Consensus Study Report

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COMMITTEE ON STRATEGIES TO ENHANCE PEDIATRIC HEALTH RESEARCH FUNDED BY NIH

PHYLLIS A. DENNERY (Co-Chair), Brown University, Warren Alpert Medical School

FREDERICK P. RIVARA (Co-Chair), University of Washington, Seattle Children’s Hospital

ALEXANDER G. BASSUK, University of Iowa Carver College of Medicine

GLENN FLORES, University of Miami Miller School of Medicine

CHRISTOPHER B. FORREST, Children’s Hospital of Philadelphia

VITTORIO GALLO, Seattle Children’s Hospital

ROSEMARY D. HIGGINS, Florida Gulf Coast University

PAMELA S. HINDS, Children’s National Hospital and George Washington University

SHAFALI S. JESTE, David Geffen School of Medicine at the University of California, Los Angeles

LEAH C. KOTTYAN, Cincinnati Children’s Hospital Medical Center and University of Cincinnati

BRENDAN LEE, Baylor College of Medicine and Texas Children’s Hospital

MARY B. LEONARD, Stanford University School of Medicine and Lucile Packard Children’s Hospital Stanford

KEILA N. LOPEZ, Children’s Hospital Colorado

KRISTY MURRAY, Emory University and Children’s Healthcare of Atlanta

ABBY R. ROSENBERG, Boston Children’s Hospital and Harvard Medical School

DAVID C. SCHWEBEL, University of Iowa

SEEMA K. SHAH, Ann & Robert H. Lurie Children’s Hospital and Northwestern University Feinberg School of Medicine

National Academy of Medicine American Board of Family Medicine/Puffer Fellow

ALYSSA TILHOU, Boston University Chobanian & Avedisian School of Medicine

Study Staff

UDARA PERERA, Study Director/Senior Program Officer, Health Care and Public Health Program Area

ALEXIS WOJTOWICZ, Program Officer, Health Care and Public Health Program Area (from February 2025)

RUTH COOPER, Program Officer, Health Care and Public Health Program Area (from February to October 2025)

ELLA MORSE, Research Associate, Health Care and Public Health Program Area (from April 2025)

L. BRIELLE DOJER, Research Associate, Health Care and Public Health Program Area (from April to July 2025)

AJA DRAIN, Research Associate, Health Care and Public Health Program Area (from September to November 2025)

ABIGAIL GODWIN, Research Associate, Health Care and Public Health Program Area (until April 2025)

ELIANA PIEROTTI, Senior Program Assistant, Health Care and Public Health Program Area

JULIE SCHUCK, Senior Program Officer, Social and Economic Systems Program Area (until October 2025)

MARC MEISNERE, Senior Program Officer, Health Care and Public Health Program Area (from November 2025)

SHARYL J. NASS, Senior Program Director, Health Care and Public Health Program Area

NATACHA BLAIN, Senior Program Director, Social and Economic Systems Program Area

Consultants

KACI MCCOY, Emory University

LAUREN J. KOENIGSBERG, Roberts Center for Pediatric Research, Children’s Hospital of Philadelphia

MICHAEL ZIERLER, Science Writer

Reviewers

This Consensus Study Report was reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published report as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process.

We thank the following individuals for their review of this report:

Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations of this report nor did they see the final draft before its release. The review of this report was overseen by RONALD KLEINMAN, Massachusetts General Brigham for Children, and DAN G. BLAZER, Duke University. They were responsible for making certain that an independent examination of this report was carried out in accordance with the standards of the National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the authoring committee and the National Academies.

Acknowledgments

The committee extends its gracious thanks to the many individuals who shared their time and expertise to support its work and inform its deliberations. This study was sponsored by the National Institutes of Health, and we thank Elizabeth Baden, Sarah Glavin, Rohan Hazra, and Tracy King for their guidance and support.

The committee benefited greatly from discussions with the individuals who presented at the committee’s webinars: Gaya Dowling, Dennis Durbin, Valerie Durrant, Matthew Gillman, Sarah Glavin, John Glod, David Goff, Kimberly Gray, Colleen Hadigan, Rohan Hazra, Amelia Karraker, Kelly King, Tracy King, George Koob, Walter Koroshetz, Jeanne Marrazzo, Susan Mendley, Mara Olenick, Gail Pearson, Eliseo Pérez-Stable, Sallie Permar, Ann Poduri, Tiina K. Urv, Sara Van Driest, Jennifer Webster-Cyriaque, and Brigitte Widemann. Agendas for the public meetings are provided in Appendix B.

