Key Dates

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Issued by

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Purpose

Children have long been considered therapeutic orphans in drug development. They have been underrepresented in the creation of new drugs and drug discovery tools, as well as in the application of quantitative systems pharmacology (including pharmacokinetics (PK), pharmacodynamics (PD), and pharmacogenomics (PG)). In addition, pediatric drug development has often been excluded from early and active participation in emerging scientific fields such as “omics” research, artificial intelligence, and drug repurposing.

Over the past two decades, however, significant legislative efforts have been made to address these gaps. Key examples include the Food and Drug Administration Modernization Act (FDAMA), the Best Pharmaceuticals for Children Act (BPCA), and the Pediatric Research and Equity Act (PREA). Together with other initiatives, these measures have advanced pediatric drug development and strengthened the regulatory framework to close the gaps in pediatric drug development.

Section 409I of the BPCA mandate includes a pediatric drug development clinical program at the National Institutes of Health (NIH), working in consultation with the Food and Drug Administration (FDA), to develop and publish a priority list of needs in pediatric therapeutics, including drugs or indications that require study, to sponsor clinical trials of prioritized therapeutics, and to submit clinical trial results to the FDA for potential label changes.  As part of this RFI, the NIH is providing the scientific community with an update on the prioritization program associated with the clinical program and is seeking expert input into:

1. Determining future priorities
2. Updating and reviewing our currently available public facing resources.

The NIH program leaders of the BPCA program are responsible for global outreach to determine gaps in pediatric therapeutics and update a list of priorities at least every 3 years. The most recent list from 2024 can be found at https://www.nichd.nih.gov/research/supported/bpca/recent-priority-lists.

The NICHD invites all stakeholders to engage and provide expert opinions into two areas related to the Best Pharmaceuticals for Children Act (BPCA) NIH 409I Clinical Program: 

1. To submit nominations for research to be prioritized in the development of the Best Pharmaceuticals for Children Act (BPCA) Program’s 2026 Priority List of Needs in Pediatric Therapeutics; and 
2. To provide expert review of resources to be added to the existing BPCA Framework to support Pediatric Drug Development.

The NICHD is gathering nominations for drugs and therapeutic areas (e.g., pediatric condition, subpopulation, or setting of care) that require further study in children. Recommendations not related to pediatric therapeutics or to a specific drug, biologic, or device will be considered out of scope for this RFI.

This is a time-sensitive Request for Information (RFI).

Written responses will be accepted through January 30, 2026. All responses will be compiled and reviewed by a committee of stakeholder representatives. The submissions and the review of the submissions at the upcoming meeting will result in an updated BPCA priority list for 2026.

The purpose of this RFI is to obtain knowledge and gather information regarding current gaps in pediatric therapeutics. The information from this RFI will be used for prioritization planning purposes only and shall not be construed as a solicitation for applications, abstracts, or quotations, or as an obligation on the part of the NICHD or NIH to provide any funds on the basis of responses.

Background

The NIH BPCA program has been successful over the years with providing important clinical research that has improved knowledge of dosing, safety and efficacy of medications used in children. To date, the research program has supported more than 50 clinical trials that have resulted in 26 drug label changes.  For more information visit the BPCA Accomplishments page at https://www.nichd.nih.gov/research/supported/bpca/accomplishments.

The 2002 BPCA established the initial prioritization process. The process was further refined in 2010 and updated in 2024 to emphasize the following principles:

• Prioritization must be a well-defined process that includes a systematic approach and clear objectives and outcomes.
• The process must have well-defined objectives and criteria for measuring priority.
• Prioritization must be a dynamic but legitimate and fair process that incorporates transparency, stakeholder input, and strong leadership.

The BPCA priority list consists of key therapeutic needs in the medical treatment of children and adolescents. New therapeutic areas are selected for discussion and further prioritization. The most recent lists can be found on the BPCA Priorities page at  https://www.nichd.nih.gov/research/supported/bpca/recent-priority-lists.

Also, as part of our prioritization program, the NIH in consultation with experts in pediatric drug development, developed a list of existing resources called the BPCA Framework to Enable Pediatric Drug Development.  This initiative was launched in 2020 as part of the BPCA prioritization process. Subject-matter experts in pediatric care collaborated to research, identify, submit and review available resources of scientific articles, guidance documents, book chapters, and reports. Volunteer members of various public assemblies in areas related to pediatric drug development reviewed and evaluated references at the request of NICHD for best practice and guidance documents for inclusion in the BPCA Framework to Enable Pediatric Drug Development. The current framework can be found at https://www.nichd.nih.gov/research/supported/bpca/framework. 

The outcome of this work is an annotated, selected, and curated collection of resources of universal interest that relate to specific topics in pediatric drug development and to specific clinical populations. 

 Information Requested

The penultimate goal of the BPCA Priority List and Framework is to provide stakeholders involved in pediatric therapeutics research, including new and junior investigators, information on current needs in pediatric therapeutics as well as provide access to a multitude of resources to review prior to writing grant proposals and/or before conducting pediatric drug development research, respectively.

In order ensure that both resources are current and include the most recent data and guidances, the BPCA Priority List and Framework are curated every 2-3 years. New resources are systematically gathered by NICHD program staff in collaboration with informationists at the NIH Library and subsequently programmed into a survey for public review. BPCA stakeholders are being asked to review the current priority list and the new resources scheduled to be added to the Framework and to provide recommendations. Stakeholders are also able to make recommendations for removal of outdated resources from the current Priority List and Framework as well.

The NICHD requests your input into two key area that are a part of the BPCA 2025-2026 priorities:

1. Review of the current list of BPCA priorities as of May 2024.   
2. Commenting on the newly curated list of resources identified in order to update the BPCA Framework Initiative development.

This is a time-sensitive Request for Information (RFI). There will be several opportunities for public input in both processes.

If you would like to provide feedback on the 2024 BPCA Priority List of Needs in Pediatric Therapeutics, please email [email protected]. Please indicate “BPCA Priority List" as the subject line of your email.

If you would like to review and provide additional information on existing resources, please visit: 

https://www.surveymonkey.com/r/BPCAFrameworkCuration

If you would like to receive updates and summaries from the Annual BPCA Stakeholders Meeting held on December 12, 2025, in Bethesda, Maryland, please email [email protected].  Please indicate “BPCA Annual Meeting" as the subject line of your email.

Please note that except for individuals' email addresses and phone numbers, all responses will also be placed in a public access file. If you choose to omit contact information, your response will still be given full consideration.

How to submit a response

Submissions in response to this RFI will be considered by the NICHD and nominations will be accepted through January 30, 2026. This survey monkey link is the primary vehicle by which information can be shared.

Inquiries for the RFI may be sent to [email protected]

Responses to this RFI are voluntary. The NIH will use the information submitted in response to this RFI at its discretion and will not provide comments to any respondent's submission. Proprietary, classified, confidential, or sensitive information should not be included in your response. The United States government reserves the right to use any non-proprietary technical information in any resultant solicitation(s). This RFI is for planning purposes only and should not be construed as a solicitation or as an obligation on the part of the Federal Government, the National Institutes of Health, or individual NIH Institutes or Centers. The NIH will use the information submitted in response to this RFI at its discretion. NIH will analyze the information submitted and may share it internally or in public reports. The information may or may not be reflected in future solicitations, as appropriate and at the Government’s discretion. NIH advises respondents the Government is under no obligation to acknowledge receipt of the information provided and will not provide feedback to respondents.

Inquiries

Please direct all inquiries to:

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB) 
Email: [email protected]