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