Abstract
Advances in pediatric research have improved the lives of infants, children, adolescents, and society at large. A decade ago the American Academy of Pediatrics identified seven research achievements in the past 40 years that reduced morbidity and mortality worldwide: preventing disease with life-saving immunizations, reducing sudden infant death with “Back to Sleep,” curing Acute Lymphoblastic Leukemia, helping premature babies breathe with surfactant, preventing Human Immunodeficiency Virus transmission from mother to baby, increasing life expectancy for children with Sickle Cell Anemia and Cystic Fibrosis, and saving lives with car seats and seat belts. This follow-up article forecasts the next great achievements in pediatric research and highlights what is needed to continue progress.
The pediatric scientific community identified 10 areas of high research promise: genomics; mental and behavioral health; vaccines; artificial intelligence and digital health; perinatal health, including fetal medicine; precision and targeted therapeutics; social determinants of health; nutrition; environmental health; and advances in cancer treatment. Advances in each of these areas are already improving health outcomes. Amid increasing public distrust in science, celebrating scientific progress and committing to continued investment are critical to improving the health of children and the adults they will become.
Impact
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Advances in pediatric research have improved the lives of infants, children, adolescents, and society at large.
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The pediatric scientific community identified 10 exciting pediatric research breakthroughs on the horizon, which are reviewed in this article.
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Amid increasing public distrust in science, celebrating scientific progress and committing to continued investment are critical to improving the health of children and the adults they will become.
Introduction
The last century has witnessed dramatic decreases in global mortality and improvements in the quality of life. Research discoveries translated to clinical and public health practice and policy have improved the health of populations across the life course. Despite this success, distrust in medicine and science has increased in the United States, and there have been threats to continuing the investment necessary to support progress.1,2 Historically, the US has been the global leader in spending on medical research; however, funding has flattened in the past decades, and peer nations are beginning to invest more.3
This paper expands on the campaign led by the American Academy of Pediatrics (AAP) Committee on Pediatric Research that recognized seven great achievements in pediatric research of the previous 40 years (Table 1),4 and predicted the next great pediatric research achievements.5
Now, a decade later, in an environment of medical and scientific distrust, it is important to honor the research successes of the past and highlight cutting-edge research of high promise for the future. Our goal was to identify the 10 most promising areas of pediatric research and the 10 highest priorities to advance pediatric research. Although forecasting the future is inherently challenging, insights from research leaders anticipating future scientific advances are instructive in sustaining continued investment and envisioning breakthroughs in health and medicine.
Methods
Members of the American Pediatric Society and the research committee members of the Association of Medical School Pediatric Department Chairs and the AAP were surveyed for their top three promising child health research topics over the next 5–10 years and their top three priorities for advancing child health research within the pediatric community. This yielded 192 responses for promising research areas and 182 responses for priorities to advise research.
The survey was conducted between July 2025 and November 2025. Analysis was performed by considering each respondent’s response (some supplied more than the 3 requested responses) as independent responses. Two different large language models identified the same top 10 topic areas from the responses (Tables 2 and 3); these topic areas were subsequently confirmed by a human reviewer (TAG). Each response was then mapped to one or more of the group topics by the large language models; the mapping was validated by the human reviewer (TAG).
Results
Responses were received from 66 faculty members. There were 192 responses to the query about the top three most exciting and promising research topic areas important for child health in the next 5–10 years. Overall, 82% (158/192) of the responses mapped into the top 10 categories. 21 responses fit into two of the top 10 categories, resulting in 179 counts across all top 10 categories, forming the denominator for the percentages in Table 2.
There were 182 responses to the query about respondents’ top priorities for advancing child health research within the pediatric community. Overall, 84% (152/182) of the responses mapped into the top 10 categories. 28 responses fit into two of the top 10 categories, resulting in 180 counts across all top 10 categories, forming the denominator for the percentages in Table 3.
