An alternative approach to confidence interval estimation for the win ratio statistic

Biometrics. 2015 Mar;71(1):139-145. doi: 10.1111/biom.12225. Epub 2014 Aug 25.

Abstract

Pocock et al. (2012, European Heart Journal 33, 176-182) proposed a win ratio approach to analyzing composite endpoints comprised of outcomes with different clinical priorities. In this article, we establish a statistical framework for this approach. We derive the null hypothesis and propose a closed-form variance estimator for the win ratio statistic in all pairwise matching situation. Our simulation study shows that the proposed variance estimator performs well regardless of the magnitude of treatment effect size and the type of the joint distribution of the outcomes.

Keywords: Clinical trials; Composite endpoints; Hypothesis testing; Variance estimation; Win ratio.

Publication types

  • Comparative Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Algorithms*
  • Confidence Intervals
  • Data Interpretation, Statistical*
  • Factor Xa Inhibitors / therapeutic use
  • Humans
  • Odds Ratio
  • Outcome Assessment, Health Care / methods*
  • Reproducibility of Results
  • Risk Assessment / methods
  • Rivaroxaban / therapeutic use*
  • Sensitivity and Specificity
  • Statistical Distributions
  • Stroke / mortality*
  • Stroke / prevention & control*
  • Survival Analysis
  • Treatment Outcome

Substances

  • Factor Xa Inhibitors
  • Rivaroxaban