Medications Development for the Treatment of Alcohol Use Disorder - CT optional (U01) | RFA AA 18 009

Frequently Asked Questions (FAQs)

What is the name and reference number of this funding opportunity?

The opportunity is titled "Medications Development for the Treatment of Alcohol Use Disorder - CT optional (U01)" and is identified as RFA-AA-18-009.

Which federal agencies are involved?

This is a National Institutes of Health (NIH) funding opportunity administered by the National Institute on Alcohol Abuse and Alcoholism (NIAAA).

What is the overall purpose of this FOA?

The purpose is to support later-stage, human-focused medications development for Alcohol Use Disorder (AUD), with an emphasis on practical, translational work that moves promising candidate compounds through specific drug development milestones rather than supporting open-ended basic research.

What stage of research and development is this FOA meant to support?

The FOA is designed for later-stage development activities aimed at advancing candidate medications through IND-enabling steps, reaching and progressing beyond an Investigational New Drug (IND) submission, and moving into early clinical testing in humans.

What does "U01 cooperative agreement" mean in this context?

The U01 mechanism is a cooperative agreement, meaning NIAAA staff are expected to have substantial scientific and programmatic involvement as the project proceeds. This reflects an expectation of ongoing coordination with NIAAA during execution of the work.

What kinds of activities does the FOA expect projects to include?

Projects are expected to focus on development activities such as IND-enabling work (as appropriate), Phase 1 clinical studies to establish human safety, tolerability, and dosing, and Phase 2 work that can include human laboratory studies and proof-of-concept trials in people.

Are Phase 1 clinical studies specifically supported?

Yes. The FOA explicitly expects Phase 1 clinical studies that establish safety, tolerability, and dosing in humans.

Are Phase 2 clinical studies specifically supported?

Yes. The FOA describes Phase 2 work, including human laboratory studies and proof-of-concept trials in people.

Is the goal to reach an IND submission, or to go beyond IND?

The FOA aims to move candidates through the steps needed to reach and progress beyond an IND submission and into early clinical testing.

What is meant by "milestone-driven" expectations?

Applicants are expected to propose a plan with clear, concrete milestones and well-defined go/no-go decision points. The plan should show how the award will reduce development risk (de-risk the compound) and advance it to the next stage of development.

Do applicants need to define where they start and where they end in the development pipeline?

Yes. Each application is expected to state the starting development stage (where the project enters the pipeline) and the concrete endpoint it will reach (the exit criteria).

What kinds of endpoints or "exit criteria" are expected?

The excerpt emphasizes that endpoints should be concrete, measurable development outcomes tied to advancing the compound (for example, reaching an IND-related milestone and/or completing early clinical testing steps), with explicit go/no-go decision points.

Is this FOA intended to fund basic research or exploratory discovery work?

No. The intent is practical and translational, focused on moving a promising candidate forward through key development milestones rather than supporting open-ended basic research.

Will the FOA support animal efficacy studies?

Generally, no. The FOA will not support animal efficacy studies intended to demonstrate that the compound works, unless those studies are specifically required by the FDA or are otherwise deemed necessary through peer review.

What is the program orientation regarding preclinical work?

The program is oriented toward development activities that directly support regulatory advancement and early-stage clinical evaluation, not broad preclinical discovery or exploratory animal testing.

Who is this opportunity meant for (in practical terms)?

It is aimed at organizations that can realistically execute clinical and regulatory development work and that can show meaningful commitment of resources, including specialized capabilities such as manufacturing, regulatory documentation, clinical operations, and data management.

Are for-profit organizations eligible to apply?

Yes. NIAAA explicitly invites applications from both for-profit and not-for-profit organizations.

Are academic institutions eligible to apply?

Yes. Institutions of higher education (public/state-controlled and private) are included in the eligible applicant categories.

Are pharmaceutical and biotechnology companies eligible to apply?

Yes. The FOA explicitly invites applications from pharmaceutical and biotechnology companies.

Are small businesses eligible to apply?

Yes. Small businesses are listed as eligible. The FOA also notes interest in small businesses that are not using the SBIR/STTR pathway.

Are nonprofit organizations eligible (including those without 501(c)(3) status)?

Yes. Nonprofit organizations with and without 501(c)(3) status are included among eligible applicants.

Can government entities apply (state, local, tribal, and related entities)?

Yes. Eligible applicants include state governments; county, city/township, and special district governments; independent school districts; federally recognized tribal governments; and public housing authorities/Indian housing authorities.

Are tribal organizations eligible if they are not federally recognized tribal governments?

Yes. Tribal organizations other than federally recognized tribal governments are listed as eligible.

Are U.S. territories or possessions eligible?

Yes. U.S. territories or possessions are listed as eligible.

Are non-U.S. (foreign) entities eligible to apply?

Yes. The eligible applicant categories include non-U.S. (foreign) entities.

Are faith-based and community-based organizations eligible?

Yes. Faith-based and community-based organizations are listed as eligible categories.

Are minority-serving institutions specifically highlighted as eligible?

Yes. The FOA highlights categories such as Historically Black Colleges and Universities (HBCUs), Hispanic-serving institutions, Tribally Controlled Colleges and Universities, Alaska Native and Native Hawaiian Serving Institutions, and AANAPISIS institutions as eligible.

Does the FOA allow a single organization to apply without partners?

Yes. The FOA notes that a single entity may apply if it can demonstrate significant resource commitment, such as providing salary support for key personnel or handling production and formulation of clinical trial materials.

What kind of "resource commitment" is NIAAA looking for?

The FOA emphasizes operational readiness and meaningful commitment of resources. Examples mentioned include salary support for key personnel and the ability to produce and formulate clinical trial materials, along with access to development capabilities like manufacturing, regulatory documentation, clinical operations, and data management.

Is it recommended to contact NIAAA before submitting an application?

Yes. Applicants are strongly encouraged to contact NIAAA Division of Medications Development staff prior to submission, consistent with the cooperative nature of the award and the value of aligning milestones and regulatory strategy early.

What is the health topic area and program focus?

The focus is on medications development for the treatment of Alcohol Use Disorder (AUD), with emphasis on human-focused clinical and regulatory development steps.

Is this a discretionary grant opportunity?

Yes. The listing describes it as a discretionary funding opportunity in the health area.

What is the CFDA number associated with this opportunity?

The CFDA number provided is 93.273.

What were the key dates shown in the listing excerpt?

The excerpt lists a creation date of January 18, 2018, and an original closing date of March 20, 2018.

Does the provided excerpt include an award ceiling or expected number of awards?

No. The excerpt notes that it does not provide an award ceiling or the expected number of awards.

What is the defining feature of the announcement based on the excerpt?

The defining features described are the U01 cooperative agreement structure, the focus on IND-enabling and early human clinical development (Phase 1 and Phase 2), and the milestone-driven expectation that funded work will move a candidate AUD medication forward in a measurable, decision-oriented way.