From Policy to Practice: How the World is Supporting Pediatric Research

Clinical Researcher—August 2026 (Volume 40, Issue 4)

PEER REVIEWED

Tasha Mohseni

 

Pediatric research is essential to improving child health worldwide. However, scientific, ethical, and infrastructural challenges serve as barriers to this essential research. International and national stakeholders have developed coordinated strategies to strengthen evidence generation, enhance safety assessments, and accelerate therapeutic discovery for children. The World Health Organization (WHO) has articulated global pediatric research priorities to address evidence gaps and align clinical trials with public health needs. The U.S. Food and Drug Administration (FDA) and partner agencies are pioneering New Approach Methods (NAMs) to advance developmental safety assessments while reducing reliance on traditional animal models. Research policy advocates, such as the National Academies, emphasize unified research strategies and the integration of advanced technologies, such as artificial intelligence (AI), to transform pediatric oncology research and clinical trial design. Together, these policy frameworks are being translated into practice, fostering innovation and building infrastructure to close critical gaps in pediatric research and care.

Pediatric Research is Receiving International Attention

Children’s physiology, disease progression, and responses to therapies differ profoundly from those of adults, creating unique scientific and ethical challenges in clinical research.{1} Historically, pediatric evidence gaps have limited optimal care for children, leading clinicians to extrapolate adult data rather than rely on pediatric-specific evidence. Extrapolating adult data to pediatric data is problematic due to long-term problems that may result from medications only tested in adults.{2}

Global and national entities are now advancing coordinated policy frameworks and practical initiatives to support pediatric research, investment, and innovation. Initiatives range from clinical trial prioritization to novel safety assessment methods and emerging technologies such as AI.

WHO’s Vision for Pediatric Clinical Trials

The WHO has underscored the need for pediatric-focused research priority setting{3} and evidence generation to close persistent knowledge gaps. In its comprehensive report on pediatric clinical trials, WHO highlights stalled progress in reducing child mortality in select age groups and the underrepresentation of children in clinical research.

Through its initiative, WHO engages stakeholders globally to identify high-impact research questions and align trial design with pressing child health needs. WHO ensures that investments and research efforts generate evidence that can influence policy and clinical practice worldwide. Such global prioritization efforts guide funders, regulators, and researchers to concentrate on studies most likely to improve survival, growth, and development outcomes for children.

Building on these priorities, this global framework also promotes ethical considerations and methodological approaches appropriate for pediatric populations. It advocates for international collaboration to harmonize research agendas and share data across borders, ensuring pediatric research is not marginalized but embedded in overarching public health strategies, especially in low-resource settings where child health burdens remain high.

FDA Zeroes in on Pediatric Developmental Safety Assessments

A crucial element of advancing pediatric research is ensuring the safety and efficacy of therapies in children, which requires robust preclinical and clinical assessment strategies. Traditional juvenile animal testing has been the mainstay of developmental safety evaluation in pediatric drug development, but regulatory and scientific communities increasingly recognize its limitations, particularly when extrapolating results to pediatric human populations.

The FDA has responded to this challenge by convening workshops focused on NAMs{4} that use innovative technologies to assess developmental safety more effectively. These workshops, co-sponsored with partners such as the Triangle Center of Excellence in Regulatory Science and Innovation (Triangle CERSI),{5} bring together regulators, industry scientists, and academic researchers to explore alternatives that can complement or replace traditional methods for pediatric safety assessment. These alternatives include in vitro tools, computational models, and model-informed drug development techniques.

NAMs represent a shift toward integrating mechanistic, quantitative, and system-based approaches that may better account for pediatric physiological complexity. For example, leveraging physiologically based pharmacokinetic modeling,{6} quantitative systems pharmacology, and multi-omics data can improve predictive accuracy for pediatric toxicities and developmental effects that are difficult to capture with adult data or juvenile animal models alone. These methodological advances not only address regulatory requirements but also support more ethical and efficient research practices.

Prioritizing NIH-Funded Pediatric Research Initiatives

At the national level, cohesive research strategies can align priorities, funding, and resources across agencies to maximize impact. A recent National Academies report highlights the need for an agency-wide strategy at the National Institutes of Health (NIH) to better prioritize and fund pediatric research.{7} The report identifies longstanding structural impediments that can limit pediatric research visibility within broader NIH initiatives and emphasizes the importance of coordinated investment and strategy to improve child health outcomes. It recommends that NIH conduct a comprehensive review of its pediatric research portfolio, improve categorization processes, include pediatric components in larger initiatives, and coordinate across Institutes and Centers. This aligns with ongoing efforts such as the NIH Pediatric Research Consortium (N-PeRC),{8} which facilitates cross-agency collaboration and harmonization of pediatric research activities and training programs.

