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Activity Number: 481
Type: Topic Contributed
Date/Time: Wednesday, August 3, 2016 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract #319313 View Presentation
Title: An Extrapolation Framework to Specify Requirements for Drug Development in Children
Author(s): Gerald Hlavin* and Franz Koenig and Christoph Male and Martin Posch and Peter Bauer
Companies: Medical University of Vienna and Medical University of Vienna and Medical University of Vienna and Medical University of Vienna and Medical University of Vienna
Keywords: small population ; extrapolation ; prior belief ; adjustment of the significance level ; reduction of sample size
Abstract:

A fully independent drug development programme to demonstrate efficacy in small populations such as children may not be feasible and/or ethical, especially if information on the efficacy of a drug is available from other sources. In a Bayesian framework and under the assumption of a successful drug development in adults, we determine the amount of additional evidence needed in children to achieve the same confidence for efficacy as in the adult population. To this end, we determine when the significance level for the test of efficacy in confirmatory trials in the target populations can be relaxed (and thus the sample size reduced) while maintaining the posterior confidence in effectiveness. An important parameter in this extrapolation framework is the so called scepticism factor that represents the Bayesian probability that a finding of efficacy in adults cannot be extrapolated to children. The framework is illustrated with an example.


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