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A Statistical Framework for Decision Making in Confirmatory Multipopulation Tailoring Clinical Trials

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Abstract

This article focuses on statistical analysis of clinical trials pursuing tailored therapy objectives, wherein evaluation of treatment effect occurs in the overall population as well as in a predefined subpopulation(s). The design and analysis principles presented provide a framework for decision making based on these novel multipopulation tailoring trial designs, considering the particular case of confirmatory trials. These principles include traditional multiple testing considerations, as well as 2 new analysis principles.

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Authors and Affiliations

  1. Lilly Research Laboratories, Eli Lilly and Company, Lilly Corporate Center, Indianapolis, IN, 46285, USA

    Brian A. Millen PhD, Stephen Ruberg PhD & Lei Shen PhD

  2. Quintiles, Overland Park, KS, USA

    Alex Dmitrienko PhD

Authors
  1. Brian A. Millen PhD
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  2. Alex Dmitrienko PhD
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  3. Stephen Ruberg PhD
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  4. Lei Shen PhD
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Corresponding author

Correspondence to Brian A. Millen PhD.

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Millen, B.A., Dmitrienko, A., Ruberg, S. et al. A Statistical Framework for Decision Making in Confirmatory Multipopulation Tailoring Clinical Trials. Ther Innov Regul Sci 46, 647–656 (2012). https://doi.org/10.1177/0092861512454116

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  • DOI: https://doi.org/10.1177/0092861512454116

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