617 – Methods in Clinical Trials 1
Considerations for Pediatric Trial Designs and Analyses
Meehyung Cho
Sanofi
Zhiying Qiu
Sanofi-Aventis
Jenny Ye
Sanofi
Hui Quan
Sanofi
Peng-Liang Zhao
Sanofi
Pediatric trials are often conducted to obtain additional exclusivity or satisfy the regulatory requirement. There are challenges in pediatric trial designs and analyses including some ethical issues and relatively small size of a pediatric trial. The application of the regular well-powered phase 3 trial design to a pediatric trial may not be realistic. In this presentation, we first review regulatory guidance and existing research in the area. We then examine different approaches for designing a pediatric trial and analyzing data. We consider the bridging trial concept in the sense that if the observed treatment effect in pediatric patients is consistent to that in adult patients, the confirmed results in adult population can be extrapolated to pediatric population. We also consider Bayesian approach using partial information from adult results as priors, a weighted combination method utilizing all available data including external data in the same therapeutic area. The performance including the required sample sizes of these methods will be assessed through computation or simulation.