Generalized Pairwise Comparisons for Prioritized Outcomes

Marc Buyse () and Julien Peron ()
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Marc Buyse: International Drug Development Institute (IDDI) Inc.
Julien Peron: CNRS, UMR 5558, Laboratoire de Biométrie et Biologie Evolutive

Chapter 95 in Principles and Practice of Clinical Trials, 2022, pp 1869-1893 from Springer

Abstract: Abstract The Wilcoxon-Mann-Whitney U-statistic can be extended to perform generalized pairwise comparisons between two groups of observations. The observations are outcomes captured by a single variable, possibly repeatedly measured, or by several variables of any type (e.g., discrete, continuous, time to event). Generalized pairwise comparisons can include an arbitrary number of (possibly prioritized) outcomes and thresholds of clinical relevance. They extend standard nonparametric tests and lead to a general measure of the difference between the groups, the “Net Benefit,” which is the probability that a patient randomly selected from the treatment group has a better outcome than a patient randomly selected from the control group, minus the probability of the opposite situation. One flexible approach to the analysis is to prioritize the outcomes from the most important to the least important. The order of priorities can be patient-dependent, and as such this approach paves the way to personalized medicine.

Keywords: Wilcoxon test; U-statistic; Generalized pairwise comparisons; Prioritized outcomes; Measure of treatment effect; Net Benefit; Win ratio (search for similar items in EconPapers)
Date: 2022
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DOI: 10.1007/978-3-319-52636-2_277

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