First in Human and Early Stage Clinical Trials of Novel Investigational Drugs or Devices for Psychiatric Disorders (U01-Clinical Trial Required) | PAR 18 427

Opportunity Information: Apply for PAR 18 427

The grant opportunity "First in Human and Early Stage Clinical Trials of Novel Investigational Drugs or Devices for Psychiatric Disorders (U01-Clinical Trial Required)" is a National Institutes of Health (NIH) cooperative agreement aimed at accelerating early clinical testing of new treatments for psychiatric disorders, especially where current options are limited or inadequate. It focuses on gathering strong, decision-quality human data as quickly as possible so that promising approaches can be advanced into later-stage development, and weak or non-viable approaches can be stopped early. In practical terms, the program is designed to "de-risk" novel drug and device candidates by producing the kinds of results that make it easier to attract follow-on private investment and support eventual FDA approval pathways.

The FOA supports milestone-driven early stage clinical trials in both pediatric and adult populations. For drug candidates, the announcement emphasizes first-in-human (FIH) and Phase Ib studies for agents with novel mechanisms of action. These early studies are expected to do more than basic safety screening: they must assess whether the drug is actually engaging its intended biological target, including evidence relevant to brain exposure when appropriate, and demonstrate measurable pharmacological effects. Alongside target engagement, the studies must carefully evaluate safety and tolerability, with the explicit purpose of deciding whether the candidate is feasible to move forward into Phase II or proof-of-concept (PoC) trials in psychiatric indications.

For Phase II/PoC work, the FOA sets expectations that go beyond symptom checklists alone. Supported studies must evaluate the drug's impact on clinically relevant physiological systems using functional measures, and they must also examine clinical indicators of effect. The intention is to link mechanism to meaningful outcomes: showing that a candidate not only reaches and affects the brain or target pathway, but also produces measurable changes in biological or functional domains that matter for psychiatric illness, while providing early signals of clinical benefit.

The announcement also explicitly includes novel devices and combination approaches. For devices, it supports first-in-human and early feasibility studies (EFS), with required evaluation of target engagement (for example, evidence that a neuromodulation device is affecting the intended circuit or biomarker), safety, tolerability, and efficacy. The device language mirrors the drug emphasis on rapid, rigorous early testing that can support clear go/no-go decisions and position a technology for the next stages of development.

A central feature of this FOA is its emphasis on collaborative partnerships between academic or biomedical researchers and biotechnology or industry partners. The cooperative agreement structure (U01) signals substantial NIH involvement and oversight compared to a standard research grant, and the partnership requirement reflects the program's translational goal: to move candidates toward real-world development, regulatory readiness, and eventual commercialization or broader clinical adoption. The overall thrust is not basic discovery, but structured early clinical evaluation that produces actionable evidence for development decisions.

In terms of eligibility, the opportunity is broadly open across many U.S.-based organization types, including state, county, and local governments; public and private institutions of higher education; federally recognized tribal governments and other tribal organizations; public housing authorities/Indian housing authorities; nonprofits (with or without 501(c)(3) status); and for-profit organizations (including small businesses, and other than small businesses). It also highlights additional eligible applicant categories such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Tribally Controlled Colleges and Universities (TCCUs), Alaska Native and Native Hawaiian Serving Institutions, and Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), along with faith-based or community-based organizations and U.S. territories or possessions. At the same time, it clearly restricts foreign participation: non-U.S. entities and non-U.S. components of U.S. organizations are not eligible, and foreign components (as defined by NIH policy) are not allowed.

Administratively, the FOA is listed as PAR-18-427 under NIH, within the health funding activity category and CFDA number 93.242, and uses the cooperative agreement funding instrument type. The original closing date provided in the source information is February 18, 2021, and while the listing includes fields such as award ceiling and expected awards, those specific values are not provided in the text you shared. Overall, the program is best understood as a translational, milestone-based NIH effort to move high-potential psychiatric drugs and devices through the earliest clinical stages with strong mechanistic and functional evidence, positioning successful candidates for Phase II/III development and eventual regulatory pathways.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "First in Human and Early Stage Clinical Trials of Novel Investigational Drugs or Devices for Psychiatric Disorders (U01-Clinical Trial Required)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.242.
• This funding opportunity was created on 2017-12-06.
• Applicants must submit their applications by 2021-02-18. (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 18 427

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