2 years; non-competing continuation years based on performance and funding.

Key Dates

Open Date: December 12, 2025.
Letter of Intent Due: January 9, 2026.
Application Due: February 10, 2026 by 11:59 PM ET.
Earliest Start Date: September 2026.

Where to Apply & More Information:

Apply via: Grants.gov Workspace or NIH ASSIST (Funding Opportunity Number RFA-FD-25-017).
Scientific Contact: Katherine Needleman ([email protected]).
Grants Management: Kimberly Pendleton ([email protected]).