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Generalized Pairwise Comparisons for Prioritized Outcomes

In: Principles and Practice of Clinical Trials

Author

Listed:
  • Marc Buyse

    (International Drug Development Institute (IDDI) Inc.
    Hasselt University, Interuniversity Institute for Biostatistics and Statistical Bioinformatics (I-BioStat)
    University of Hasselt, CluePoints S.A., Louvain-la-Neuve, Belgium and I-BioStat)

  • Julien Peron

    (CNRS, UMR 5558, Laboratoire de Biométrie et Biologie Evolutive
    Centre Hospitalier Lyon-Sud, Institut de Cancérologie des Hospices Civils de Lyon, Departments of Biostatistics and Medical Oncology)

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.

Suggested Citation

  • Marc Buyse & Julien Peron, 2022. "Generalized Pairwise Comparisons for Prioritized Outcomes," Springer Books, in: Steven Piantadosi & Curtis L. Meinert (ed.), Principles and Practice of Clinical Trials, chapter 95, pages 1869-1893, Springer.
    • Handle: RePEc:spr:sprchp:978-3-319-52636-2_277
    DOI: 10.1007/978-3-319-52636-2_277
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