Win statistics (win ratio, win odds, and net benefit) can complement one another to show the strength of the treatment effect on time‐to‐event outcomes

审查(临床试验) 统计 补语(音乐) 可能性 计量经济学 统计的 成对比较 事件(粒子物理) 无效假设 数学 统计假设检验 逻辑回归 表型 物理 化学 互补 基因 量子力学 生物化学
作者
Gaohong Dong,Bo Huang,Johan Verbeeck,Ying Cui,James Song,Margaret Gamalo,Duolao Wang,David C. Hoaglin,Yodit Seifu,Tobias Mütze,John E. Kolassa
出处
期刊:Pharmaceutical Statistics [Wiley]
卷期号:22 (1): 20-33 被引量:42
标识
DOI:10.1002/pst.2251
摘要

Abstract Conventional analyses of a composite of multiple time‐to‐event outcomes use the time to the first event. However, the first event may not be the most important outcome. To address this limitation, generalized pairwise comparisons and win statistics (win ratio, win odds, and net benefit) have become popular and have been applied to clinical trial practice. However, win ratio, win odds, and net benefit have typically been used separately. In this article, we examine the use of these three win statistics jointly for time‐to‐event outcomes. First, we explain the relation of point estimates and variances among the three win statistics, and the relation between the net benefit and the Mann–Whitney U statistic. Then we explain that the three win statistics are based on the same win proportions, and they test the same null hypothesis of equal win probabilities in two groups. We show theoretically that the Z‐values of the corresponding statistical tests are approximately equal; therefore, the three win statistics provide very similar p ‐values and statistical powers. Finally, using simulation studies and data from a clinical trial, we demonstrate that, when there is no (or little) censoring, the three win statistics can complement one another to show the strength of the treatment effect. However, when the amount of censoring is not small, and without adjustment for censoring, the win odds and the net benefit may have an advantage for interpreting the treatment effect; with adjustment (e.g., IPCW adjustment) for censoring, the three win statistics can complement one another to show the strength of the treatment effect. For calculations we use the R package WINS, available on the CRAN (Comprehensive R Archive Network).
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