By fostering structural improvements and aligning funding mechanisms, these strategies aim to strengthen the research infrastructure that supports pediatric investigators. This ensures that pediatric research remains a visible and prioritized component of broader biomedical initiatives and sets the stage for leveraging advanced technologies in subsequent areas, such as oncology.

Harnessing AI for Pediatric Oncology

One of the most striking recent developments in pediatric research policy is the U.S. administration’s explicit emphasis on integrating AI to accelerate pediatric cancer research. In 2025, an executive order on “Unlocking Cures for Pediatric Cancer with Artificial Intelligence”{9} directed the Make America Healthy Again Commission and federal partners to develop innovative ways to utilize AI for improved diagnoses, therapies, and prevention strategies in pediatric oncology.

This emphasis on AI marks a pivotal shift in the capabilities available for pediatric cancer research and care. By leveraging rich, multimodal datasets, researchers can now identify novel biomarkers, optimize clinical trial design, and enhance predictive modeling of disease progression and treatment responses. This policy initiative also targets improved data infrastructure for AI-ready analysis, the integration of electronic health records and claims data, and the promotion of public-private collaborations that accelerate the translation of AI insights into clinical practice. Though promising, a major challenge to this approach is the limited data on rare cancers in children.{10}

Translating Policy into Trial Design and Conduct

However, policy advancements alone are insufficient without attention to how research is conducted at the operational level. To fully realize these strategic priorities, global and national directives must be thoughtfully integrated into traditional clinical research design and conduct. For example, clinical teams can incorporate AI tools for real-time data analysis and adaptive trial designs{11} to improve pediatric trial efficiency. These strategies enable better predictions of treatment response, identification of subpopulations, and optimization of safety monitoring. AI can also be operationalized in adaptive pediatric oncology trials by providing patient-specific treatment options{12} via predicting clinical outcomes, which guide these personalized therapies.{13} These means of operationalizing AI can potentially improve survival rates and quality of life.{14}

Teams can also use these technologies to improve recruitment and retention by precisely identifying eligible participants and supporting personalized risk-benefit communication. Similarly, sponsors and investigators can use innovative safety assessment methods developed through FDA workshops and regulatory guidance{15} to inform trial protocols. By adopting validated alternative methodologies, they can reduce reliance on juvenile animal models and improve translational relevance. As stakeholders accept these NAMs, sponsors and investigators can design pediatric research that better reflects developmental biology and pediatric physiology.

Ethical and Practical Considerations

A robust pediatric research landscape must also grapple with ethical and practical challenges. AI integration raises important considerations around data privacy, consent, and equitable access, given that the data would be received from a special (vulnerable) population. Policy frameworks{16} emphasize the need to protect individual privacy while enabling data sharing to improve research quality and clinical outcomes.

Research leaders must also ensure that diverse populations are represented in pediatric trials. Research strategies should prioritize inclusion across age, race, socioeconomic status, and geography to make findings generalizable and equitable.{17} Further, a U.K.-based study{18} found that with appropriate safeguards such as careful integration into clinical practice and supported parental decision making, large language models (LLMs) can enhance pediatric autonomy and comprehension. Training programs must equip pediatric researchers to use advanced methodologies and technologies for sustained progress.

Global and Cross-Sector Collaboration

Supporting pediatric research effectively also requires collaboration across sectors and borders. Global priorities set by WHO and other international partners encourage data sharing, harmonization of regulatory standards, and multisite clinical trial networks that can share resources and expertise. These collaborations not only accelerate evidence generation but also strengthen capacity in lower-resource settings where child health needs may be greatest.

Public-private partnerships among industry, academia, government, and patient advocacy groups are equally essential. Pooling knowledge and resources accelerates innovation, reduces duplication, and increases the likelihood that promising discoveries translate into approved therapies and improved standards of care.

We Are Entering a New Era

The landscape of pediatric clinical research is evolving from fragmented efforts into a more coordinated, technology-enabled, and strategically funded enterprise. Global priorities articulated by WHO, regulatory innovations from agencies such as the FDA, national strategic alignment through the NIH, and forward-looking policies promoting AI integration exemplify how policy can drive practical change.

These efforts collectively address longstanding barriers to pediatric research, creating an environment in which evidence generation for child health is prioritized and resourced. For clinical researchers, understanding these policy shifts and translating them into trial design, data practices, and interdisciplinary collaboration will be key to improving outcomes for children worldwide.

As we move forward, sustained commitment, investment, and thoughtful implementation of emerging methods and technologies will determine our success in closing pediatric evidence gaps and delivering better health for future generations.

