4

Prioritization, Coordination, and Integration of Pediatric Research Within and Across NIH Institutes and Centers

National Institutes of Health (NIH) studies are established, prioritized, and funded through a variety of mechanisms (see Chapter 2). Once a research priority has been articulated, the coordination of the research endeavor can occur from within a single institute, center, or office, or multiple institutes and centers (ICs) can collaborate on a research priority. As Chapter 3 makes clear, nearly every IC supports some amount of pediatric health research. For the past 7 years, pediatric health research has gained a higher profile at NIH thanks to the work of the NIH Pediatric Research Consortium, or NPeRC, which coordinates and facilitates pediatric research across NIH.

In this chapter, the committee evaluates efforts by NIH to incorporate pediatric research priorities within and across ICs, along with the challenges to doing so and how these challenges may be mitigated or eliminated. Several examples of successful NIH initiatives are summarized, including ones focused fully on pediatrics and others that included both adult and pediatric participants. Factors are identified that influenced the success of these studies in advancing our understanding of pediatric health and disease.

HOW INSTITUTES AND CENTERS PRIORITIZE PEDIATRIC RESEARCH

Every IC at NIH prepares and regularly updates its strategic plan, which serves as that IC’s roadmap for establishing goals and research priorities as well as for identifying challenges and opportunities associated with implementing the plan to achieve the IC’s research objectives

(NIH RePORT, n.d.). The committee reviewed all available IC strategic plans, focusing on how pediatric research was highlighted, articulated, and evaluated in each plan. Recognizing that pediatric research initiatives are funded in each IC, the committee was most interested in whether and how these pediatric research initiatives were featured within the current strategic plans. To further inform this chapter, the committee interviewed representatives from several ICs about their prioritization and planning around pediatric research (see Appendix B for the open session agendas).

Each IC engages in its own strategic planning process, typically producing a new plan every 5 years (FABBS, 2025). These plans are public facing and are meant to share the goals and priorities of the specific IC with other ICs at NIH, the extramural scientific community, Congress, advocacy groups, and the general public. The strategic plans are generally similar to one another in structure, and each relates to the NIH-wide strategic plan. However, there is considerable heterogeneity in the level of detail provided on the process of developing the plan, identifying priority research areas, assessing success, and modifying goals, priorities, and methodologies based on research outcomes.

Though strategic planning across NIH ICs is not standardized, most ICs report using a process that includes an internal analysis of major areas, a coordinated effort to collect input from stakeholders, an interim call for feedback on strategic focus areas, and the finalization of strategic plans by IC leadership. Engaged stakeholders typically include subject experts, patient advocacy groups, and the broader public. Feedback is generally collected through workshops, requests for written feedback, and institute councils. The committee found no information about the specific identities or profiles of these subject experts or stakeholders, so there was no opportunity for the committee to learn more from them about the IC strategic planning process generally and about the incorporation of pediatric research priorities specifically. Community engagement with patient advocacy groups and individuals impacted by specific health issues is highlighted in many strategic plans as a crucial input into the strategic planning process. Finally, some opportunities are developed in response to time-sensitive needs (e.g., HIV, Zika, COVID-19).

The Inclusion of Pediatric Research in IC Strategic Plans

The committee reviewed all the available strategic plans (or the equivalent documents found among the strategic plan reports) of the 25 ICs that fund research and found that 19 of them included the term “pediatrics” or “children” (CSR, 2025; FIC, 2025; NCATS, 2025; NCCIH, 2021; NCI, 2025; NEI, 2021; NHGRI, 2020; NHLBI, 2025; NIA, 2020; NIAAA, 2024; NIAID, 2025; NIAMS, 2025; NIBIB, 2012; NICHD, 2025b; NIDA, 2022;

NIDCD, 2023; NIDCR, 2025; NIDDK, 2021; NIEHS, 2025; NIGMS, 2021; NIH Clinical Center, n.d.-b; NIMH, 2025; NIMHD, 2021; NINDS, 2021; NINR, n.d.; NLM, 2017).¹ Despite that, every IC supports intramural and/or extramural projects categorized as “pediatric,” based on the Research, Condition, and Disease Categorization system (see Figure 3-7). Of note, and as discussed in Chapter 3, the Clinical Center produced its Pediatric Research Strategic Plan in 2023. Within the 19 strategic plans that address children or pediatrics, the details around strategic direction and plans for pediatric health are generally not directly articulated and often are embedded within larger strategic priorities. For instance, numerous ICs highlight research on specific topics “across a lifespan,” which includes childhood origins of chronic diseases. Nearly all strategic plans also emphasize efforts that broadly impact child health, such as advancing medical diagnostics and technologies for high-consequence viral pathogens (e.g., the National Institute of Allergy and Infectious Diseases [NIAID]), interventions as early in the lifespan as possible (e.g., the National Institute of Mental Health [NIMH] and the National Institute of Diabetes and Digestive and Kidney Diseases [NIDDK]), and disease prevention (e.g., NIMH and the National Institute on Drug Abuse [NIDA]). In fact, most ICs prioritize diseases that originate in or are specific to children but don’t necessarily support research related to lifelong care of those conditions. For example, strategic plans underscore the importance of pediatric diseases, including monogenic traits (the National Human Genome Research Institute), childhood cancers (the National Cancer Institute [NCI]), epilepsy (the National Institute of Neurological Disorders and Stroke [NINDS]), pediatric liver diseases (NIDDK), and type 1 diabetes (NIDDK).