Research achievements and promising opportunities (Table 2)
Genetics, genomics, gene editing and gene therapy
Recent developments in genetics, genomics, gene editing and gene therapy are revolutionizing pediatric medicine. A recent meta-analysis found that for the diagnosis of suspected genetic conditions in children, the pooled diagnostic yield for whole-genome sequencing (WGS, 38.6% (95% CI: [32.6–45.0])) and whole-exome sequencing (WES, 37.8% (95% CI: [32.9–42.9])) were significantly higher than usual care, 7.8% (95% CI: [4.4–13.2]).6 For non-acute neurodevelopmental disorders, studies have confirmed high diagnostic yields, along with demonstrating cost-effectiveness in identifying genetic causes.7,8 Similar high diagnostic yields for WGS and WES were found for critically ill infants; importantly, these results were available in just days, leading to important changes in immediate management and substantial healthcare cost savings.9,10,11,12 As such, these innovations carry the potential to address root causes before irreversible harm occurs with one-time, disease-modifying, or curative treatments.
Prenatal and newborn metabolic screening have revolutionized early diagnosis, and the incorporation of genomic testing has the potential for even greater impact. Noninvasive prenatal testing (NIPT) uses next-generation sequencing to analyze cell-free DNA from maternal blood to screen for common chromosomal conditions. In 2022, the estimated proportion of pregnant persons who used NIPT in the U.S. was 49%.13
Early diagnosis enhances management and the effectiveness of gene therapy and gene editing, particularly for severe childhood disorders caused by single-gene mutations. Since 2016, gene therapies are currently approved for a variety of pediatric disorders, including but not limited to sickle cell disease, Duchenne muscular dystrophy, spinal muscular atrophy, dystrophic epidermolysis bullosa, acute lymphoblastic leukemia (ALL), cerebral adrenoleukodystrophy, transfusion-dependent β thalassemia, acute hepatic porphyrias, primary hyperoxaluria type 1, and adenosine deaminase deficient-severe combined immunodeficiency, along with numerous additional clinical trials underway in other conditions.14 These advances offer new hope for patients with previously incurable genetic conditions, which collectively represent a significant portion of chronic pediatric illnesses.
Over the next decade, the integration of WGS with personalized gene therapies can shift pediatric care from symptom management to curative interventions. Additional research will discover technologies that improve gene therapy precision while reducing off-target effects and identify long-term outcomes of gene-based therapies. Achieving this vision will require updated regulatory frameworks, scalable manufacturing solutions, ethical considerations, and financial models that support therapies for small patient populations, along with those from low-resource environments. Research is essential to understand the safety, durability, ethical considerations, and long-term outcomes of gene-based therapies.
Discoveries that result in reliable functional interpretation of non-coding variants, structural variants, repeat expansions, and regulatory elements will significantly reduce “variants of uncertain significance (VUS),” provide better risk prediction for common diseases, while helping improve diagnostic yield in rare diseases and neurodevelopmental disorders. Research into the use of genomic sequencing in newborn screening will lead to an era of polygenic risk scores that can enhance our prevention-focused care with new actionable information. As studies of the relationship between epigenetic biomarkers and stress, environmental exposure, and disease progression become available, the link between genetics and environment will become clearer, which will provide objective methods of examining new approaches to reducing negative influences on health.
Lastly, the use of advanced artificial intelligence (AI) models that accurately forecast gene function, pathogenicity, and therapeutic response have the potential to facilitate better understanding of disease mechanisms, reduce the time and cost of drug and gene therapy development, enhance clinical care through improved clinical decision support tools, and reduce disparities in access to genomic expertise.
