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Activity Number: 304 - Extrapolation as a Default Strategy in Pediatric Drug Development: Implications to Clinical Trial Designs and Statistical Analysis
Type: Topic Contributed
Date/Time: Wednesday, August 5, 2020 : 10:00 AM to 11:50 AM
Sponsor: Biopharmaceutical Section
Abstract #313465
Title: Clinical Development Using Pediatric Extrapolation
Author(s): Robert "Skip" Nelson*
Companies: J7J
Keywords: extrapolation; inference; uncertainty; Bayesian; children; pediatrics

Providing children timely access to safe and effective drugs requires extrapolation to reduce the need for pediatric data. Extrapolation extends known experience into the unknown to draw credible but inherently uncertain inferences about the unknown area. The scientific and clinical assumptions behind using prior knowledge to draw conclusions about the efficacy of drugs in children will be explored. Extrapolation involves a value judgment about tolerable uncertainty appropriate for a pediatric development program. The tolerable uncertainty for approving a new drug is framed by the risk of being wrong across an entire population. The tolerable uncertainty for exposing a child to the risks of an investigational drug is framed by the risk/potential benefit to an individual child. The lack of safe and effective treatments for children with severe life-threatening disease argues for a tolerable uncertainty level closer to that of individual clinical decisions. The clinical and ethical issues concerning the use of extrapolation in pediatric drug will be illustrated conceptually through the use of Bayesian statistical methods and prior knowledge in model-based exposure-response analysis.

Authors who are presenting talks have a * after their name.

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