References

1. U.S. Institute of Medicine Committee on Clinical Research Involving Children; Field MJ, Behrman RE, editors. 2004. Ethical Conduct of Clinical Research Involving Children. Washington, D.C.: National Academies Press. (Chapter 2) The Necessity and Challenges of Clinical Research Involving Children. https://www.ncbi.nlm.nih.gov/books/NBK25553/
2. Regulatory Affairs Professionals Society. 2023. Provider groups, researchers raise concerns about extrapolating adult data in children. Regulatory Focus. https://www.raps.org/news-and-articles/news-articles/2023/1/provider-groups-researchers-raise-concerns-about-e
3. World Health Organization. 2025. The future of paediatric clinical trials–setting research priorities for child health. https://www.who.int/publications/i/item/9789240116597
4. U.S. Food and Drug Administration. 2025. Pediatric developmental safety assessment with new approach methods. https://www.fda.gov/drugs/news-events-human-drugs/pediatric-developmental-safety-assessment-new-approach-methods-12052025
5. Triangle Center of Excellence in Regulatory Science and Innovation. 2025. Announcing NAMeRS: An Accelerator Program to Support the FDA Roadmap to Reducing Animal Testing. https://trianglecersi.org/news/namers-accelerator-program-fdaroadmap
6. Salerno SN, Carreño FO, Edginton AN, Cohen-Wolkowiez M, Gonzalez D. 2021. Leveraging Physiologically Based Pharmacokinetic Modeling and Experimental Data to Guide Dosing Modification of CYP3A-Mediated Drug-Drug Interactions in the Pediatric Population. Drug Metabolism and Disposition: The Biological Fate of Chemicals 49(9):844–55. https://doi.org/10.1124/dmd.120.000318
7. National Academies of Sciences, Engineering, and Medicine. 2026. National Institutes of Health Need Agencywide Strategy to Prioritize and Fund Pediatric Research, Says New Report. https://www.nationalacademies.org/news/national-institutes-of-health-need-agencywide-strategy-to-prioritize-and-fund-pediatric-research-says-new-report
8. Eunice Kennedy Shriver National Institute of Child Health and Human Development. NIH Pediatric Research Consortium (N-PeRC). https://www.nichd.nih.gov/research/supported/nperc
9. The White House. 2025. Unlocking cures for pediatric cancer with artificial intelligence. https://www.whitehouse.gov/presidential-actions/2025/09/unlocking-cures-for-pediatric-cancer-with-artificial-intelligence/
10. Hassan M, Shahzadi S, Kloczkowski A. 2025. Harnessing Artificial Intelligence in Pediatric Oncology Diagnosis and Treatment: A Review. Cancers 17(11):1828. https://doi.org/10.3390/cancers17111828
11. Badani A, de Moraes FY, Vollmuth P, Chung C, Mansouri A. 2025. AI and innovation in clinical trials. NPJ Digital Medicine 8(1):683. https://doi.org/10.1038/s41746-025-02048-5
12. MedTech Intelligence. 2025. AI and Functional Precision Medicine: A Defining Moment for Pediatric Cancer Care. https://medtechintelligence.com/viewpoint/ai-and-functional-precision-medicine-a-defining-moment-for-pediatric-cancer-care/
13. Bongurala AR, Save D, Virmani A. 2025. Progressive role of artificial intelligence in treatment decision-making in the field of medical oncology. Front Med. https://doi.org/10.3389/fmed.2025.1533910
14. Pediatric Cancer Research Foundation. 2024. Artificial Intelligence in Pediatric Cancer Diagnosis and Care. https://www.pcrf-kids.org/2024/04/12/artificial-intelligence-in-pediatric-cancer-diagnosis-and-care/
15. U.S. Food and Drug Administration. 2023. E11A: Pediatric Extrapolation. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/e11a-pediatric-extrapolation
16. Ibrahim AM, Abdel-Aziz HR, Mohamed HAH, Zaghamir DEF, Wahba NMI, Hassan GA, Shaban M, El-Nablaway M, Aldughmi ON, Aboelola TH. 2024. Balancing confidentiality and care coordination: challenges in patient privacy. BMC Nursing 23(1):564. https://doi.org/10.1186/s12912-024-02231-1
17. SAGE Publications. Inclusive Research Methodologies. https://learningresources.sagepub.com/research-methods/inclusive-research-methodologies#:~:text=Design%20and%20deliver%20research%20that,research%20through%20inclusive%20best%20practices
18. Allen JW, Earp BD, Wilkinson D. 2025. AI-assisted consent in paediatric medicine: ethical implications of using large language models to support decision-making. Journal of Medical Ethics. https://jme.bmj.com/content/early/2025/08/06/jme-2024-110624

Tasha Mohseni is President and Founder of RD Research Services LLC.