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Evaluation of inferential methods for the net benefit and win ratio statistics

Johan Verbeeck, Brice Ozenne, William N. Anderson

2020Journal of Biopharmaceutical Statistics25 citationsDOI

Abstract

General Pairwise Comparison (GPC) statistics, such as the net benefit and the win ratio, have been applied in clinical trial data analysis and design. In the literature, inferential methods based on re-sampling, asymptotic or exact methods have been proposed for these GPC statistics, but they have not been compared to each other. In this paper, the small sample bias of the variance estimation, Type I error control and 95% confidence interval coverage of the GPC inferential methods are evaluated using simulations. The exact permutation and bootstrap tests perform best in all evaluated aspects for the net benefit, while the exact bootstrap test performs best for the win ratio.

Topics & Concepts

StatisticsPairwise comparisonConfidence intervalSample size determinationResamplingMathematicsPermutation (music)Type I and type II errorsExact statisticsComputer scienceAcousticsPhysicsStatistical Methods in Clinical TrialsOptimal Experimental Design MethodsStatistical Methods and Inference
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