In typical clinical development programs, a new drug is first developed for the adult use. Drugs are often approved for adult use or in the process of obtaining approval in adults in the target indication before pediatric development is initiated. In designing the first pediatric clinical trial, one of the challenges is to select the initial dose to be tested. The ICH E11 R1 guidance advises that chronologic age alone may not always be the most appropriate categorical determinant to define developmental subgroups in pediatric studies. In this manuscript, the approaches to utilize available data in adults related to those factors beyond age to inform the starting dose selection in pediatric drug development are discussed. Practical considerations and approaches are provided for informing pediatric starting dose. Additional considerations to use pre-clinical information are provided in the case when adult information is limited or not available.
Keywords: Bayesian approach; Pediatric drug development; exposure-matching; pharmacokinetics; starting dose.