Regulatory and scientific challenges in paediatric oncology

INTRODUCTION: Pediatric oncology poses distinct scientific, ethical, regulatory, and commercial challenges compared to adult oncology. Although survival rates for certain childhood malignancies have improved significantly, progress in relapsed, refractory, and rare pediatric cancers remains limited. AREAS COVERED: A comprehensive literature search was performed using major biomedical databases, including PubMed, Scopus, and Web of Science. Historically, pediatric drug development followed adult approvals, resulting in significant delays in access to innovative therapies. Recent pediatric regulatory reforms have shifted requirements from indication-based approaches toward mechanism-of-action-based evaluation, promoting earlier and more biologically relevant pediatric investigation. This report examines the biological differences between pediatric and adult cancer populations, reviews the evolution of pediatric oncology from a regulatory perspective, identifies the principal scientific and operational barriers limiting progress, and outlines strategic pathways to accelerate the development of safe and effective therapies for children with cancer. EXPERT OPINION: Despite meaningful therapeutic advances, pediatric oncology drug development remains constrained by small patient populations, biological divergence from adult malignancies, long-term safety considerations, and limited commercial incentives. Overcoming these challenges will require global collaboration, regulatory alignment, innovative trial design, and a deliberate focus on child-specific tumor biology rather than extrapolating from adult oncology paradigms.