The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) strategic plan focuses on “Improving Child and Adolescent Health and the Transition to Adulthood” and “Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities” (NICHD, 2025b). Similarly, the National Institute on Alcohol Abuse and Alcoholism (NIAAA) has a specific initiative focused on developing mechanisms to identify children affected by prenatal alcohol exposure (NIAAA, 2024). The National

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¹ The following ICs’ strategic plans or equivalent documentation mentioned “children” or “pediatrics”: CC; FIC; National Center for Complementary and Integrative Health; National Eye Institute; National Heart, Lung, and Blood Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Environmental Health Sciences; National Institute of Dental and Craniofacial Research; National Institute on Aging; National Institute on Minority Health and Health Disparities; NCI (using the Annual Plan & Professional Judgment Budget, which NIH links under strategic plans and visions); NIAAA; NIAID (six disease-specific strategic plans, four of which mention children); NICHD; NIDCD; NIDDK; NIDA; NIMH; and NINDS.

Institute on Deafness and Other Communication Disorders (NIDCD) presents a strategic initiative specifically focused on “early detection, diagnosis, and treatment for newborns, infants, and young children who are deaf or hard-of-hearing” (NIDCD, 2023). NCI articulates the importance of its strategic focus on cancers of childhood, identifies challenges to research in this area, and prioritizes focus areas within childhood cancer research (NCI, 2025). The Clinical Center’s strategic plan highlights the importance of advances in gene therapy, cellular engineering, and diagnosis of heritable rare diseases to the ability to care for pediatric patients. It also identifies a short-term goal of performing a “gap analysis between current capabilities and those required to care for younger patients safely” (NIH Clinical Center, n.d.-a).

Measuring the Success of Strategic Plan Goals

Few plans articulate exactly how success in achieving their goals will be measured or monitored. Some plans point out broadly that benchmarks are developed and measured, and in several plans, there is a mention of identifying new ways to track research progress and identify opportunities. For example, the 2025 NICHD strategic plan states that the IC has “defined metrics to measure success and implementation efforts and will track and report on those” (NICHD, 2025b). Most ICs provide yearly updates of their strategic plans, with revisions that result from an assessment of the prior year’s activities. For example, NIMH provides a section called “Progress” for each of its four goals, with a high-level description of grants funded and discoveries made as a result of funding relevant to the programmatic goal (NIMH, 2025). Specific notices of special interest (NOSIs) and requests for application (RFAs) within each goal are described as examples of progress. However, there is no public-facing avenue to determine what grants were funded under a specific NOSI.² For this reason, the committee was unable to determine what NPeRC-driven NOSIs culminated in funded grants. Compared with most other ICs, NIAID provides considerable detail about outcome measurement through a section in its strategic plan called “Program Evaluation” (NIAID, 2025). The narrative in this section describes NIAID’s process of assessment, which includes a needs assessment, feasibility studies, and process and outcome evaluations. The institute’s evaluation metrics include bibliometrics (publications, patents), changes to clinical guidelines that lead to changes in clinical practice, evolution of collaborative networks, and program influence and impact.

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² Going forward, NIH will no longer use NOSIs. Instead, NIH will use Highlighted Topics, which are not replacing NOSIs but will serve a similar purpose. More information is available at https://grants.nih.gov/funding/find-a-fit-for-your-research/highlighted-topics.

In addition to measuring success through goal monitoring, NIH has also successfully engaged with the pediatric research community to establish ongoing research priorities, as indicated by a number of examples provided by NIH grant recipients in response to the committee’s call for perspectives (see Box 4-1).

A combination of both transparent, measurable benchmarks and increased opportunities to collaboratively establish research priorities is critical for improving the success of NIH’s pediatric research portfolio. There remain opportunities in this process for more granularity around outcome measurement regarding pediatric research and, ultimately, the impact of that research on pediatric health.

Conclusion 4-1: Although each National Institutes of Health institute and center (IC) allocates a portion of its budget to pediatric research, a substantial proportion of ICs do not mention children, child health, a life course approach, or pediatrics in their strategic plans. Among ICs that do address child health in their strategic plans, there is limited standardization of how ICs prioritize and measure success around pediatric research in the strategic planning process, both within and across ICs. As an example of the life course benefits of pediatric research, investments in research to study and address the childhood antecedents of chronic disease could improve the health of older adults in the long term.

Recommendation 4-1: All directors of National Institutes of Health institutes and centers that fund research should explicitly incorporate pediatric health and potential downstream life course impacts into their strategic plans, as childhood chronic conditions, exposures, and experiences may have disproportionate health impacts into adulthood. Include metrics such as those focused on investigators (e.g., funding rates and career advancement), scientific discovery (i.e., publications and innovations), impact on child health (e.g., morbidity, mortality), and downstream life course impacts (e.g., premature cardiovascular disease) to measure success in meeting their goals.

