Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (UG3/UH3 Clinical Trial Required) | RFA TR 20 031

Opportunity Information: Apply for RFA TR 20 031

The National Institutes of Health (NIH) funding opportunity titled "Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (UG3/UH3 Clinical Trial Required)" (Funding Opportunity Number RFA-TR-20-031; CFDA 93.350) is a discretionary, health-focused cooperative agreement designed to move promising therapeutics into clinical testing across multiple rare diseases that share the same underlying molecular cause. The central idea is to support "basket" clinical trials, meaning a single clinical trial framework can evaluate one drug (or drug approach) across more than one rare disease, as long as those diseases are linked by a common molecular etiology. This approach is intended to increase efficiency in rare disease drug development by leveraging shared biology, reducing duplication across separate trials, and generating broader evidence for a therapy that targets a specific molecular mechanism rather than a single diagnosis.

The award mechanism uses the UG3/UH3 phased cooperative agreement structure and explicitly requires a clinical trial. In practice, this structure typically supports a milestone-driven transition from an initial planning and start-up phase (UG3) to a later implementation and execution phase (UH3). Because it is a cooperative agreement, NIH is expected to have substantial programmatic involvement during the life of the project, which can include collaboration on study conduct expectations, milestone tracking, and other aspects of trial oversight consistent with NIH cooperative agreement policies.

Eligibility is broad and includes many standard domestic applicant types, such as state, county, and city or township governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations other than federally recognized tribal governments; public housing authorities and Indian housing authorities; nonprofit organizations with and without 501(c)(3) status (as long as they are not institutions of higher education, which are separately listed); for-profit organizations other than small businesses; small businesses; and other organizations. The FOA also highlights additional eligible applicant categories, including Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISIs); Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Tribally Controlled Colleges and Universities (TCCUs); faith-based or community-based organizations; eligible federal agencies; regional organizations; Indian/Native American tribal governments that are not federally recognized; and U.S. territories or possessions.

At the same time, the opportunity places clear limits on non-U.S. participation at the applicant level. Non-domestic (non-U.S.) entities and foreign institutions are not eligible to apply, and non-domestic components of U.S. organizations are not eligible to apply. However, "foreign components" are allowed as defined in the NIH Grants Policy Statement, which generally means a U.S. applicant may include certain well-justified elements of the project that take place outside the United States if they meet NIH policy requirements and receive appropriate approvals.

Key administrative details included in the source information are that the sponsoring agency is NIH, the funding instrument is a cooperative agreement, and the original closing date for applications was November 12, 2020 (with a creation date of September 15, 2020). The provided summary data does not specify an award ceiling or the expected number of awards, indicating those fields were not populated in the excerpt. Overall, the FOA is aimed at accelerating therapeutics for rare diseases by supporting molecularly driven, multi-disease clinical trials under a structured, milestone-based UG3/UH3 model with active NIH involvement.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Basket Clinical Trials of Drugs Targeting Shared Molecular Etiologies in Multiple Rare Diseases (UG3/UH3 Clinical Trial Required)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.350.
• This funding opportunity was created on 2020-09-15.
• Applicants must submit their applications by 2020-11-12. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), Nonprofits having a 501 (c) (3) status with the IRS, other than institutions of higher education, Nonprofits that do not have a 501 (c) (3) status with the IRS, other than institutions of higher education, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for RFA TR 20 031

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