Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01) | PAR 18 534

Opportunity Information: Apply for PAR 18 534

The Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01) opportunity (Funding Opportunity Number PAR 18 534) is a National Institutes of Health program, specifically through the National Institute of Neurological Disorders and Stroke (NINDS), designed to speed up and strengthen the path toward successful clinical trials in rare neurological and neuromuscular diseases. Rather than funding large, definitive efficacy trials right away, this announcement focuses on the practical work that often determines whether a future trial can launch quickly and produce interpretable results. The core idea is to support clinical studies that close key knowledge gaps that commonly slow rare-disease trial development, such as uncertainty about which endpoints to use, whether proposed biomarkers are reliable and meaningful, and whether enough well-characterized patients can be identified and followed in a consistent way across sites.

The studies supported under this program are meant to be explicitly "trial readiness" efforts. That typically includes validating clinical outcome measures (for example, showing that a functional scale is sensitive to change, reliable across raters and centers, and relevant to patient function), validating or qualifying biomarkers (for example, demonstrating that a lab measure, imaging readout, or physiological signal is reproducible and tracks with disease progression or treatment-relevant biology), and characterizing patient cohorts (for example, establishing natural history information, defining inclusion and exclusion criteria, mapping variability in progression, or identifying subgroups that may respond differently). These are the kinds of preparatory studies that can reduce risk for later interventional trials by improving trial design choices like endpoints, sample size assumptions, visit schedules, stratification factors, and feasibility of recruitment.

This grant uses the U01 funding mechanism, which is a cooperative agreement. In practice, that means the project is expected to be run with substantial involvement from NIH staff compared with a standard research project grant. Cooperative agreements are commonly used when an agency anticipates an active partnership to help ensure the work aligns closely with program goals, supports standardization where needed (such as common data elements or harmonized measures across sites), and produces outputs that are directly usable in imminent trial planning. The activity category is health, and the CFDA number associated with this opportunity is 93.853.

A wide range of organizations are eligible to apply, reflecting the fact that rare disease research often requires collaboration across academic medical centers, patient foundations, community-based groups, and in some cases industry partners. Eligible applicants include state, county, and local governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; nonprofit organizations with or without 501(c)(3) status (other than institutions of higher education); for-profit organizations other than small businesses; and small businesses. It also allows participation from Native American tribal governments (federally recognized) and Native American tribal organizations (other than federally recognized tribal governments), as well as public housing authorities and Indian housing authorities where relevant.

The announcement also explicitly highlights additional eligible applicant types that are often important for broadening participation and strengthening community engagement. These include Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); 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; U.S. territories or possessions; Indian/Native American tribal governments that are not federally recognized; and non-domestic (non-U.S.) entities, meaning foreign organizations can apply or participate. This broad eligibility is consistent with the reality that rare disease expertise and patient populations may be geographically dispersed, and meaningful cohort characterization or endpoint validation may require international or multi-regional recruitment.

Key administrative details from the source data include that the opportunity is categorized as discretionary funding, with the funding instrument type listed as a cooperative agreement (U01). The original closing date in the provided record is March 13, 2019, and the record creation date is December 20, 2017. The award ceiling and expected number of awards are not specified in the data shown, which usually means applicants would need to consult the full FOA text for budget guidance, project period limits, and any program-specific expectations around scope, milestones, or deliverables.

Overall, this FOA is aimed at making future rare-disease clinical trials more feasible, faster to start, and more likely to produce clear answers by investing early in the measurement tools and patient-cohort knowledge that trials depend on. By funding careful validation of outcomes and biomarkers and by supporting rigorous cohort characterization, NINDS is targeting the common bottlenecks that can derail rare disease trials, with the explicit expectation that these readiness studies will accelerate the initiation of later trials and improve their odds of success.

• 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)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.853.
• This funding opportunity was created on 2017-12-20.
• Applicants must submit their applications by 2019-03-13. (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 PAR 18 534

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