The Impact of Executive Branch and Congressional Mandates

Executive branch and congressional mandates impact the strategic focus and funding of specific research areas by NIH ICs. Sometimes a mandate is the result of Congress passing a law (e.g., the 21st Century Cures Act) or inserting specific language in an annual appropriations bill (e.g., a directive for NIH to fund certain types of research, establish programs, or meet specific goals such as advancing childhood cancer or autism research). It is the responsibility of the NIH director, through the Office of the Director, to interpret the mandate and assign responsibility to specific ICs. When the language is broad, it can be coordinated across multiple ICs by developing inter-NIH initiatives. When language is topic-specific (e.g., pediatric cancer), the responsibility is assigned to one or a few relevant ICs. Congress authorizes and can appropriate funding directly for an initiative to an IC. For example, congressional support for the Undiagnosed Diseases Network was given to NINDS.³ Ultimately, other funds were needed, including from the Office of the Director, so this, de facto, is a direction for an IC to implement a program.

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³ Consolidated Appropriations Act, 2023, H.R. 2617, Public Law 117-328, 117th Congress (2021–2022).

The IC impacted by executive and congressional mandates operationalizes the request through a variety of mechanisms. Sometimes, the mandate is formally added to the strategic planning process. The IC then sets aside funding specifically designated for that mandate and may create new funding lines. Often, new requests for RFAs and NOSIs are developed to engage extramural researchers, and new research programs, clinical trial networks, or training grants are developed around the mandated topic. Some congressional mandates also result in the development of new data repositories or biobanks to support the research topic (e.g., the All of Us research program).

ICs often convene workshops, advisory councils, and stakeholder meetings to solicit input from the scientific community and the public to ensure that the proposed implementation is responsive to the needs of the public. ICs must track their mandate-related activities and report to Congress—through annual NIH reporting and/or ad hoc updates—the relevant accomplishments, as requested by congressional committees (e.g., Children’s Health Act [2000]⁴, Best Pharmaceuticals for Children Act [2002] for safer therapies in children, 42 U.S. Code 282 to support pediatric device development) (NICHD, n.d.-b). In June 2024, for example, NIH submitted a report to Congress detailing its pediatric research activities in fiscal years (FY) 2021 and FY 2022 (NIH, 2024d). Another example is Environmental Influences on Child Health Outcomes (ECHO), which as of June 2024 consolidated data from over 107,000 participants, including over 64,000 children from many cohort studies that were harmonized. It also released these data in de-identified fashion and expanded access for use in research studies. It has generated over 1,700 publications and led to the development of interventional trial networks including support of Institutional Development Award states. It has a robust outreach and policy arm publicizing its achievements to the media and public. This was driven by support and funding that spurred a strategic planning and assessment operationalized by a program office in the Office of the Director, which led an infrastructure that combined all IC activities studying childhood environmental exposures (ECHO, n.d.-b, 2021, 2024; NIH, 2025a). Congressional mandates are thus one way that pediatric research and downstream impacts of chronic disease at NIH might be specifically enhanced.

THE NIH PEDIATRIC RESEARCH CONSORTIUM

In 2018, representatives from each of the NIH ICs created a collaboration known as the NPeRC, with the goal “to improve child health research collaboration and coordination” across its 27 ICs and offices (NICHD,

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⁴ Children’s Health Act, 2000, H.R. 4365, Public Law 106-310, 106th Congress (1999-2000).

2025a). NICHD is the lead IC for the consortium (NICHD, 2025a). NPeRC was intended to harmonize pediatric research activities across ICs, identify gaps within the NIH pediatric research portfolio, and take advantage of existing resources, infrastructure, and expertise (NICHD, 2025a). NPeRC does not receive any funding or resources, with engagement from representatives from each of the ICs on a volunteer basis. It does not directly support research projects or initiatives (NICHD, 2025a), nor does it have funding for any administrative support or other infrastructure supports. Although there are discrete examples where NPeRC was successful, it has been limited in its reach and effectiveness due to a lack of dedicated resources, formalized leadership structure, and the agency to enforce requirements for inclusion of children. As of October 2025, the NPeRC website listed 37 individuals representing 25 ICs, two research programs (i.e., All of Us and ECHO), and five offices (i.e., Office of Strategic Coordination, Office of Disease Prevention, Office of AIDS Research, Office of Behavioral and Social Sciences Research, Office of Research on Women’s Health). NPeRC members meet several times a year to evaluate ICs’ pediatric research portfolios, meet with American Academy of Pediatrics committee liaisons, develop workshops, evaluate research programs (including the NIH Clinical Center Pediatric Research Strategic Plan), discuss scientific opportunities and potential new areas of collaboration, and promote research training for the next generation of pediatrician–scientists and surgeon–scientists (see meeting agenda items in Appendix F). Since its inception, NPeRC has strived to (1) identify and address research gaps, (2) enable a nimble response to emerging threats to pediatric health, (3) promote greater efficiency and reach of ongoing initiatives through data harmonization, (4) spearhead novel partnerships, and (5) provide consultation to ambitious inter-IC initiatives. The progress made has the potential for much larger-scale success with adequate funding and infrastructure, as there is a need for dedicated staff, a communications platform, and an infrastructure to support collaborative pediatric-focused projects.

