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Issued by

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

Purpose

Pediatric drug development processes have been established over the past two decades through legislative efforts such as the Food and Drug Administration Modernization Act (FDAMA), the Best Pharmaceuticals for Children Act (BPCA), the Pediatric Research and Equity Act (PREA), European legislation, and other initiatives to advance the regulatory science.

Children have historically been therapeutic orphans when it comes to drug development; particularly with respect to missing or limited drug development and drug discovery tools, quality pharmacology (both pharmacokinetics-PK and pharmacodynamics-PD) in pediatrics, and limitations in science advances in areas such as “omics” research, artificial intelligence, and drug repurposing. Despite the important advances brought about through legislation and regulatory initiatives, several needs remain apparent, including:

• A universally available programmatic approach to pediatric drug development, where many studies are currently designed and conducted without reference to preceding or subsequent steps in drug development, such as dosing finding studies as the gold standard before or as a part of larger clinical trials.
• Use of appropriate study designs in clinical trials that incorporate validation of endpoints, biomarkers, pharmacometrics models relevant to pediatric populations.
• Integration of approaches that are currently underutilized in pediatric drug development, such as team science approaches in pediatric drug development (including collaborations amongst clinical trialists, clinicians, statisticians, epidemiologists, pharmacologists, toxicologists, chemists, regulatory specialists, biomedical experts)
• Application of good practice recommendations and improvements in engagement with practitioners and patients relating to pediatric drug development.

Background

Historically it has been mandated by congress that the BPCA program at the National Institutes of Health (NIH), in consultation with the Food and Drug Administration (FDA) develop and publish a priority list of needs in pediatric therapeutics, including drugs or indications that require study, update this list every 3 years, sponsor clinical studies of prioritized drugs, and submit study data to the FDA for label change considerations. 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. Despite the advances in labeling by the legislative mandates, the NIH BPCA Program has identified remaining gaps in pediatric drug development. At the annual BPCA stakeholders meeting in 2020, the concept of a BPCA led program to develop a Framework to Enable Pediatric Drug Development initiative was introduced.

The overarching call for a framework that enables pediatric drug development includes:

1. The need to centralize and effectively advertise to the various contributors to pediatric drug development the large amount of good practice* tools and resources that currently exist in pediatric drug development.[*Good practice recommendations can be existing regulatory guidelines or existing well-constructed multi-stakeholder consensus documents].
2. The need to identify and address pathways towards closing remaining gaps in good practice (where more guidelines or methodological studies are needed).
3. The need to promote integration between the various stages AND bridge collaborations between the various stakeholders of pediatric drug development (preclinical and clinical stakeholders).
4. The need to integrate approaches such as big data, real world evidence, the use of medical technology in drug development (e.g. m-health), patient and public involvement with research, and broader inclusion of diverse populations in trials.

Information Requested

The NICHD requests your input into this framework development as part of the BPCA 2021 - 2022 priorities.

The purpose of the framework is to create an annotated and curated collection of documents that will provide existing resources on relevant topics as well as to identify gaps that need to be addressed before conducting pediatric drug development research. The development of this framework aligns with the mandate of the BPCA to improve knowledge gaps in Pediatric therapeutics and can serve as a toolkit of resources and approaches (with actionable items) that can subsequently be customized to specific therapeutic areas, indications, type of drug, biologics, developmental stages, and phenotypic expression. Such a framework will further enable pediatric drug development, while being customizable for specific therapeutic programs.

1. For 2021 through 2022 the BPCA therapeutic priorities will align with six prioritized areas. These include:

• Advancing Clinical Trial Designs and Conduct in Pediatric Drug Development
• The Role of Pharmacodynamic Biomarkers Research in Pediatric Drug Development
• How Pharmacokinetic Modeling Can Be Used to Inform Drug Dosing
• Enhancing Research in Pediatric-Friendly Formulations
• The Role of Pharmacoepidemiology in Pediatric Drug Development
• The Role of Qualitative Systems Pharmacology in Pediatric Drug Development

2. There will be an annual stakeholder meeting in November 2021 to provide updates on the BPCA Clinical Program and the BPCA Framework.

This is a time-sensitive Request for Information (RFI). There will be several opportunities for public input in both processes in 2021 through 2022:

If you would like to provide additional information on existing resources or remaining gaps in the above six Prioritized areas, please visit:

http://surveymonkey.com/r/bpcaframework.

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, the FDA, and an ad hoc evaluation panel to be convened by the NICHD as part of this framework development. Nominations will be accepted through August 31, 2021. This survey monkey link is the primary vehicle by which information can be shared.

For reference, the 2020 BPCA Framework to Enable Pediatric Drug Development is located on the BPCA website: https://www.nichd.nih.gov/research/supported/bpca/activities/framework

Inquiries for the RFI may be sent to [email protected] or [email protected] . Please indicate “BPCA Framework" as the subject line of your email.

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 responder'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 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:

Perdita Taylor-Zapata, M.D.
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Telephone: 301-496-9584
Email: [email protected]