Statistical Considerations in Optimization of Pediatric Drug Development

• Activity Number: 398 - Considerations in Optimization of Pediatric Drug Development
• Type: Topic Contributed
• Date/Time: Tuesday, July 30, 2019 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Amy Xia* and Lynne Yao* and Fanni Natanegara* and Gary Noel*
• Companies: Amgen Inc and FDA/CDER and Eli Lilly and Company and Johnson & Johnson
• Keywords: Pediatric Extrapolation; ICH E11; Regulatory Science; Pediatric Clinical Development

Abstract

The addendum (R1) to ICH E11: Clinical Investigations of Medicinal Products in the Pediatric Population has been adopted by Committee for Human Medicinal Products (CHMP) in Sep. 2017 and come into effect in Feb. 2018. This addendum supplements the original ICH guidance adopted in 2000 by providing additional clarification and current regulatory perspectives on topics in pediatric drug development. Such topics include age classification, scientific and technical knowledge advances. The addendum clarifies extrapolation definition and then further details steps one should take and aspects one should consider in the extrapolation process. However, specifications, such as how statistical models can facilitate implementing this extrapolation concept in a drug development program, and when, where and how a statistician can contribute, leave a great room for deliberation. This panel session will use addendum ICH E11 (R1) as a venue to discuss key points in the modernized pediatric drug development program and dive into details of how they could be practiced in pediatric clinical trials. Both regulatory and industry representatives will join this panel session for an in-depth discussion.