Addressing Research Gaps

NPeRC designated the transition from pediatric to adult health care, especially for youth with special health care needs, as a scientific priority area containing substantial knowledge gaps (Calabrese et al., 2022). The NPeRC Health Care Transition Subgroup conducted an analysis of NIH’s portfolio of research grants and scientific publications from 2007 to 2020 in order to “identify topics, research gaps, and opportunities in various chronic health conditions that affect child health and well-being for which transition research could synergize and expand scholarship” (Calabrese et al., 2022). NPeRC developed a definition of health care transition that

served as the framework for the coding of NIH-supported research (Calabrese et al., 2022). NIH ICs were then solicited to catalog NIH-funded grants related to transition. The ICs nominated 88 NIH-funded grants, and 60 were deemed to meet the inclusion criteria. Of these 60 grants, 20 were identified as gold-standard NIH-supported projects (Calabrese et al., 2022). Further searches, with adjudication by 11 experts, resulted in a final health care transition analysis database of 166 NIH-funded grants across 14 ICs. This included five ICs not previously identified as supporting transition research: NINDS, NIDCD, the National Institute on Minority Health and Health Disparities, NIAID, and the National Center for Complementary and Integrative Health (Calabrese et al., 2022).

The portfolio analysis culminated in a 2-day workshop in 2020 entitled Lost in Transition: Navigating Pediatric to Adult Healthcare, organized by NPeRC, NICHD, the Office of Behavioral and Social Sciences Research, and other NIH ICs (including NCI; the National Heart, Lung, and Blood Institute (NHLBI); NIDDK; the National Institute of Nursing Research; and NIMH). The workshop engaged experts from various disciplines, along with patients, families, and caregivers, to examine high-priority research topics related to services and supports that facilitate the transition from pediatric to adult care. The workshop helped identify themes, gaps, and opportunities to supplement ongoing NIH disease-specific work and promote collaboration with public and private organizations. After the workshop, NPeRC efforts to support research gaps in this area continued with a subsequent NOSI—initially supported by eight NIH ICs—entitled Navigating Pediatric to Adult Health Care: Lost in Transition (NOT-HD-21-027). The NOSI encouraged applications in “high-priority research areas related to pediatric health care transition for youth with chronic physical/medical conditions or intellectual or developmental disabilities with the goal of improving care quality and patient and family outcomes” during and after health care transition (NIH, 2021).

Responding to Emerging Threats in Pediatric Health

In March 2020, immediately after the start of the COVID-19 pandemic, NPeRC quickly established a working group with representation from 18 ICs, led by NICHD and NIDA, to facilitate an immediate response to the looming global health crisis (NICHD, n.d.-a). The Trans-NIH COVID Workgroup on Pregnant and Lactating Women and Children focus included examining the risk of infection and severity of disease (particularly in the presence of comorbid conditions) as well as investigating factors underlying mild illness, community transmission, and protective factors. Additional areas of emphasis included severe disease (e.g., multisystem inflammatory syndrome in children), return to school or daycare, and the social,

behavioral, economic, and environmental impacts of exposure (NICHD, n.d.-a). The working group coordinated funding strategies and other activities across NIH related to COVID-19 in pregnant and lactating people and in children (Bianchi, 2025).

In 2022, to facilitate additional and long-term research on the physical, mental, and behavioral health impacts of COVID–19 on children, NIH released two NOSIs: Emerging and Existing Issues of Coronavirus Disease 2019 (COVID-19) Research Related to the Health and Well-Being of Women, Children and Individuals with Physical and/or Intellectual Disabilities (NOT-HD-22-002); and Administrative Supplements for Research of Emerging and Existing Issues of COVID-19 Related to the Health and Well-Being of Women, Children and Individuals with Physical and/or Intellectual Disabilities (NOT-HD-22-003) (NIH, 2022a, 2022b).

The working group facilitated supplemental funding for projects in FY 2020 and FY 2021 amounting to more than $5 million total. These projects, funded by various ICs and the Office of the Director, included the following:

1. Augmenting a large cohort study including more than 10,000 children to examine pandemic-related perturbations in developmental trajectories of brain functioning, cognition, substance use, academics, social functioning, and physical and mental health (Jernigan, 2021; NIMH, 2022).
2. “A study assessing the impact of a universal intervention on the mental health of adolescent Black children during COVID-19 and identifying longitudinal multi-level risk and protective factors that predicted adolescent mental health during the pandemic” (Mendelson, 2021).
3. “Testing a coping intervention in preadolescent urban youth designed to prevent the onset of anxiety, depression, and post-traumatic stress symptoms in children facing chronic stress” (Wadsworth, 2020).
4. “A study to examine COVID-related changes in substance use and the relationships between substance use and psychosocial and other variables that may be affected by the pandemic among indigenous adolescents who live on or near reservations” (Stanley, 2021).

