Session (CTI)

Purpose Children are often not included in randomized trials, leaving major evidence gaps for many medications used in pediatric care. Off-label prescribing is common across pediatric specialties, which increases the need for reliable real-world evidence (RWE) to inform safety and benefit–risk decisions. However, generating high-quality pediatric RWE is challenging because of low event rates, changing treatment patterns, and limited pediatric-specific clinical information in many datasets. This session aims to provide practical strategies to improve the design, analysis, and interpretation of pediatric RWE studies and to discuss how these approaches can support regulatory and clinical decision-making. Description Panelists will outline how to evaluate whether a real-world data source is fit for use in pediatric studies, including assessing completeness of variables, follow-up, capture of off-label use, and the ability to define exposures, covariates, and outcomes relevant to children. The session will describe approaches for addressing confounding and bias when sample sizes are small or covariates are imbalanced, such as overlap weighting. Examples from pediatric studies of biologics and other widely used therapies will illustrate how these methods work in practice, especially when studying rare safety outcomes such as serious infections. The session will also consider how strengthened RWE can inform safety assessment for off-label pediatric use and complement existing regulatory frameworks for benefit–risk evaluation. Panelists will highlight the importance of transparency, reproducibility, and clear communication of uncertainty when estimates are imprecise. Attendees will gain practical guidance for evaluating data fitness, selecting appropriate analytical methods, and interpreting findings in settings where traditional trial evidence is limited or unavailable. The discussion will emphasize actionable steps to support pediatric RWE generation.