Our appreciation goes to the reviewers for their invaluable feedback on an earlier draft of the report and to the monitor and coordinator who oversaw the report review process. The committee acknowledges the many staff within the Center for Health, People, and Places (formerly of the Health and Medicine Division) who provided support in various ways to this project, including Udara Perera (study director), Alexis Wojtowicz (program officer), Ruth Cooper (program officer), Julie Schuck (senior program officer), Marc Meisnere (senior program officer), Abigail Godwin (research associate), Ella Morse (research associate), Brielle Dojer (research associate), Aja Drain (research associate), Eliana Pierotti (senior program

assistant), Natacha Blain (senior program director), Greysi Patton (finance business partner), and Wahidullah Nazari (finance business partner). The committee extends great thanks and appreciation to Sharyl J. Nass, senior program director, Health Care and Public Health Program Area, who oversaw the project. The report review, production, and communications staff all provided valuable guidance to ensure the success of the final product. Kaci McCoy is credited with her assistance in the investigation of study sections. Lauren Koenigsberg is credited for her assistance with data analysis and visualization, and Michael Zierler is credited for superb editorial assistance in preparing the final report.

Contents

PREFACE

ACRONYMS AND ABBREVIATIONS

SUMMARY

Study Task and Approach

Pediatric Health Research at NIH: Past and Present

The Current Crisis in Child Health

The Current Pediatric Research Portfolio at NIH

Pediatric Research at the NIH CC

Pediatric Research Prioritization and Inclusion

Facilitators of Successful Inter-IC Initiatives

Challenges to Conducting Pediatric Health Research

A Vision for the Future of Pediatric Health Research and Child Health

1 INTRODUCTION

Study Background and Context

Project Origin and Statement of Task

Significance of Pediatric Health Research

Study Approach

Terms Used in This Report

Organization of the Report

References

2 BACKGROUND: HISTORY, FUNDING LANDSCAPE, AND NIH STRUCTURE

History of NIH-Supported Pediatric Research

Federal Laws and Policies Supporting and Protecting Children in Health Research: A Brief History

The Organization of NIH and Its Capacity and Support for Pediatric Health Research

The Broader Landscape: Other Pediatric Research Funders

References

3 CURRENT NIH PEDIATRIC RESEARCH PORTFOLIO

Delineating Pediatric Research at NIH

NIH Data Systems for Monitoring Pediatric Research Support

Gaps and Opportunities for the RCDC Pediatric Spending Category

Data on Pediatric Research Funding by NIH

Current Pediatric Research at the Clinical Center

References

4 PRIORITIZATION, COORDINATION, AND INTEGRATION OF PEDIATRIC RESEARCH WITHIN AND ACROSS NIH INSTITUTES AND CENTERS

How Institutes and Centers Prioritize Pediatric Research

The NIH Pediatric Research Consortium

Inter-IC Initiatives: Successes and Challenges

References

5 SUPPORTING PEDIATRIC RESEARCH AND ADDRESSING CHALLENGES

Ethical and Legal Impediments

Scientific Challenges to Conducting Pediatric Research

Methodologic Barriers to Conducting Pediatric Research

Structural and Procedural Challenges and Opportunities

Financial Barriers Faced by Pediatric Health Researchers

The Strategic Importance of Investing in Pediatric Research

References

6 IMPLEMENTING THE COMMITTEE’S VISION FOR THE FUTURE OF PEDIATRIC HEALTH RESEARCH AT NIH

Goals and Recommendations for NIH-Supported Pediatric Research

A Coda

References

APPENDIXES

A BIOGRAPHICAL SKETCHES OF COMMITTEE MEMBERS

B PUBLIC MEETING AGENDAS

C CALLS FOR PERSPECTIVES QUESTIONS

D HISTORY OF PEDIATRIC HEALTH AND NIH STRUCTURE AND ORGANIZATION

E PRINCIPAL INVESTIGATOR DEPARTMENT CLASSIFICATIONS

F NIH PEDIATRIC RESEARCH CONSORTIUM AGENDA ITEMS

G PEDIATRIC REPRESENTATION ON SCIENTIFIC REVIEW

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Boxes, Figures, and Tables

BOXES

1-1 Statement of Task

1-2 The Committee’s Vision and Goals

3-1 Insights from Grantees: Perceived Funding Discrepancies Between ICs

3-2 Pediatric Scientific Priorities Identified in the 2023 PRSP

4-1 Successful Engagement with the Pediatric Research Community

5-1 U.S. Federal Regulations for Pediatric Research Definition of a Child

5-2 The Challenge of Having Insufficient Pediatric Representation on Study Sections

5-3 Financial Impediments

6-1 The Committee’s Vision and Goals for Pediatric Research at NIH

D-1 Selected Recent Historical Events at the NIH CC

FIGURES

S-1 Pediatric research expenditures, FY 2015–FY 2024

S-2 Pediatric research expenditures as a proportion of NIH non-administrative expenditures, FY 2015–FY 2024

S-3 Pediatric research expenditures by IC and Office of the Director, FY 2015–FY 2024

S-4 Committee goals and downstream impacts

1-1 Pediatric health in the United States

1-2 Pediatric research across the life course

2-1 The National Institutes of Health, as of March 14, 2025

3-1 Examples of category fingerprints for the tobacco, back pain, and obesity RCDC categories

