Weighted win loss approach for analyzing prioritized outcomes

被引：31
|
作者：
Luo, Xiaodong [1 ]
Qiu, Junshan [2 ]
Bai, Steven [2 ]
Tian, Hong [3 ]
机构：
[1] Sanofi US, Res & Dev, Bridgewater, NJ 08807 USA
[2] US FDA, Ctr Drug Evaluat & Res, Div Biometr 1, Off Biostat, Silver Spring, MD 20993 USA
[3] Janssen Res & Dev, Raritan, NJ 08869 USA
来源：
STATISTICS IN MEDICINE | 2017年 / 36卷 / 15期
关键词：
clinical trials; composite end points; contribution index; prioritized outcomes; weighted win ratio; variance estimation; CLINICAL-TRIALS; RATIO APPROACH;
D O I：
10.1002/sim.7284
中图分类号：
Q [生物科学];
学科分类号：
07 ; 0710 ; 09 ;
摘要：
To analyze prioritized outcomes, Buyse (2010) and Pocock etal. (2012) proposed the win loss approach. In this paper, we first study the relationship between the win loss approach and the traditional survival analysis on the time to the first event. We then propose the weighted win loss statistics to improve the efficiency of the unweighted methods. A closed-form variance estimator of the weighted win loss statistics is derived to facilitate hypothesis testing and study design. We also calculated the contribution index to better interpret the results of the weighted win loss approach. Simulation studies and real data analysis demonstrated the characteristics of the proposed statistics. Copyright (c) 2017 John Wiley & Sons, Ltd.
引用
收藏
页码：2452 / 2465
页数：14
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