Adaptive Designs for Confirmatory Clinical Trials with Subgroup Selection
- 2 January 2014
- journal article
- review article
- Published by Taylor & Francis Ltd in Journal of Biopharmaceutical Statistics
- Vol. 24 (1), 168-187
- https://doi.org/10.1080/10543406.2013.857238
Abstract
Growing interest in stratified medicine is leading to increasing importance of subgroup analyses in confirmatory clinical trials. Conventionally, confirmatory clinical trials either focus on a subgroup identified in advance or assess subgroup effects once the trial is completed. The focus of this article is methodology for adaptive clinical trials that both identify whether a treatment is particularly effective in a predefined subgroup, potentially enabling alteration of recruitment, and assess the effectiveness in the subgroup and/or whole population. Methods for such adaptive trials are described and compared, and the logistical and regulatory issues associated with such approaches are discussed.Keywords
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