3-2 Total NIH non-administrative spending for research, FY 2015–FY 2024

3-3 Pediatric research expenditures, FY 2015–FY 2024

3-4 Pediatric research expenditures as a proportion of NIH non-administrative expenditures, FY 2015–FY 2024

3-5 Proportions of pediatric research projects and expenditures by type of grantee institution, FY 2024

3-6 Academic departmental location of the principal investigator, FY 2015–FY 2024

3-7 Pediatric research expenditures by IC and Office of the Director, FY 2015–FY 2024

5-1 Off-ramps preventing the inclusion of children in research

5-2 Proposed approach to the inclusion of children in research

5-3 Payment for participation in research

5-4 NIH grant application process

5-5 NIH grant application timeline

5-6 Comparison of sites needed for adult versus child enrollees in concurrent studies about renal disease

6-1 Committee goals and downstream impacts

TABLES

2-1 Pediatric Health Achievements Funded by NIH

2-2 The 27 NIH ICs, Year of Establishment, and Primary Focus Area, as of March 21, 2025

2-3 NIH Research Center Awards

3-1 Clinical Center’s Pediatric Capacity from FY 2018 to FY 2024

4-1 Facilitators and Inhibitors of Successful Inter-IC Pediatric Research Initiatives

5-1 Potential Justifications and Examples of Appropriate Exclusion of Children under Existing NIH Policy

5-2 R18 or Pediatric Dissemination and Implementation Funding, FY 2021–FY 2024

E-1 Principal Investigator Department Classifications

G-1 Pediatric Representation Within NIH Study Sections, as of April 24, 2025

G-2 Pediatric Representation Within NIH Study Sections/Special Emphasis Panels, as of April 24, 2025

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Preface

Thanks in no small part to research funded by the National Institutes of Health (NIH), and particularly the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the mortality of infants, children, and adolescents in the United States has decreased dramatically since the turn of the 20th century. Beyond this, since the turn of the 20th century we have witnessed myriad other remarkable advancements in health and medical research that have dramatically altered the quality and length of the lives of infants, children, and adolescents. The numbers speak for themselves: Since 1900, the percentage of children born alive who die before their first birthday has decreased by nearly half in the United States. Over the last 75 years, children with cystic fibrosis have gone from rarely surviving past age 5 to having a median life expectancy of 61 years. Fewer than 10 percent of children with acute lymphocytic leukemia survived 60 years ago; now, at least 90 percent will survive for 5 years or more. More recently, infant mortality decreased in the United States nearly 25 percent between 1999 and 2022.

Advances in sanitation, nutrition, public health, health care, and more have occurred thanks to the rigorous and incremental march of scientific discoveries, building one on another through sharing of knowledge and through collaboration across disciplines and nations. This scientific research would have been impossible to conduct without the support of NIH, which since 1887 has in some form funded research ranging from basic molecular insights to research that impacts whole communities. As the world’s largest funder of health and basic biomedical research, it has involved hundreds of thousands of scientists and staff at NIH and in our nation’s academic

centers, children’s hospitals, research organizations, and industry while engaging millions of individuals as research participants.

Yet, there is so much more to be done, particularly for our nation’s youth. In the United States, while declines in child and adolescent mortality have occurred across all races, the rate in Black youth remains twice that of White youth; firearm-related deaths of all kinds among adolescents are increasing; and mental, emotional, and behavioral disorders among children and adolescents are more prevalent than ever. In addition, many chronic diseases of adulthood have antecedents in infancy or childhood. Adverse childhood experiences, exposure to chronic and toxic stress, and childhood hypertension and dyslipidemia are just a few antecedents linked with the onset of cancer, cardiovascular disease, depression, diabetes, and hypertension in adulthood. Pediatric research provides cornerstone knowledge and understanding about the causes of these issues as well as their prevention and treatment.

The current scrutiny that NIH is undergoing may result in the most substantial changes in the organization since its official establishment in 1930. In preparing this report, which was mandated by Congress and commissioned by NIH before these current considerations, the committee took seriously its charge to examine NIH’s current pediatric research portfolio and structure and make recommendations focused on improving NIH’s overall support of pediatric research. The committee strived to balance celebrating the progress made in pediatric research supported by NIH by identifying where meaningful changes could be made in its approach. The committee also recognized the importance of acknowledging the reality of NIH’s financial and staff capacity while still identifying longer-term goals and possibilities. As such, some of the committee’s recommendations can be implemented with relatively quick actions, while others may take more time and a wider range of stakeholders to fully implement.

The committee is grateful to the U.S. Congress and NICHD for supporting this work. This report lays out a framework for how pediatric research should be situated and prioritized within NIH, emphasizing a life course approach, so as best to return dividends in health for our future generations of youth and adults. Pediatric research must continue, building on the enormous knowledge gained from the last century of research. This is necessary, not just for the health of our nation’s youth but for everyone.

Frederick P. Rivara, Co-Chair
Phyllis Dennery, Co-Chair
Committee on Strategies to Enhance
Pediatric Health Research Funded by NIH
December 2025

Acronyms and Abbreviations

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