Rosane Charlab1, Ruby Leong1, Stacy S. Shord1 and Gregory H. Reaman2 1Office of Clinical Pharmacology, Office of Translational Sciences, U.S. Food and Drug Administration, Silver Spring, Maryland 20993, USA 2Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, Maryland 20892, USA Correspondence: Rosane.CharlabOrbachfda.hhs.gov

The discovery and development of anticancer drugs for pediatric patients have historically languished when compared to both past and recent activity in drug development for adult patients, notably the dramatic spike of targeted and immune-oncology therapies. The reasons for this difference are multifactorial. Recent changes in the regulatory landscape surrounding pediatric cancer drug development and the understanding that some pediatric cancers are driven by genetic perturbations that also drive disparate adult cancers afford new opportunities. The unique cancer-initiating events and dependencies of many pediatric cancers, however, require additional pediatric-specific strategies. Research efforts to unravel the underlying biology of pediatric cancers, innovative clinical trial designs, model-informed drug development, extrapolation from adult data, addressing the unique considerations in pediatric patients, and use of pediatric appropriate formulations, should all be considered for efficient development and dosage optimization of anticancer drugs for pediatric patients.