Keywords: Pediatric, regulatory, strategy
The challenges in the pediatric drug development are mainly due to the special characteristics of the pediatric patient population and the considerations in protecting this population. These challenges manifest in the clinical trials as limited sample sizes and a lack of suitable control groups, which in turn affect the subsequent data analyses and efficacy interpretation. The challenges present opportunities for innovative thinking and strategies. The complexity of the overall pediatric drug development program requires statisticians to play an essential role throughout the study planning, trial design, and efficacy evaluation. Strategies that emerge include enrolling adolescents in the adult trials, leveraging of existing adult data in the pediatric trials to minimize the unnecessary risk exposure to pediatric populations, sharing a common control group within the framework of master protocol, applying real-world-data as the control when a concurrent control is not ethical or feasible, and much more. In this presentation, strategies at different development stages of pediatric trial will be discussed and case examples will be presented.