ABSTRACT
The pediatric oncology community as well as regulatory bodies such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) agrees that pediatric patients should be treated with medicines that have been evaluated in children. In order to ensure timely access to agents that may be beneficial to children, pediatric studies should be incorporated into the product development plans of agents for which there is reasonable expectation of future pediatric use. The incentive did not require a positive pediatric study but rather simply the conduct of a phase I and II pediatric trial in exchange for a 6-month extension to all existing market exclusivities and patents for the drug moiety. The lack of pediatric formulations that allow dosage flexibility can lead to large differences between targeted and deliverable doses due to body surface area (BSA)-adjusted dosing employed in children. The use of BSA-adjusted doses provides an additional motivation for implementing model-based designs in pediatric dose-finding trials.