Mental health, behavioral health, and neurodevelopment
Significant increases in the occurrence and disability resulting from mental health disorders in children and adolescents have been noted both in the US and globally for the past 3 decades.15,16 In 2021, a national emergency in child and adolescent mental health was declared by the AAP, American Academy of Child and Adolescent Psychiatry, and the Children’s Hospital Association, with a call to action to address unmet clinical needs.17,18
Concurrently, ongoing research in pediatric mental health, behavioral health, and neurodevelopment has profoundly deepened our understanding of how the developing brain is shaped by early experiences—experiences that leave lasting imprints on a person’s mental health and overall well-being.19 When key developmental processes such as synaptogenesis, circuit refinement, and myelination are disrupted by genetic factors, inflammation, trauma, nutrition, or stress, the resulting alterations in neuronal architecture and connectivity can fundamentally change how an individual perceives and interacts with the world. A recent study of 9082 participants in the Adolescent Brain Cognitive Development study found that MRI scans of 9- and 10-year-old children exposed to various adverse experiences (e.g., prenatal risk factors, interpersonal adversity, household economic deprivation, and neighborhood adversity) had changes in their white matter microstructure throughout the whole brain, and that this was associated with lower performance on mental arithmetic and receptive language.20
While early life is a period of heightened sensitivity, adolescence emerges as a second critical window, during which essential skills, such as executive function and emotional regulation, reach maturity. Data from the Bucharest Early Intervention Project revealed that higher quality caregiving in adolescence was associated with greater reward responsivity, executive function, and lower levels of internalizing and externalizing symptoms, supporting the idea that adolescence is a potential window of recovery opportunity from early life adversity.21
By elucidating risk factors and identifying these pivotal developmental windows, research paves the way for prevention strategies, early diagnosis, and the design of personalized interventions that can redirect developmental trajectories before disruptive disorders become entrenched. The convergence of neuroscience, psychology, psychiatry, immunology, and systems biology is now enabling the prediction of risk, early detection of pathology, and the tailoring of interventions across the continuum of development.
Major research opportunities exist that would allow us to either move upstream in addressing disease development and progression or significantly improve current therapies to address this growing worldwide crisis. Research on early recognition, prevention, and intervention of behavioral and mental health disorders (including those in chronic disease and community-based populations) could prevent disorders from either (i) progressing to diagnosis or (ii) escalating in severity level beyond outpatient care. This approach could include the use of advanced computational research (including AI and machine learning (ML)) to develop tools for early recognition and to identify patients on a trajectory toward (i) a diagnosis of a behavioral or mental health disorder or (ii) escalation of existing disorders. A second opportunity is research focused on developing, implementing, and disseminating evidence-based behavioral and mental healthcare into clinical practice and across the care continuum (in the community and academic settings). Creating advanced behavioral, pharmacological, and device therapies for complex disorders with an emphasis on anxiety and depressive disorders is needed. Lastly, enhancing mental and behavioral health basic science efforts will complement clinical research efforts and other more discovery science approaches (e.g., gene therapy, brain organoid research). This could include cohort genetic/genomics/epigenetics studies of mental health disorders, preclinical studies utilizing novel genomic therapeutic approaches, neuroimaging biomarkers of disease and response, and pharmacogenetic studies of new medications.
Vaccines, immunization, and infectious disease prevention
Vaccination and other infectious disease prevention efforts continue to significantly impact child health worldwide. Highly effective COVID-19 vaccines for children based on novel mRNA platforms played a key role not only in decreasing COVID-19 illness and hospitalizations and allowing children to return to school, but also in protecting against long COVID symptoms.22,23,24,25 Additionally, advances in maternal vaccination strategies during pregnancy provide substantial protection for vulnerable neonates against COVID-19 and Respiratory Syncytial Virus (RSV). Infants whose mothers were vaccinated against COVID-19 infection during pregnancy are less likely to be hospitalized during their first 6 months of life.26 For the first time, RSV protection is available to all infants via maternal vaccination with a bivalent RSV prefusion F protein-based vaccine during pregnancy, or via the long-acting monoclonal antibody nirsevemab for all infants less than 8 months old.27,28,29 It is critical to support ongoing work to promote maternal vaccination during pregnancy through public health measures and partnerships with obstetric providers.
The positive impact of childhood vaccines introduced in prior decades continues to grow across the world. Human papillomavirus (HPV) vaccines were introduced for children and adolescents in the US beginning in 2009. Dramatic decreases in the frequency of HPV disease, such as cervical abnormalities and anogenital warts are already apparent, with long-term improvements in cervical and other HPV-related cancers anticipated in the decades to come.30 Similarly, conjugate pneumococcal vaccines led to declines in potentially lethal Streptococcus pneumoniae meningitis, bacteremia, and other invasive infections across the world.31,32,33,34 Collectively, these interventions have contributed to longer life expectancy, fewer disability-adjusted life years, and improved population-level child health resilience, underscoring vaccination and prevention as among the most impactful pediatric public health advances. Unfortunately, the clear benefits of immunization are further emphasized by the increase in measles and other preventable infections in outbreaks across the US with decreasing vaccination rates.35 Ongoing community engagement and advocacy for evidence-based vaccination programs are needed to promote vaccine confidence and reduce barriers to immunization access for all children.
