Multiplicity in confirmatory clinical trials: a case study with discussion from a JSM panel |
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| Authors: | Frank Bretz Alex Dmitrienko Jason Hsu Gary Koch Willi Maurer Walt Offen Robert O'Neill |
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| Affiliation: | 1. Novartis, Basel, Switzerland;2. Quintiles, U.S.A.;3. Ohio State University, Columbus, OH, U.S.A.;4. University of North Carolina, NC, U.S.A.;5. AbbVie, IL, U.S.A. |
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| Abstract: | An invited panel session was conducted in the 2012 Joint Statistical Meetings, San Diego, California, USA, to stimulate the discussion on multiplicity issues in confirmatory clinical trials for drug development. A total of 11 expert panel members were invited and 9 participated. Prior to the session, a case study was previously provided to the panel members to facilitate the discussion, focusing on the key components of the study design and multiplicity. The Phase 3 development program for this new experimental treatment was based on a single randomized controlled trial alone. Each panelist was asked to clarify if he or she responded as if he or she were a pharmaceutical drug sponsor, an academic panelist or a health regulatory scientist. Copyright © 2015 John Wiley & Sons, Ltd. |
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| Keywords: | accelerated approval biomarker endpoint decision theoretic multiplicity in regions primary and key secondary endpoints |
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