The NPeRC COVID-19 working group contributed to broader NIH initiatives facilitating the inclusion of children in the Rapid Acceleration of Diagnostics (RADx) programs and the Researching COVID to Enhance Recovery (RECOVER) effort (Horwitz et al., 2023; NIH, 2025c; RECOVER Initiative, n.d.). The RADx–RadicalSM program includes the Predicting Viral-Associated Inflammatory Disease Severity in Children with Laboratory Diagnostics and Artificial Intelligence (PreVAIL kIds) study, which

seeks to identify innovative diagnostic and analytic methods for examining factors that shape the diverse clinical manifestations of SARS-CoV-2 infection in children (Goff and Pearson, 2025).

Participation in the COVID-19 research efforts can be mutually beneficial for both the child volunteers and the NIH researchers. From the participant perspective,

Data Harmonization

When integrated across datasets, biomedical and psychosocial common data elements, measures, and terminology can enhance collective understanding of a specific research topic and improve the efficiency of ongoing NIH initiatives. Led by NPeRC, the NIH COVID workgroup coordinated “data and terminology harmonization efforts related to its populations of interest, including pregnant and lactating people, infants, and children” (NICHD, n.d.-a). NICHD convened representatives from the NIH Office of the Director, NIDA, NIAID, and its intramural and extramural programs to develop recommendations for a standardized set of biomedical and psychosocial common data elements and measures. Three working groups (Biomedical, Psychosocial, and Biospecimens) were formed with representation from more than eight intramural and extramural NIH-funded pregnancy cohort studies, including ECHO, NIH Helping to End Addiction Long-term (HEAL), and the Early Intervention to Promote Cardiovascular Health of Mothers and Children (ENRICH) program. Participating NIH ICs included 13 institutes, three centers (Fogarty International Center, National Center for Advancing Translational Sciences, and National Center for Complementary and Integrative Health), and the Office of Research on Women’s Health.

Pediatric Medical Device Development Through a Public–Private Partnership

Despite significant legislative, regulatory, and scientific efforts in recent years, innovative pediatric medical devices continue to reach the market at slower pace than adult medical devices (Reamer, 2022). Often this is due to the lack of incentives for the industry to research and develop pediatric

medical devices. Between 2011 and 2023, the Food and Drug Administration (FDA) Center for Devices and Radiological Health premarket approvals and humanitarian device exemptions that were labeled “only for adults” increased at about twice the rate of devices labeled for “both adult and pediatric use” and about 21 times the rate of devices labeled solely for pediatric use (FNIH, n.d.). This is a longstanding and significant health problem.

In response, NPeRC formed a Pediatric Medical Devices subgroup and sponsored a System of Hospitals for Innovation in Pediatrics Medical Devices webinar in partnership with the American Academy of Pediatrics, the FDA, the Critical Path Institute, and others (NICHD, 2021). In 2022, a request for information was published (NOT-EB-22-008) “to seek public input on research gaps, needs, best practices, innovative study designs and measurement, resources and data resources, and opportunities to inform a public–private partnership to enhance pediatric medical device development” (NIBIB, 2022).

The nonprofit Foundation for the National Institutes of Health (FNIH) launched the design phase of the public–private partnership in September 2023 with key federal partners including the FDA and the Biomedical Advanced Research and Development Authority (FNIH, 2023). Both agencies have provided scientific and regulatory insight and expertise as well as funding. Additional funding and expertise have been provided by the private sector, including industry and nonprofit organizations working through FNIH. Within NIH, NICHD and the National Institute of Biomedical Imaging and Bioengineering are leading this effort, with a number of other ICs engaged through NPeRC. Project goals include optimizing the translation of “technological advancements in medical device design, evaluation, and approval for pediatric populations”; minimizing risk; and streamlining processes to translate research on pediatric medical devices from the bench to the bedside (FNIH, 2023). The public–private partnership is designed to facilitate the successful development and commercialization of pediatric medical devices.

These projects highlight NPeRC’s potential to facilitate and coordinate pediatric research across NIH and beyond. As this report demonstrates, NIH can do even more to advance pediatric health in the United States, and the committee believes that NPeRC is an excellent model for getting more value from NIH-funded pediatric research. But to do that, NPeRC will need to be provided the financial resources and human expertise to enable it to mature and grow.

Conclusion 4-2: The National Institutes of Health Pediatric Research Consortium (NPeRC) has insufficient infrastructure, financial support, and representation of designated expertise in bioethics, patient advocacy, and communications. There is considerable potential for

expansion and formalization of NPeRC’s influence with adequate committed resources and leverage to support successful research across institutes and centers.