Artificial intelligence (AI), digital health, and informatics
Pediatric care is being transformed by AI, big data analytics, and digital health tools. These technologies enable predictive analytics, personalized therapeutic planning, and improved operational efficiency.36 AI-driven decision support systems can enhance prediction, diagnosis, classification, risk stratification, prognosis, and treatment. Studies have shown AI technology can assist in the early detection of late-onset sepsis and necrotizing enterocolitis (NEC) in infants in the NICU,37 screen for retinopathy of prematurity from retinal images,38 and adjust insulin delivery based on real-time patient data.39 Since 1995, 149 (17%) of the 876 AI/ML-enabled devices with FDA approval are authorized for pediatric use, with radiology 80%, neurology 9%, and cardiology 7% dominating the focus.40
Clinical workflows benefit from automation of tasks such as documentation, prior authorization, and patient information management. Studies demonstrate that AI models are able to accurately forecast pediatric emergency department overcrowding and also significantly optimize the physician shift schedule.41,42 However, several challenges must be addressed to fully realize the benefits of AI. These include seamless workflow integration, improved explainability and interpretability, identification and elimination of bias, workforce education and training, and greater engagement of clinicians, patients, and families.
Over the next decade, continued advances in AI and broader acceptance will yield more precise and reliable predictions, diagnoses, and treatment recommendations. These solutions will emphasize explainability, minimize bias, and enhance outcomes with minimal disruption to clinical workflows. Achieving these goals will require robust governance frameworks, ethical safeguards, and sustained investment in clinician education and infrastructure.
The impact of AI-specific research on future pediatric care will be widespread. Incorporating longitudinal, age-specific data that documents normal developmental trajectories will lead to algorithms that can distinguish between transient variation, divergence, or delay, facilitating earlier and more accurate detection of autism, learning disorders, ADHD, mental and behavioral health disorders, and mental health crises. Development of AI algorithms that integrate data from wearables, smartphones, and home environment sensors will enhance data accuracy, provide objective information on factors that impact mental health, and provide a real time surveillance system for health conditions. In the clinic, AI algorithms that integrate clinical data, genomic data, imaging findings, and environmental data will provide personalized risk prediction and treatment options that will improve care in acute and chronic conditions such as epilepsy and asthma. As electronic health records utilize AI algorithms more, learning health systems will become more efficient and impactful, providing greater practical insights for providers and families.
Neonatal/prematurity, perinatal health, and fetal origins of disease
An expanding body of evidence indicates that preconception, in utero, and neonatal factors contribute to the risk of childhood and adult disease, underscoring the need for sustained research and programmatic investment in maternal and child health. While substantial progress has been made in perinatal and neonatal survival, attention is increasingly shifting from surviving to promoting thriving, with an emphasis on long-term neurodevelopmental and functional outcomes and a life course perspective.43,44 In addition, earlier risk assessment and antenatal interventions have made great progress.
Perinatal prevention, including maternal vaccination and passive immunity offer maternal, child, and family protection as cited above. Prenatal and perinatal diagnostics have included biomarkers and predictive modeling for preterm birth, fetal growth restriction, preeclampsia; improvements in prenatal imaging and fetal assessment; use of fetal genomics and NIPT; and risk stratification in perinatal management.45
Advances in fetal surgical and interventional techniques have led to in utero interventions to correct or mitigate congenital anomalies before birth, aiming to improve survival, reduce morbidity, and optimize long-term outcomes. These interventions include fetal surgery clinical trials for myelomeningocele,46 congenital diaphragmatic hernia,47 congenital heart disease,48 tumor resection, and fetal thoracoamniotic shunting to drain large pleural effusions or cystic lung lesions, and minimally invasive fetoscopic techniques like laser ablation for twin-to-twin transfusion syndrome49,50 and fetoscopic endoluminal procedures for congenital lung lesions.51 Research explores regenerative approaches, artificial wombs, and fetal gene editing (CRISPR) to expand treatment options. Though there are ethical, regulatory, and societal considerations to manage, science has pushed the limits to identify and address risks earlier in the life course.
