Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed) | PAR 22 184

Opportunity Information: Apply for PAR 22 184

The National Institutes of Health (NIH) is offering a discretionary funding opportunity titled "Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)" under Funding Opportunity Number PAR-22-184. This program uses the U01 cooperative agreement mechanism, which generally means NIH staff will have substantial scientific and programmatic involvement during the project, beyond what is typical in a standard grant. The opportunity is a reissue of PAR-19-220 and is intended to help researchers and organizations do the practical, pre-trial work needed to make future clinical trials in rare neurological and neuromuscular diseases more feasible and more likely to succeed, while explicitly not allowing the award itself to support an actual clinical trial.

The central purpose of the program is to support activities that move a field or a specific candidate intervention closer to being ready for a well-designed clinical study. In the rare disease space, many promising ideas fail to translate because basic readiness pieces are missing, such as clear natural history data, validated or well-justified outcome measures, workable recruitment strategies, standardized protocols across sites, or operational tools like data standards and registries. This FOA is aimed at closing those gaps so that a subsequent, appropriately designed trial (funded through a different mechanism) can launch on stronger footing. The "clinical trial not allowed" label is an important boundary: applicants should propose preparatory and enabling work rather than interventional testing meant to evaluate safety or efficacy in a trial framework.

NIH is the sponsoring agency, and the opportunity falls under the Health activity category. It is associated with CFDA numbers 93.350 and 93.853, reflecting NIH program areas relevant to neurological and neuromuscular research. The award ceiling is listed as $750,000, indicating the maximum budget level anticipated per award (as presented in the source data). The original closing date is 2024-08-19, and the record creation date is 2022-05-13. The number of expected awards is not provided in the supplied listing, so prospective applicants typically would need to consult the full FOA text or NIH notices for any later updates on award volume, budgets, or related administrative details.

Eligibility is broad and includes many types of domestic U.S. applicants: 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; public housing authorities/Indian housing authorities; nonprofits with and without 501(c)(3) status (other than institutions of higher education); for-profit organizations other than small businesses; small businesses; and an "other" category. The announcement also explicitly calls out additional eligible applicants and institution types that NIH wants to ensure are included, such as Alaska Native and Native Hawaiian Serving Institutions; Asian American, Native American, and 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 agencies of the federal government; Indian/Native American tribal governments that are not federally recognized; U.S. territories or possessions; regional organizations; and non-domestic (non-U.S.) entities, meaning foreign organizations can be eligible as well.

Taken together, PAR-22-184 is best understood as a translational infrastructure and planning opportunity for rare neurological and neuromuscular disease research. It is designed to fund the careful groundwork that makes future interventional studies realistic, ethical, and scientifically interpretable, especially in settings where patient populations are small and heterogeneity is high. Applicants who are considering this program would typically frame their project around concrete readiness deliverables (for example, harmonized datasets, validated endpoints, recruitment and retention approaches, site networks, or other tools that de-risk later trials) rather than proposing a trial itself, since clinical trials are expressly not permitted under this U01.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.350, 93.853.
• This funding opportunity was created on 2022-05-13.
• Applicants must submit their applications by 2024-08-19. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $750,000.00 in funding.
• 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 PAR 22 184

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