Recommendation 4-2: The National Institutes of Health (NIH) director should

1. Elevate the NIH Pediatric Research Consortium (NPeRC) (or its equivalent) to the Office of the Director, with membership including senior leaders within each institute or center (IC) with pediatric health and life course expertise who systematically review all relevant components of strategic plans, including progress and impact made in the past year. Each senior leader should then report back to their IC and to the NIH director.
2. Provide appropriate infrastructure funding and adequate resources (including personnel) to enable NPeRC (or its equivalent) to accomplish its goals and for its members to participate in its work, which includes (1) identifying and addressing research gaps, (2) enabling a nimble response to emerging threats to pediatric health, (3) promoting greater efficiency and reach of ongoing initiatives through data harmonization, (4) spearheading novel partnerships, (5) providing consultation to ambitious inter-IC initiatives, and (6) ensuring representation and visibility at national scientific meetings and societies.
3. Have NPeRC’s membership (or its equivalent) include expertise in pediatric bioethics. NPeRC should also partner with patient advocacy or community representatives and develop a communications plan to disseminate pediatric research findings to both the scientific community and the general public. NPeRC (or its equivalent) should be prepared to coordinate the NIH-wide response to any emerging pediatric research needs.

INTER-IC INITIATIVES: SUCCESSES AND CHALLENGES

Inter-IC pediatric health research initiatives are those supported by two or more ICs and/or the Common Fund within the Office of the Director (Common Fund, 2024). They are not necessarily supported or coordinated by NPeRC. These initiatives may be congressionally mandated or initiated by an IC that engages other ICs for support. Typically, one IC will manage the inter-IC project programmatically, and, in the case of Common Fund projects, one or more ICs are chosen to take the lead. While inter-IC initiatives begun by an IC are driven by the strategic plans of participating ICs, inter-IC studies initiated from the Common Fund reflect priorities across

NIH and emphasize transformative, catalytic, goal-driven, synergistic, and novel areas established by the NIH director with programmatic input. Another category of NIH-wide programs includes new initiatives from the NIH director. Historically, these have often fallen under the Common Fund but not always.

There is no NIH mandate or requirement per se to address or consider pediatric health through the vehicle of inter-IC initiatives. In the context of preclinical science, targeting pediatric-relevant health issues has been driven ad hoc by the topic studied. In these cases, scientific advancement has been largely driven by the solicited and/or submitted projects, especially because the relevant goals can be highly technical. For clinical and translational studies, a limited number of inter-IC initiatives has been successful in addressing pediatric health problems. Those that have succeeded tend to have several things in common (see Table 4-1).⁵ First, they ask scientific questions that are best answered by crosscutting, collaborative efforts among the appropriate ICs. Second, those scientific questions raise

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

NOTE: IC = institute and center; NIH = National Institutes of Health.

SOURCE: Synthesized from comments made by inter-IC initiative leadership at the committee’s March 13 webinar; see https://www.nationalacademies.org/event/44604_03-2025_opensession-part-1-strategies-to-enhance-pediatric-health-research-funded-by-nih.

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⁵ The characteristics that either facilitate or inhibit successful pediatric research within inter-IC initiatives have been synthesized from presentations and discussions with several NIH leaders at a public webinar on March 13, 2025. See https://www.nationalacademies.org/event/44604_03-2025_open-session-part-1-strategies-to-enhance-pediatric-health-researchfunded-by-nih for more information.

issues critically important to pediatric populations; moreover, they cannot be answered without inclusion of pediatric populations. Third, they include processes and procedures to establish pediatric-focused scientific infrastructure. Fourth, children are recruited and seen as integral to the study from the beginning. And, fifth, successful inter-IC initiatives receive sustainable funding. In contrast, other projects were limited by the inhibitors delineated in Table 4-1. NPeRC did not have sufficient resources or a mandate to overcome these barriers.

Conclusion 4-3: Facilitators of pediatric research initiatives include collaborative efforts from the start, a scientific premise and question that warrant pediatric representation, infrastructures for the conduct of pediatric research and pediatric institutions, inclusion of pediatric patients from the beginning of enrollment, and stable funding.

Recommendation 4-3: Initiatives within and across the National Institutes of Health (NIH) institutes and centers should coordinate with the NIH Pediatric Research Consortium (NPeRC) (or its equivalent) to include children and/or the downstream life course impact of childhood chronic disease, exposures, and experiences in studies in a strategic and deliberate way. NPeRC (or its equivalent) should support the enforcement of existing policies requiring that studies not exclude children or childhood downstream impacts on the life course without clear and adequate justification.

To close this chapter, the committee presents four inter-IC projects that highlight how these facilitators and inhibitors impact the successful advancement of pediatric health research.

Adolescent Brain Cognitive Development

One of the most successful cross-IC initiatives is the Adolescent Brain Cognitive Development (ABCD) Study. Established in 2015, ABCD was founded with an initial goal of fostering research on poly-substance use. Collaborators within the NIAAA, NIDA, and NCI came together with a shared understanding that to improve the risk factors and mitigate the consequences of substance use, it is necessary to first examine how childhood experiences influence childhood development (Dowling, 2025). ABCD uses multiple methods and data sources (e.g., neuro-imaging, neurocognitive testing, biospecimens, geocoded data), evaluates multiple exposures (e.g., environment, culture, prior physical and family health), and examines multiple outcomes (e.g., physical health, mental health, substance use) to achieve its goals (Hawes et al., 2025; NIMH, 2022).