In neonatology, the gestational limits of viability have been challenged with the discovery of enhanced respiratory and cardiopulmonary support. This has included the use of surfactant highlighted in the previous “7 Great Achievements” as well as noninvasive ventilation strategies, lung-protective ventilation, and prevention of bronchopulmonary dysplasia, pulmonary vasodilator therapy, and management of neonatal pulmonary hypertension and extracorporeal life support. Innovations in nutrition, microbiome, and growth have contributed to improved outcomes and reduced complications such as NEC.52,53 Additional therapies, including precision and targeted therapies, anti-inflammatory and immunomodulatory treatments, and cell-based and regenerative therapies, are undergoing clinical trials.
Finally, neuroprotection and brain health research has advanced therapeutic hypothermia and adjunctive neuroprotective strategies, seizure detection and management, neuroimaging and electrophysiologic monitoring innovations, and long-term neurodevelopmental follow-up and outcome prediction.54 AI is expected to further advance these therapies.
In the US, pregnancy-related deaths increased from 2018 to 2022, with large variations by state and race and ethnicity.55 Neonatal and infant mortality rates have declined substantially over the last six decades, but significant racial disparities in mortality and other important outcomes persist.56 While the epidemiology of these inequities has been well described, research on family-centered and systems-based care innovations is of high priority. Expanding the reach of old and new innovations globally remains a goal for all children to benefit.
Precision/Personalized medicine and targeted therapeutics
The concept of precision or personalized medicine has existed for thousands of years.57 The reality of delivering it accelerated with the discovery of DNA, identification of CYP enzymes, and the success of the human genome project, and then its first appearance as a scientific term in 1991. Recent advances in genetics, pharmacology,58 AI, environmental science,59,60 and molecular biology have significantly enhanced diagnostic accuracy, facilitated tailoring of treatments, and improved outcomes. As many childhood diseases have strong genetic or biologically distinct drivers, precision medicine can conclude challenging diagnostic odysseys and guide early interventions to prevent irreversible morbidity while reducing overall healthcare costs. Examples include gene therapy and CRISPR-based gene editing61 that address the underlying cause of pediatric genetic disorders, pharmacogenetic testing that can optimize medication selection and dosing in psychiatry,62 model-informed dosing that improves outcomes in multiple conditions (e.g., immunosuppression, oncology, inflammatory bowel disease)63, and rapid WGS in critically ill newborns that helps make diagnoses within days. Molecularly targeted treatments and immunotherapies, e.g., chimeric antigen receptor (CAR)-T therapy, have revolutionized survival in pediatric oncology.64,65 These breakthroughs illustrate the clinical power of matching therapies to underlying biology.
There are multiple transformative opportunities for future personalized medicine and targeted therapies. Clinical decision support tools created using advanced AI techniques could integrate a patient’s comprehensive medical history with their WGS, transcriptomics, proteomics, metabolomics, and environmental exposures for early diagnosis, risk prediction/stratification, or guide therapy selection or dosing. In utero gene editing techniques could be used to cure life-threatening fetal genetic diseases. Personalized immunotherapy/cell therapy will expand past pediatric oncology into neurologic, inflammatory, and metabolic disorders.
Social drivers, health disparities, and health equity
International studies indicate that the public’s highest priorities for scientific research include improving public health and reducing poverty.66 Scientific breakthroughs in health disparities have focused on understanding root causes, quantifying inequities, and developing targeted, scalable interventions that integrate social, biological, technological, and policy approaches to improve equity in health outcomes and address social determinants of health (SDH).
