Clinical trials are essential to improving outcomes for children and adolescents with cancer, but access and participation remain uneven around the world. A new cross-sectional analysis published in JAMA Network Open offers the most comprehensive global picture to date of where paediatric oncology clinical trials are conducted, what types of studies are available, and how equitable access is across countries of differing income levels.
Using the World Health Organization’s International Clinical Trials Registry Platform (ICTRP), researchers identified 5 645 interventional paediatric oncology clinical trials registered through May 8, 2024, capturing studies that enrolled patients younger than 18 years with an active cancer diagnosis. The analysis provides a detailed breakdown of trial characteristics, geographic distribution, intervention types, and sponsorship patterns.
CLINICAL SUMMARY
What was examined
A global cross-sectional analysis of registered interventional clinical trials evaluating where and how paediatric oncology studies are conducted worldwide, using data from the WHO International Clinical Trials Registry Platform.
Key findings
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More than half of registered paediatric oncology trials were paediatric-only, with over 80% sponsored in high-income countries.
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Low-income countries accounted for a negligible proportion of paediatric oncology trials, despite bearing a substantial share of the global childhood cancer burden.
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Trials conducted in lower- and middle-income settings were more likely to focus on supportive care rather than cancer-directed interventions.
Clinical implications
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Unequal access to clinical trials may contribute to persistent global disparities in childhood cancer outcomes.
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Expanding research infrastructure and international trial networks in resource-limited settings could improve access to evidence-based care.
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Greater geographic diversity in paediatric oncology trials may enhance the relevance and generalisability of future research findings.
Where trials are conducted
Of the 5,645 interventional trials identified, 3,149 (55.8 %) were pediatric-only studies (those exclusively for children and adolescents). Among these, an overwhelming majority — 81.2 % — were sponsored in high-income countries (HICs). Upper-middle-income countries hosted 12.5 % of paediatric-only trials, lower-middle-income countries 8.9 %, and low-income countries only 0.2 %.
North America dominated the landscape, with most trials accruing patients there, followed by Asia and Europe. Countries with the highest number of trials included the United States, Canada, Australia, France, and the United Kingdom — all high-income settings.
Trial types and phases
Across paediatrics-only trials:
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70.5 % were treatment-oriented studies.
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26.3 % focused on supportive care.
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3.2 % were diagnostic interventions.
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Early-phase trials (phase 1 or phase 1/2) accounted for about 32.7 %, with later-phase studies (phase 3 and beyond) making up about 17.4 %.
Trials conducted in lower- and middle-income settings were disproportionately supportive-care focused, and were less likely to be multi-institutional or include cancer-directed treatments compared with those in high-income countries.
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Across all groups, just under half of paediatric trials had results published in peer-reviewed literature, and supportive care and later-phase trials were more likely to be published within 10 years of registration.
Global inequities in paediatric oncology research
This study highlights persistent global inequities in paediatric oncology research. While the majority of childhood cancers occur in low- and middle-income countries, the vast majority of clinical trials remain concentrated in wealthier nations, limiting opportunities for patients and constraining the generalisability of research findings.
Access to clinical trials has been a driver of improved survival in childhood cancer in high-income settings over the past five decades. Systematic and sustainable efforts to expand research infrastructure and collaborative networks in resource-limited settings could help improve care and outcomes for children with cancer worldwide.
Additionally, the concentration of trials in certain geographies and the limited presence of later-phase, cancer-directed studies outside high-income settings underscore the need for targeted support, funding mechanisms, and policy frameworks that facilitate broader participation and equity in paediatric oncology research.
Paper: Mikkelsen MK, et al. The Global Clinical Trial Landscape for Children and Adolescents With Cancer. JAMA Netw Open. 2026;9(1):e2552510. doi:10.1001/jamanetworkopen.2025.52510. Access online here.
