Considerations in pediatric trial designs and analysis
*Meehyung Cho, Sanofi 

Keywords: sample size, pediatric, James-Stein shrinkage estimator, empirical shrinkage estimator, Bayesian method, simulation

Pediatric trials are often conducted to obtain extended marketing exclusivity or to satisfy regulatory requirements. There are many challenges in designing and analyzing pediatric trials arising from special ethical issues and the relatively small accessible patient population. The application of conventional phase 3 trial designs to pediatrics is not realistic in some therapeutic areas. To address this issue we propose various approaches for designing pediatric trials that incorporate data available from adult studies including weighted combination methods such as James-Stein shrinkage estimation, empirical shrinkage estimation and Bayesian methods. We also apply the concept of consistency used in multi-regional trials to the design and analysis of pediatric trials. The performance of these methods is assessed through simulations.