The World Health Organization broadly defines SDH as “the conditions in which people are born, grow, live, work and age and people’s access to power, money and resources” which are a powerful contributor to health inequities.67 Advancement in data tools and ample research have demonstrated differences in health outcomes by income, education, housing, race, and environment, with recognition that social conditions shape health. Research has also demonstrated that early life conditions (prenatal care, nutrition, toxic stress, early education, family support) have lifelong health impacts.68 Further, early-life adversity has been shown to influence immune function, metabolism, epigenetics, and neurodevelopment, demonstrating how adversity gets “under the skin” and providing a biological explanation for disparities in chronic disease.69,70,71
While there has much description of health disparities, there is growing research on community-based and place-based interventions, clinical integration of SDOH screening, and addressing upstream systems and “Health in All Policies,” recognizing that transportation, housing, education, labor, and environmental policies are health policies. Community-based participatory research and community-driven solutions that leverage local knowledge, trust, and power-sharing have shown promise to improve health outcomes.68,72 For example, community health worker models integrated in well-child care have demonstrated improved outcomes in limited trials.73 Targeted programs addressing housing, food insecurity, and education demonstrate measurable improvements in child health outcomes. There is strong and growing evidence that stable housing and family economic supports improve physical and mental health.74,75,76,77,78 Research shows that policies like Medicaid expansion, paid parental leave,79,80 child tax credits,81,82 housing assistance,83 and upstream value-based investments are associated with improvements in child health and reduced healthcare costs. Again, expanded dissemination and implementation research, institutionalization of successful models, and public health and policy-based solutions for children across the globe would qualify as research and advocacy great achievements.
Obesity, nutrition, and weight management
The prevalence and severity of obesity in children and adolescents have steadily increased over the past three decades. The current prevalence in the United States is approximately 21%.84 This trend is also present globally, with over 8% of 5–19-year-olds having obesity.85 There are likely multiple causes for this increase, including unhealthful changes in diet and physical activity for children over time. Obesity in childhood is associated with a wide range of comorbid conditions, including hypertension, dyslipidemia, diabetes, and liver disease, as well as increased likelihood of obesity in adulthood. In adulthood, obesity is associated with increased risk of cardiovascular disease, certain cancers, end-stage liver disease, and other major adverse medical conditions.
Until recently, there were few, if any, effective pharmacologic treatments for childhood obesity. Clinicians were limited to either behavior-change therapy or bariatric surgery. However, with the advent of glucagon-like peptide-1 (GLP-1) receptor agonist agents, the therapeutic approach to obesity has changed dramatically.86
The GLP-1 story is instructive as this class of medications was developed from basic science research, leading to a drug class that was directed at treating type 2 diabetes mellitus. As more was learned about this class of agents, it became clear that they had broader effects, including weight loss and cardiovascular risk reduction. This demonstrates that development of effective therapeutics is not linear and requires both basic and translational science to achieve ultimate goals.
Future work is clearly needed to understand the role of GLP-1 medications and other therapeutics for obesity in a population that is growing and developing. Key interest will focus on the potential loss of muscle mass, along with fat mass, in individuals treated with these medications.
Nutrition is an important element of overall health beyond the development of obesity. Historically, research in nutrition has focused on which components make a diet healthful across the population. In pediatrics, research has focused on optimum nutrition at different stages of development. More recently, there has been increased recognition of the concept that individuals may differ in metabolism, microbiome, and other factors that may impact the relationship between nutrition and health. This has produced questions about precision or personalized nutrition, which will be an important pediatric research direction for the future. Understanding precision nutrition will require a multi-omics and trans-omics approach to identify biomarkers and possibly genetic-environmental interactions to better understand the impact of different nutrition interventions.
Environmental health, climate change, and exposures
The influence of early-life environmental exposures on lifelong health has become increasingly clear over the past decade. Large longitudinal cohorts and advanced techniques to map exposures and the resulting changes in the growth and development of children have clarified the impact of air pollution, endocrine-disrupting chemicals, heavy metals, and climate change. For example, the National Institutes of Health-funded Environmental Influences of Child Health Outcomes cohort demonstrated associations between prenatal and early-childhood exposures to fine particulate matter, pesticides, phthalates, and per- and polyfluoroalkyl substances (PFAS) with asthma, preterm birth, neurodevelopment changes, and obesity.87,88,89,90 Dissecting these complex interactions requires large-scale, multisite longitudinal studies to identify specific modifiable risks for child development and to inform changes in public policy.
