Real-world data sources for pediatric cancers: a targeted literature review
TL;DR
Pediatric oncology RWD are numerous but concentrated in high-income regions and vary in completeness, yet core demographics and major tumor features are commonly recorded yet sociodemographic detail and many clinical outcomes are often missing.
Abstract
Background Pediatric cancers cause substantial childhood mortality and evidence gaps remain for rare tumors. Real-world data (RWD) can complement trials by providing long-term follow-up, broader populations, and external controls. We reviewed global pediatric cancer RWD to characterize sources and key data elements. Materials and methods PubMed was searched (February 2015-February 2025) for studies referencing cancer databases or initiatives collecting RWD on patients aged 0-25 years. After screening 1666 records, 225 articles yielded 134 unique data sources. We extracted database type, geography, population, tumor/treatment coverage, and availability of demographic, clinical, and outcome variables. Results Of 134 sources, 34.1% were pediatric-only. Most were in Europe (47.0%) or the Americas (18.7%). About 75% were population-based registries and ∼10% genomic/biobank resources. Age and sex were documented in >60% of sources; tumor site, histology, and stage in >50%; tumor grade in ∼30%; treatment modalities in 36%-40%; and survival in 61.2%. Race, medical history, and clinical outcomes (progression and response) were infrequently reported. Conclusions Pediatric oncology RWD are numerous but concentrated in high-income regions and vary in completeness. Core demographics and major tumor features are commonly recorded yet sociodemographic detail and many clinical outcomes are often missing. Improving data completeness, quality assessments, and representation from low- and middle-income regions will strengthen RWD utility for research and regulatory use.