ABCD has expanded beyond its original focus on substance use to meet the scientific interests of several additional institutes. NICHD participates to advance understanding of healthy brain development and traumatic brain injury. NIMH focuses on identifying factors that influence the course and severity of mental disorders that commonly emerge during adolescence and are associated with substance use. NINDS contributes by measuring how frequently sports injuries (e.g., concussions) take place during adolescence and to determine their impact on brain development. More recently, the NIH Office of Research on Women’s Health joined to examine sex and gender influences on adolescent brain development, and NHLBI was added to address issues including adolescent cardiovascular and hematologic health.

Today, ABCD is the largest long-term study of cognitive development and child health in the United States. The consortium infrastructure consists of a coordinating center; a data analysis, informatics, and resource center; and 21 research sites across the country. The project has included nearly 12,000 children who joined the study at the ages of 9–10 years (NIMH, 2022). Researchers track participants’ biological and behavioral development through adolescence and into young adulthood. Specifically, ABCD uses its multiple methods and technologies to determine how childhood experiences (e.g., use of videogames, sleep habits, tobacco use, substance use, and sexual activity) interact with each other to affect a child’s social, behavioral, academic, health, and other outcomes.

During a public information-gathering session, the director of the ABCD project, Dr. Gaya Dowling, reported that NPeRC played an important role in its success:

The impacts of ABCD have been substantial. The program has published approximately 1,300 manuscripts and has been cited in 83 policy papers and clinical guidelines worldwide (ABCD, n.d.). Its continued success is in large part because it has always used an open-data sharing model, acted on the numerous RFAs to use the data, and received sustainable funding from NIDA (NIH RePORT, 2025). ABCD has inspired and laid the

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⁶ The webinar recordings can be accessed at https://www.nationalacademies.org/event/44616_03-2025_open-sessions-part-2-strategies-to-enhance-pediatric-health-researchfunded-by-nih.

groundwork for other inter-IC projects, including the HEALthy Brain and Child Development (HBCD) Study (focused on early brain and child development from the prenatal period through age 9) (HBCD, n.d.). Together, ABCD and HBCD are elucidating numerous facets of cognitive, social, emotional, and physical development during childhood that would be difficult to detect from single-IC sponsored projects (Dowling et al., 2024).

Environmental Influences on Child Health Outcomes

Whereas ABCD started with a scientific question, other successful cross-IC endeavors started with a policy mandate. The ECHO program, for example, was mandated by the Cures Act of 2016⁷ with a mission to better understand the impacts of early environmental exposures on later child health and development (DPCPSI, 2024). Organizationally, ECHO is located within the Office of the Director. Like ABCD, ECHO addresses scientific questions of interest to multiple ICs, including pre-, peri-, and postnatal outcomes. Current priorities include lung diseases, obesity, neurodevelopment, and “positive health” behaviors (Gillman, 2025). Unlike ABCD, ECHO goes beyond an observational cohort study to also support a clinical trial network for child intervention research (ECHO, n.d.-a). RFAs to use ECHO’s cohort or trial network are sponsored by multiple ICs (NIH, 2024c).

To date, the ECHO cohort has included more than 107,000 participants, including people traditionally underrepresented in research such as members of rural and low-income populations (NIH, 2025a). The project’s impacts include approximately 2,000 publications, endorsements and adoptions of practice recommendations from state governments, and the development of influential public health guidelines (ECHO, n.d.-c). Despite these achievements, ECHO leaders highlighted several current challenges that the program faces: difficulties with research infrastructure, including capacity-building and data-harmonization across sites; changes in funding appropriations; and shifting federal priorities (Gillman, 2025). These challenges and uncertainties may impact the program’s continued success.

All of Us

The All of Us research program is an example of an NIH initiative that, to date, has fallen short of its potential to address child health. Funded in 2016, the program proposed a platform of precision medicine to “accelerate health research and medical breakthroughs, enabling individualized prevention, treatment, and care for all of us” (NIH, 2025b). Specifically, the

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⁷ 21st Century Cures Act of 2016, Public Law 114-255, 114th Congress, 1st Sess.

program argued that there is no one-size-fits-all approach to health care, and a large dataset representing diverse populations would better facilitate tailored, successful treatment plans.

At first glance, All of Us seems to have had the necessary elements of success. It was funded through federal appropriations from Congress as part of the Cures Act⁸ as well as through an additional base appropriation to the NIH Office of the Director, which is intended to offset fluctuations in Cures Act funding levels. Despite historical bipartisan support, however, funding for both Cures Act and All of Us base appropriations have diminished. Notably, the budget was cut by 34 percent in FY 2024 and by 71 percent in FY 2025. The result has been a substantial decrease in enrollment and data collection and, as noted on the All of Us homepage, delays in the “ability to build a robust pediatric cohort” (NIH, 2024a), making it difficult for researchers to recruit enough children to validly answer the study questions (NIH, 2024b).

