From gene therapy and AI to vaccines, nutrition, and cancer care, pediatric research leaders outline where the next decade of child health gains may come from, and what is needed to make them happen.
In a recent special article published in the journal Pediatric Research, a group of researchers identified the most promising areas of pediatric research and the key priorities needed to accelerate future improvements in child health.
Background
What breakthroughs will improve the quality of life for children over the next 10 years? For over 100 years, child health research has improved children's health through advancements such as vaccines for communicable diseases, improved infant care, better cancer treatments, and better management of chronic illnesses. Not only did these advancements help save many lives, but they also improved the quality of life. However, as public trust in science declines and research funding comes under threat, future progress may depend on identifying emerging scientific opportunities to invest in for the benefit of future generations. The findings also highlight the importance of sustaining investment in fields already identified by pediatric research leaders as high-potential.
About the study
Researchers surveyed members of the American Pediatric Society, research committee members of the Association of Medical School Pediatric Department Chairs, and the American Academy of Pediatrics to identify promising areas of child health research over the next five to ten years. Participants were also asked to identify priorities that could help advance pediatric research within the scientific community.
During the survey period between July and November 2025, researchers analyzed 66 faculty respondents’ answers, including those who provided more than the requested number of responses. A total of 192 responses addressed future research opportunities, while 182 responses focused on research priorities. Two independent large language models categorized the responses and identified the ten most frequently mentioned themes. These classifications were subsequently reviewed and confirmed by a human evaluator. Responses were classified into one or more thematic categories, which enabled researchers to identify the most frequently cited areas of pediatric research from the perspective of pediatric research leaders.
Study results
The survey highlighted recurring themes among pediatric experts regarding the scientific areas most likely to shape the future of child health. In fact, the most commonly identified areas of future promise were gene therapy, gene editing, and genomics. Advances such as whole-genome and whole-exome sequencing, and gene-based therapies, are improving diagnosis and creating opportunities to treat diseases at their biological sources rather than simply managing symptoms. Therefore, these technologies may offer disease-modifying or curative approaches for some lifelong conditions.
Mental health, behavioral health, and neurodevelopment ranked among the highest research opportunities. Researchers noted rising concern about childhood anxiety, depression, and developmental disorders. Early-life experiences have been shown to impact brain development and overall future well-being. Some expected future innovations include: earlier diagnosis; individualized treatment options and methods; and artificial intelligence-based tools to identify children at risk for developing significant symptoms of mental health and behavioral issues before they reach critical levels.
Immunizations and disease prevention through immunization have been achieved as major successes and opportunities for future achievement in healthcare. The development of COVID-19 vaccines, new strategies for maternal immunization, and the ability to protect infants against respiratory syncytial virus (RSV) have already improved outcomes for infants and children. However, the decline in vaccination rates and the subsequent rise in preventable diseases emphasize the importance of continuing public health programs to educate and promote immunization.
In addition, technology-based solutions, such as artificial intelligence, digital health, and health informatics, were identified as areas with the potential to significantly impact healthcare. More specifically, artificial intelligence is increasingly used for disease diagnosis, risk prediction, treatment planning, and improving healthcare efficiency. Future user applications may also involve integrating genetic, clinical, and environmental data for personalized medicine. However, the paper also emphasized the need for explainable, unbiased, ethically governed AI tools that can be integrated into clinical workflows without disrupting care.
Experts also emphasized neonatal and perinatal health, including fetal medicine. New technologies in prenatal screening, fetal surgery, regenerative medicine, and neonatology are increasing newborns' chances of surviving high-risk birth events while also shifting attention toward long-term neurodevelopment, function, and thriving. Research in precision medicine and targeted therapeutics similarly promises more individualized treatments based on genetic and biological characteristics.
Experts stressed the impact of social determinants of health on child health, health equity efforts, and addressing disparities in children's health outcomes. Housing, education, income, nutrition, and access to healthcare are major influences on child health outcomes. Community-level interventions and public policy changes are seen as vital to reducing health disparities.
Obesity, nutrition, and weight management also emerged among the promising research areas. New treatments, including glucagon-like peptide-1 (GLP-1) receptor agonists, have expanded options for managing childhood obesity, while personalized nutrition research may further improve health outcomes.
Environmental health research will continue to clarify how air pollution, climate change, and chemical exposures are affecting a child's growth and long-term health.
Lastly, advancements in pediatric oncology, including chimeric antigen receptor T-cell therapy, targeted treatments, and genomic profiling, continue to improve survival rates and quality of life for children with cancer.
Conclusion
The findings show that pediatric research is entering a period of remarkable opportunity. Experts recognized ten interconnected areas of research to improve child health, including genomics, artificial intelligence, mental health, environmental science, and cancer treatment.
All of these advances have the potential to assist in early diagnosis, effective treatments, and improved lasting outcomes for many children, provided barriers in funding, access, equity, implementation, and public trust are addressed. However, to accomplish these goals, long-term research funding, increased public confidence in science, workforce development, and continued support for pediatric research will be necessary.
Supporting pediatric research now will help ensure children have improved health, providing lasting benefits for future generations and adults.
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