Catalyzing Innovation in Pediatric Pharmacology Clinical Trial Design and Resource Access (X01 Clinical Trial Not Allowed) | PAR 20 161

Opportunity Information: Apply for PAR 20 161

Catalyzing Innovation in Pediatric Pharmacology Clinical Trial Design and Resource Access (PAR-20-161) is an NIH grant opportunity designed to help researchers move promising pediatric therapeutics ideas closer to a feasible, high-quality clinical trial, without actually funding or conducting the clinical trial itself. The core aim is to give investigators structured, expert support for planning activities and concept development for single-site or multi-site investigator-initiated trials that address important gaps in pediatric drug development. The opportunity is tied to the Best Pharmaceuticals for Children Act (BPCA) Program, meaning applicants are expected to propose trial concepts that align with BPCA priorities and respond to unmet needs in how medicines are studied, dosed, and evaluated in children.

A central emphasis of this program is innovation in trial design for pediatric pharmacology, especially when standard clinical trial approaches are difficult, inefficient, or ethically complicated in children. The FOA explicitly encourages concepts for future well-designed studies that may require non-traditional or novel designs in pediatric therapeutics. In practice, this points toward approaches such as adaptive designs, Bayesian methods, opportunistic or sparse sampling pharmacokinetic studies, extrapolation strategies, master protocols, pragmatic designs, enrichment strategies, or other methods that can reduce burden, improve feasibility, and still generate rigorous evidence in pediatric populations where recruitment is limited or heterogeneity is high.

Rather than primarily providing money for patient enrollment or trial operations, the award provides access to high-value consultative resources. Awardees can tap into the Pediatric Trials Network (PTN) and the BPCA Data Coordinating Center for expertise in pediatric drug development and clinical trial design. This support is meant to strengthen the scientific and operational foundation of a proposed trial concept, helping investigators refine the research question, endpoint strategy, design framework, feasibility assumptions, and analysis considerations so the concept can mature into a competitive and executable clinical trial proposal for later submission through an appropriate funding mechanism.

This is an X01 mechanism and is labeled "Clinical Trial Not Allowed," which is important for applicants to understand. It means the supported activities are limited to planning and concept development and do not include initiating or running a clinical trial under this award. The end product is typically a well-developed trial concept and associated planning outputs that position the team to pursue subsequent clinical trial funding through NIH or another sponsor.

Eligibility is broad and includes many domestic organization types: state, county, 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 and other tribal organizations; public housing authorities/Indian housing authorities; nonprofit organizations with or without 501(c)(3) status (excluding institutions of higher education where relevant); for-profit organizations other than small businesses; and small businesses. The FOA also highlights additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, AANAPISIs, Hispanic-serving institutions, historically Black colleges and universities, tribally controlled colleges and universities, eligible federal agencies, faith-based or community-based organizations, regional organizations, and US territories or possessions. At the same time, non-domestic (non-US) entities (foreign organizations and foreign institutions) are not eligible to apply, and non-domestic components of US organizations are not eligible. However, foreign components, as defined in NIH policy, are allowed, which generally means limited, well-justified parts of the work may occur outside the US under a US applicant organization when permitted by NIH rules.

Administratively, the opportunity is listed as a discretionary grant under the NIH, in the Health, Income Security and Social Services category, with CFDA number 93.865. The original closing date provided in the source information is 2023-03-17, and the posting indicates no stated award ceiling and no specified expected number of awards in the excerpted data. The FOA was created on 2020-04-02. Overall, this program is best viewed as a pathway for pediatric pharmacology researchers who have a clinically important trial idea but need specialized design and coordinating-center input to shape it into a robust, innovative, and fundable pediatric clinical trial concept aligned with BPCA priorities.

• The National Institutes of Health in the health, income security and social services sector is offering a public funding opportunity titled "Catalyzing Innovation in Pediatric Pharmacology Clinical Trial Design and Resource Access (X01 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): 93.865.
• This funding opportunity was created on 2020-04-02.
• Applicants must submit their applications by 2023-03-17. (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 20 161

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