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Tuesday, September 24
Tue, Sep 24, 4:15 PM - 5:30 PM
Innovative Trial Design and Analysis for Pediatric Drug Development

Striking a Delicate Balance: An Industry Perspective on Benefit-Risk as a Driver in Pediatric Design Innovation (301003)

Margaret Gamalo-Siebers, Eli Lilly and Company 
*Sarah Stanley, Eli Lilly and Company 

Keywords: Bayesian analysis, benefit-risk

The drive for innovation and efficiency from recent regulatory action has greatly impacted pediatric drug development as researchers strive to balance the need for information with the ethics of enrolling children, a vulnerable population. Innovative techniques such as extrapolation have come into focus as clinicians seek to reduce the need for pediatric exposure by leveraging prior information and developing analysis models that take this existing information into account. The ever evolving clinical and regulatory spaces demand an analytical perspective informed by the current pediatric landscape as well as a knowledge of designs that have been proposed in the past. The focus of this presentation centers on what can be learned from several case examples of recent pediatric trials. Specific attention will be given to the integral role that benefit-risk plays in the success of a design proposition and approval as well as in the types of innovative techniques that can be applied in the creation of an optimized, efficient trial design.