Considerations for Pediatric Trial Designs and Analyses

• Activity Number: 617
• Type: Contributed
• Date/Time: Wednesday, August 12, 2015 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Meehyung Cho* and Zhiying Qiu and Jenny Ye and Hui Quan and Peng-Liang Zhao
• Companies: Sanofi and Sanofi-Aventis and Sanofi and Sanofi and Sanofi
• Keywords: sample size ; consistency ; Bayesian method ; weighted combination ; simulation

Abstract

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.