Climate change also introduces several risks for child health, including the direct effects of extreme weather on chronic diseases such as seizures or kidney disorders, and indirect effects via food insecurity, reduced physical activity, and particulate effects associated with wildfire and other events.91,92 Temperature changes and habitat destruction also alter the potential spread of tropical and zoonotic infectious diseases to vulnerable pediatric populations. These include Dengue, Zika, Chikungunya, and Malaria, which have increased in frequency due to expanding mosquito activity.93,94,95 Similarly, the epidemiology of tick-borne illness is changing as tick habitats expand, requiring a high index of suspicion for clinicians as infections emerge in new areas. The increased risks associated with climate change will disproportionately impact children with chronic disease, living at a lower socioeconomic status, or with other potential inequities.96 Importantly, the impact of chemical and environmental exposures in early life has the potential to not only impact child health but also lifelong wellness. Children must be a particular focus in future studies within the “One Health” paradigm to understand the interactions between human, animal, and environmental health.97
Cancer, cancer treatment, and immunotherapy
The transformational power of pediatric research is arguably best demonstrated in the dramatic improvements observed in childhood cancer outcomes.98 Large-scale genomic profiling efforts, such as the NIH-funded Therapeutically Applicable Research to Generate Effective Treatments and Pediatric Cancer Genome projects, are defining the molecular drivers of leukemias, brain tumors, sarcomas, and other pediatric malignancies.99,100,101,102 These findings not only allow more targeted risk stratification and diagnosis but also identify new targets for therapeutic development. Precision therapies now include BCR-ABL inhibitors for Philadelphia chromosome-positive leukemia, ALK inhibitors for neuroblastoma, and BRAF and MEK inhibitors for select pediatric brain tumors.103,104,105 Concurrently, expansive cooperative clinical trial networks such as the Children’s Oncology Group drive gains in survival such that overall childhood cancer survival in the US now exceeds 80% for many malignancies.106
Most notably, CAR T-cell therapy has transformed the treatment of relapsed or refractory pediatric ALL, producing durable remissions in children with otherwise fatal disease. CAR-T therapy involves the genetic modification of a patient’s own T cells to express synthetic receptors that recognize tumor-specific antigens, enabling targeted immune-mediated destruction of cancer cells. In pediatric oncology, the most significant impact has been observed in B-cell acute lymphoblastic leukemia (B-ALL), where CD19-targeting CAR-T cells have achieved unprecedented remission rates in children who had exhausted all conventional treatment options. Early-phase clinical trials demonstrated complete remission rates exceeding 70% even in heavily pretreated pediatric patients, leading to the first FDA approval of a CAR-T therapy (tisagenlecleucel) for children and young adults with relapsed or refractory B-ALL in 2017.107,108 These advances, many emerging from federally funded translational research and early-phase pediatric trials, represent a paradigm shift toward biologically driven, less toxic cancer care for children.
Hand-in-hand with increased survival, progress in survivorship research has also reshaped pediatric cancer care to address long-term health and quality-of-life outcomes. Childhood cancer survivors are at an increased risk of mortality decades beyond their initial diagnosis, due to both disease and treatment effects, and other factors still unknown. Many late effects of cancer therapy have been described, including cardiotoxicity, secondary malignancies, neurocognitive impairment, and endocrine dysfunction, leading to evidence-based survivorship guidelines and risk-adapted treatment de-intensification.109,110,111 However, further work is needed to understand cancer survivorship as increasing numbers of pediatric cancer survivors reach adulthood with decades of lifespan and potential children of their own ahead. Together, these advances have shifted U.S. pediatric oncology toward a comprehensive model that emphasizes cure, functional recovery, and long-term well-being.
Pediatric community priorities to advance child health research (Table 3)
Table 3 shows responses to the question: What are three things that the pediatric community can and should focus on to promote the child health research agenda? Advocacy, public awareness of the importance of science, and research funding were top responses. The responses can generally be classified into two categories. The first category includes responses that speak to the infrastructure that supports child health research and how to improve that infrastructure. This includes improved funding for pediatric research, training, and career development for physician-scientist research careers and collaboration to improve the quality of research. The second category includes specific areas of child health research that should be a focus of future research. These areas include vaccines, health equity, and access to healthcare, neurodevelopment, and prevention of neurodevelopmental disorders. Responses that might be included in both categories are public education, combating misinformation, and the need for strong research advocacy.