These delays compounded existing challenges to including children in the All of Us study. Despite early recognition that precision medicine should include people across the life course, program leaders did not create pediatric-focused procedures when they launched the program’s infrastructure. It was not until the fifth year of funding (2021) that All of Us called for a pediatric director and not until the seventh year (2023) that the first pediatric participant was enrolled (Van Driest, 2025). Even after these delays in including children, some researchers suggested starting with older children and then gradually enrolling younger children over time. This strategy, known as age de-escalation, is a form of protection that is widely and sometimes reflexively used, even when it may not be warranted (Harbin et al., 2023). The All of Us study posed relatively low risk for children of all ages, so there was no valid reason to wait to enroll younger children in the study.

When the committee asked All of Us leaders why the program struggled to include pediatric patients, they answered by referring to the commonly used approach to clinical research: that adult-aged participants go first (Van Driest, 2025). This practice can be important when considering vulnerable subjects such as minors for research that entails some risk. In the All of Us study, however, this protectionist approach did not make sense because the study did not pose significant biomedical risks to any of the participants. All of Us compiled data from people in a range of ways, including through collecting biological samples and using wearable devices to collect data about local environments. Community consultation could have provided avenues for assessing whether data privacy was a significant concern for members of the public and, if so, how to mitigate these concerns.

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⁸ 21st Century Cures Act of 2016, Public Law 114-255, 114th Congress, 1st Sess.

Indeed, community-based and other stakeholder-type partnerships, be they between researchers and families or clinicians and advocates, play key roles in pediatric health care delivery and policy (Chung et al., 2022). Had the All of Us investigators worked with community representatives, they may have found that most parents support their family’s or child’s research participation (Kim et al., 2020; McCarthy et al., 2025). Moreover, they feel disappointed when clinicians and researchers make assumptions about their willingness or “vulnerability”; rather, they suggest that research participation is a critical way to amplify their voice and experience (McCarthy et al., 2025). While this community engagement is not unique to pediatrics or to All of Us, it is key when considering how best to support research that will inform pediatric patient and family outcomes.

Had there been a permanent structure within NIH focused on the benefits of research for children and adolescents and with the authority and resources to support pediatric enrollment, the repeated delays that resulted in near exclusion of children from the All of Us study might not have occurred. A strengthened NPeRC with such a mandate might serve as that structure.

The Patient-Reported Outcomes Measurement Information System Initiative

The Patient-Reported Outcomes Measurement Information System (PROMIS) was designed to create and validate self-report symptoms and quality of life measures that could be used to document the health status of people experiencing a variety of health conditions (Garcia et al., 2007). In 2001, Congress requested that the Institute of Medicine prepare a report on how to optimize collaborative science in the 21st century (Rettig, 2004). The 2003 report included recommendations for planning across NIH to identify ideas for multi-year research initiatives that could address crosscutting issues (IOM, 2003). The ensuing initial RFA did not specifically name pediatrics research as being eligible, but a strong application submitted by a pediatric-focused team was selected for funding along with several adult-focused submissions (Wang et al., 2018). The second PROMIS RFA that followed was informed by the strong evidence base for pediatrics that resulted from the first RFA.

Since then, PROMIS has been a formative force behind 300 patient-reported outcome measures as well as proxy measures developed, validated, and made readily and freely available for use in research and clinical care (Health Measures, 2025). PROMIS measures were the focus of 4,464 peer-reviewed publications between 2005 and 2025, of which 757 were pediatric in focus. The PROMIS measures are available in many languages and are

used globally, enabling multiple types of population and health comparisons to be made, which adds immeasurably to our knowledge about diseases.

As with other initiatives spanning NIH ICs, the facilitators of PROMIS are its collaborative design, attention to infrastructure, and sustainable funding (see Table 4-1). The early inclusion of pediatric-relevant scientific questions occurred by happenstance because a single team of pediatric-focused investigators responded to RFAs. Importantly, the inclusion of pediatric science and populations of children added considerably to the PROMIS program’s overall impact.

Pediatric- and adolescent/young adult–focused PROMIS measures have been incorporated into questionnaires for use in cooperative group clinical trials to document the impact of treatment for cancer and have been widely used across a variety of other adult and pediatric diseases to measure the impact of disease-specific treatments (Bense et al., 2023; Dahir et al., 2024; Dovern et al., 2023; Gershon et al., 2010; Hermans et al., 2023; Kliewer et al., 2023; Maurer et al., 2021; Reeve et al., 2017; Walters et al., 2025). These outcomes of the PROMIS adult and pediatric measures in research have increased their usefulness in clinical care in identifying health improvements in patients and in discovering profiles of the patients most in need of supportive care for symptom relief (Cheng et al., 2023; Schuchard et al., 2022; Thissen et al., 2016; Troost et al., 2019; Zigler et al., 2024). Furthermore, the existence of adult- and pediatric-specific PROMIS measures for the same symptoms occurring in the same disease in pediatric and adult patients has advanced our understanding of how a disease evolves across parts of the life course.

PROMIS has been successful in large part because it included considerations of the elements listed in Table 4-1. Its concept and design included collaboration across ICs; the scientific question and implementation recognized the necessity of including pediatric- and lifespan-inclusive populations; early implementation included consideration of pediatric research practices and procedures; and funding included plans for sustainability.

REFERENCES