Progress Pulse

Global gaps in access to pediatric cancer clinical trials

Margit Mikkelsen

Margit Mikkelsen, MD, Department of Oncology, led a global analysis that quantified gaps in access to pediatric cancer clinical trials in resource-limited countries.

Children and adolescents in countries with limited resources face reduced access to clinical trials for cancer, despite accounting for 80% of new pediatric cancer cases annually. An analysis of 5,645 trials from the World Health Organization’s International Clinical Trials Registry Platform found that over 80% of pediatric oncology clinical trials and patient enrollment occur in highly resourced countries. Trials in resource-limited countries (sometimes called low- and middle-income countries or LMICs) are fewer, single-institution efforts and focus more on supportive care than cancer-directed therapies.

“For years, we’ve suspected that children in LMICs lack comparable access to clinical trials, but that gap had never been systematically quantified,” said first author and St. Jude clinical fellow Margit Mikkelsen, MD, Department of Oncology. “By quantifying these disparities, we move from assumption to evidence, with concrete data to act on.”

Notably, the study found that investigators in countries with limited resources still publish trial results at rates comparable to those in well-resourced countries, highlighting that the gaps in trial access are caused by opportunity, not capability. The findings, published in JAMA Network Open, underscore the urgent need to expand trial infrastructure in countries where resources are limited to improve care, reduce gaps in care and broaden the impact of pediatric oncology research globally.

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