The Global Clinical Trial Landscape for Children and Adolescents With Cancer

The improvement of childhood cancer outcomes has relied on clinical trials, primarily from high-income countries (HICs). Inconsistent access to clinical trials, especially in low-income countries (LICs) and middle-income countries, contributes to disparate care and limits the generalizability of conclusions from trials conducted in HICs.

To describe the global availability of clinical trials for children and adolescents with cancer in different economic settings.

In this cross-sectional study, the World Health Organization's International Clinical Trials Registry Platform was used to search for clinical trials on May 8, 2024. Trials were included if they were interventional studies for pediatric patients (<18 years old) with an active cancer diagnosis.

Clinical trials were analyzed based on geographic location, World Bank country income classification, and trial characteristics, including study phase, sponsor type, intervention type, and inclusion criteria. The main outcome was differences between HICs and low- and middle-income countries in the availability, design, and results of clinical trials for children and adolescents with cancer.

Of 138 595 oncology trials, 5645 met the inclusion criteria. Of these, 3149 (55.8%) were pediatrics-only trials. Most pediatrics-only trials (2558 of 3149 [81.2%]) were conducted by sponsors in HICs. Among identified trials, 12.5% (394 of 3149) were open for accrual in upper-middle-income countries (UMICs), 8.9% (281 of 3149) in lower-middle-income countries (LMICs), and 0.2% (7 of 3149) in LICs. Compared with HICs, LMICs or LICs registered fewer early-phase trials (28.8% [42 of 146] vs 81.4% [1709 of 2100]; P < .001), trials with cancer-directed treatments (17.6% [46 of 262] vs 76.8% [1964 of 2558]; P < .001), and multi-institutional trials (6.5% [17 of 262] vs 54.2% [1386 of 2558]; P < .001). LMICs or LICs registered more supportive care-based trials (81.7% [214 of 262] vs 19.9% [509 or 2558]; P < .001). Supportive care trials, later-phase trials, and trials from UMICs, LMICs, and LICs were more likely to publish clinical trial results within 10 years.

In this cross-sectional study of global pediatric oncology clinical trials, low- and middle-income countries were underrepresented. These data illustrate the need to improve the clinical research infrastructure in low- and middle-income countries and foster inclusive collaborations to promote equity in global pediatric cancer clinical trials.