Discussion
Acknowledging great scientific achievements of the past, this paper outlines exciting research breakthroughs as well as needed areas of advocacy to spread discoveries and accelerate progress. The emergence of new research tools provides opportunities to speed scientific discovery, including the use of electronic health records, advanced big data analytics, multi-omics approaches, AI, and novel functional and structural imaging modalities.5
When reviewing the 10 promising research topics, it is clear that there is substantial overlap. New technologies in genetics and omics are being applied across many research areas. Research on the microbiome or genomics is relevant to many body systems and may contribute to developing precision therapeutics. Future approaches to research related to child health should take advantage of the overlap in these most promising research areas. It is likely that advances in one domain will be applicable to other domains and across the lifespan. While this survey highlights 10 promising areas in pediatric research, it is not an exhaustive list, as there are additional areas beyond these 10 that are poised for major scientific progress and impact (e.g., regenerative medicine and organoids, pediatric pharmacotherapeutics).
The promising areas identified highlight the life course aspect of disease development and the importance of exposures and early disease processes in the fetus, newborn, child, and adolescent on adult health outcomes. It can be argued that, because of the lifelong impact of pediatric health and early disease development, investment in child health research will ultimately have the highest societal return on investment.112 Allocation of research resources should include strong consideration of return on investment across the life course.
Progress in the 10 areas identified has arisen from previous scientific investment in research involving all four phases of translational research, including (T1) basic science discovery, (T2) clinical or population efficacy, (T3) effectiveness trials, and (T4) implementation and dissemination, and health services and policy research to improve individual and population health.113 To realize the promise of the 10 areas requires funding and support to maintain and accelerate momentum and to fully translate science to practice and policy. However, we often take for granted the research progress of the past. Recently, U.S. federal support for biomedical research has been seriously challenged. Child health research has been historically underfunded compared to research on adult health issues, even in the best economic circumstances.114 It is more important than ever that research on child health issues receive an equitable share of available resources.
Areas of needed advocacy and enabling infrastructure to support research were also identified (e.g., funding, workforce development, advocacy). Career pathways supporting the development of the next generation of pediatric researchers have been threatened by recent changes in federal research policy and public distrust in science, occurring against a backdrop of decades-long declines in the proportion of physicians pursuing research careers.115 Attention must be given to the range of factors that hinder careers in child health research, including concerns about the long-term sustainability of funding across different career stages and lower salaries in pediatrics and pediatric research.116,117,118
A decade ago when the 7 Great Achievements was originally published, Chung et al. warned of partisan scientific bias, public unease regarding scientific accuracy, the “replication crisis” in research, and concern about a profitable “healthcare-industrial complex.”119 The importance of conducting and disseminating research within an environment that values and respects the scientific process and avoids conflicts of interest cannot be overstated. Additional research is needed to better understand how misinformation and distrust emerge and to identify effective preventive interventions. Addressing distrust and increasing transparency in the scientific process are urgent priorities for advancing research success. Effective communication of research findings to the public and policymakers remains a critical component of sustained progress.
The 10 promising areas in child health research provide stories and hope to guide advocacy efforts to enhance child health research. It is essential to highlight success stories in research. For example, a mother with sickle cell disease who experienced multiple hospitalizations for pain crises in childhood now has a child with the same condition who has never had a pain crisis, as a result of research demonstrating the effectiveness of penicillin and hydroxyurea therapy or curative genetic treatments. Children with the most common childhood cancer, ALL, are also part of a success story. Because of research, the 5-year survival rate for children diagnosed with ALL has increased from 57% in 1975 to 92.3% in 2014–2020.120 Advocating for the resources and public engagement needed for continued research is essential to address old and new health challenges and to maintain and advance health starting early in the life course. It is a time of great research promise. The next chapter of great achievements in science has yet to be written.
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Cheng, T.L., Daniels, S.R., Snowden, J. et al. Advancing child health: forecasting the next great research achievements. Pediatr Res (2026). https://doi.org/10.1038/s41390-026-05126-w
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DOI: https://doi.org/10.1038/s41390-026-05126-w
