Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)

This NIH program funds preclinical proof-of-concept studies for rare diseases using established disease models to test whether a therapeutic candidate warrants further development. It also supports accompanying pharmacodynamic and pharmacokinetic studies, but not clinical trials or development of the model itself. Eligible applicants include U.S. higher education institutions, nonprofits, for-profits, governments, tribal organizations, faith-based or community-based organizations, regional organizations, and some federal entities; foreign organizations are not eligible, though foreign components are allowed and foreign subawards/subcontracts are not. The NIH expects to commit up to $1,200,000 in FY26, with 3 to 5 awards anticipated; the two-year project period may not exceed $275,000 in total direct costs, with no more than $200,000 in any single year. No cost sharing is required, and applications focused on rare cancers are not responsive.

✳ AI-generated summary — verify against the announcement

• Health
• Research & Discovery
• Technology & Product Development
• Patients & People with Health Conditions
• Researchers & Scholars
• Biomedical & Disease Research

• ✓ City or township governments
• ✓ County governments
• ✓ For-profit organizations other than small businesses
• ✓ Independent school districts
• ✓ Native American tribal governments (Federally recognized)
• ✓ Native American tribal organizations (other than Federally recognized)
• ✓ Nonprofits with 501(c)(3) status (other than higher education)
• ✓ Nonprofits without 501(c)(3) status (other than higher education)
• ✓ Others
• ✓ Private institutions of higher education
• ✓ Public and State controlled institutions of higher education
• ✓ Public housing authorities / Indian housing authorities
• ✓ Small businesses
• ✓ Special district governments
• ✓ State governments