Why Including Children in Clinical Trials Is Good Science: Rethinking Disease Study Design

Despite growing calls for pediatric inclusion, many clinical trials, especially in rare diseases, continue to exclude children or delay their enrollment until late stages. Yet, in many rare diseases, the point of maximal impact (PMI) occurs in childhood, when disease burden is highest and therapeutic windows are narrow. Excluding these patients not only delays access, but it also weakens trial validity, limits generalizability, and promotes off-label use in an evidence vacuum. This panel will challenge the outdated dichotomy of "adult-first" trial sequencing and instead propose scientifically grounded, ethically sound, and operationally feasible strategies for pediatric-first or concurrent inclusion. Attendees will leave with actionable strategies to confidently include pediatric patients in early-to mid-phase studies, supported by regulatory precedents, trial frameworks, and published outcomes.