The Coming of Age of Model-Informed Precision Therapeutics for Children

This article provides a summary of the Sumner Yaffe Award lecture presented at the annual meeting of the Pediatric Pharmacy Association in Portland, Oregon, on April 10, 2025. The article provides a short history of pediatric clinical pharmacology and elaborates on the ongoing paradox of precision medicine and the drug development process that is still geared toward the labeling of doses for the average patient. The article highlights contemporary clinical decision support approaches that integrate model-informed precision dosing (MIPD) to improve treatment outcomes in children. A series of case studies of successful implementation of model-informed clinical decision support tools is described including examples of full integration of such platforms into the electronic health record at Cincinnati Children's Hospital Medical Center. These studies show that pediatric precision dosing is progressing beyond the conceptual and proof-of-concept stages toward implementation into clinical workflows. The evidence provided indicates that model-informed therapeutic drug management for children is clinically feasible, with an increasing number of studies documenting improved clinical outcomes as compared with standard of care.