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Activity Number: 647
Type: Contributed
Date/Time: Thursday, August 8, 2013 : 8:30 AM to 10:20 AM
Sponsor: Biopharmaceutical Section
Abstract - #309685
Title: Evaluating Regression to the Mean of Treatment Effect from Phase II to Phase III
Author(s): Jianliang Zhang*+
Companies: Medimmune Inc.
Keywords: treatment effect shrinkage ; regression-to-the-mean ; pre-phase II portfolio
Abstract:

It is commonly observed in drug development that the treatment effect demonstrated in phase III trials is generally smaller than what was observed in the preceding phase II trials. Potential causes for this shrinkage in treatment effect have been discussed in the literature, and various approaches have been proposed to account for the phenomenon in phase III trial planning (e.g. raising the Go criteria or discounting phase II results). Instead of qualitative factors in trial design and conduct, in this paper, we focus on evaluating and quantifying the size of the impact of the pre-phase II portfolio on the shrinkage. The impact varies by the quality of the pre-phase II portfolio and the size of the phase II program. For reasonable portfolio assumptions with commonly used phase II sample sizes, it is found that the expected shrinkage of treatment effect in phase III is non-negligible. In fact, the majority of the observed shrinkage is explained by the pre-phase II portfolio, and should be taken into account when planning phase III trials.


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