Improving sample size recalculation in adaptive clinical trials by resampling |
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Authors: | Carolin Herrmann Corinna Kluge Maximilian Pilz Meinhard Kieser Geraldine Rauch |
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Affiliation: | 1. Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, 10117 Berlin, Germany;2. Institute of Medical Biometry and Informatics, University Medical Center Ruprechts-Karls University Heidelberg, Heidelberg, Germany |
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Abstract: | Sample size calculations in clinical trials need to be based on profound parameter assumptions. Wrong parameter choices may lead to too small or too high sample sizes and can have severe ethical and economical consequences. Adaptive group sequential study designs are one solution to deal with planning uncertainties. Here, the sample size can be updated during an ongoing trial based on the observed interim effect. However, the observed interim effect is a random variable and thus does not necessarily correspond to the true effect. One way of dealing with the uncertainty related to this random variable is to include resampling elements in the recalculation strategy. In this paper, we focus on clinical trials with a normally distributed endpoint. We consider resampling of the observed interim test statistic and apply this principle to several established sample size recalculation approaches. The resulting recalculation rules are smoother than the original ones and thus the variability in sample size is lower. In particular, we found that some resampling approaches mimic a group sequential design. In general, incorporating resampling of the interim test statistic in existing sample size recalculation rules results in a substantial performance improvement with respect to a recently published conditional performance score. |
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Keywords: | adaptive group sequential design clinical trial resampling sample size recalculation |
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