Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R01 - Clinical Trial Optional) | PAR 25 265

Frequently Asked Questions (FAQs)

What is the NIH funding opportunity PAR-25-265?

PAR-25-265 is a National Institutes of Health (NIH) funding opportunity titled "Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R01 - Clinical Trial Optional)." It supports research projects aimed at improving how serious conditions are identified and treated as early as possible in newborns.

What is the main goal of this grant program?

The program aims to speed progress from scientific discovery to real-world impact in newborn health by supporting (1) better tools and strategies to screen newborns for serious conditions and (2) stronger evidence and/or improved therapies that make early diagnosis beneficial.

What kinds of disorders are in scope?

The focus is on potentially fatal or disabling disorders that are either already identified through newborn screening programs or are strong candidates to be screened in the near future.

Why does the opportunity emphasize both screening and treatment?

The opportunity is built on the idea that newborn screening is only truly justified when two conditions are met: there is a reliable and accurate screening test that can be used at scale, and there is clear evidence that early identification improves outcomes because timely treatment or intervention helps.

What does NIH mean by "high priority" genetic conditions in this FOA?

In this announcement, "high priority" does not mean a condition is already recommended for routine screening. It refers to conditions where early identification would meaningfully change an infant's clinical course because an effective treatment or early intervention exists (or is close enough that screening would be valuable).

Is this opportunity limited to conditions already on newborn screening panels?

No. It includes conditions already being identified through newborn screening programs as well as conditions that are strong candidates for screening in the near future.

What research gaps is NIH trying to address?

NIH is looking for projects that help close the gap between promising science and real-world newborn screening adoption. Examples of gaps mentioned include limitations in test performance, lack of confirmatory testing workflows, insufficient evidence that early treatment improves outcomes, and a shortage of feasible therapeutic options for infants identified through screening.

What are the main research directions supported by this FOA?

The scope includes two major directions that can be proposed independently or combined: (1) development of novel screening approaches and (2) development of therapeutic interventions or early-treatment strategies for infants identified via screening, with an emphasis on evidence that early intervention improves outcomes.

What counts as "novel screening approaches" under this opportunity?

Examples described include new laboratory methods, improved biomarkers, enhanced assay platforms, and more robust analytical strategies designed specifically for newborn screening settings.

What does the FOA say about therapy development or early-treatment strategies?

The FOA encourages development of therapeutic interventions or early-treatment strategies for infants identified through screening, especially work that generates evidence showing early intervention improves health outcomes.

Can a single application include both screening development and therapy development?

Yes. The opportunity states that the two major research directions can stand alone or be combined in a single program of work.

What type of NIH grant mechanism is this?

This is an NIH R01 funding opportunity, which generally indicates a substantial, hypothesis-driven research project with a clear plan to generate impactful data.

What does "Clinical Trial Optional" mean in this announcement?

"Clinical Trial Optional" means applicants are allowed to propose a clinical trial if it fits their aims, but a clinical trial is not required.

What is the CFDA number and category for this opportunity?

The listing describes it as a discretionary grant opportunity under health and related social services with CFDA 93.865.

When is the application window open until?

Based on the provided information, the application window is open until the listed closing date of 2027-11-16.

Does the provided listing specify an award ceiling or the expected number of awards?

No. The provided source information does not specify an award ceiling or an expected number of awards. The description notes that applicants typically should rely on the full FOA and NIH institute guidance for budget expectations and competitiveness.

Who is eligible to apply?

Eligibility is broad. Eligible applicants include many U.S.-based organizations and several special categories, including government entities, higher education institutions (public and private), tribal governments and tribal organizations, public housing authorities, nonprofits (with or without 501(c)(3) status, outside higher education), for-profit organizations (other than small businesses), and small businesses.

Which government entities are eligible?

Eligible government applicants include state governments, county governments, city/township governments, special district governments, federally recognized Native American tribal governments, and eligible federal agencies.

Are schools and universities eligible?

Yes. Independent school districts, public and state-controlled institutions of higher education, and private institutions of higher education are included as eligible applicants.

Are tribal entities eligible even if they are not federally recognized?

Yes. The eligibility list includes tribal organizations that are not federally recognized, in addition to federally recognized Native American tribal governments.

Are nonprofit organizations eligible?

Yes. Nonprofits with 501(c)(3) status and nonprofits without 501(c)(3) status (outside higher education) are eligible.

Are for-profit organizations eligible?

Yes. For-profit organizations (other than small businesses) are listed as eligible, and small businesses are also listed as eligible.

Are faith-based or community-based organizations eligible?

Yes. Faith-based or community-based organizations are explicitly called out as eligible groups.

Are U.S. territories or possessions eligible?

Yes. The eligibility list includes U.S. territories or possessions.

Are non-U.S. (foreign) organizations eligible?

Yes. The provided information states that non-domestic (non-U.S.) entities (foreign organizations) are included in the eligibility list.

Why does the opportunity allow such broad eligibility?

The description explains that newborn screening research often requires collaboration across academic laboratories, clinical centers, public health programs, community organizations, and technology developers, which supports the need for wide eligibility.

What kinds of outcomes is NIH hoping these projects will influence?

The opportunity is designed to support data and advances that could improve broader screening implementation and improve decisions about what belongs on newborn screening panels by strengthening screening accuracy and demonstrating the benefit of early intervention.