Weighted win loss approach for analyzing prioritized outcomes

Stat Med. 2017 Jul 10;36(15):2452-2465. doi: 10.1002/sim.7284. Epub 2017 Mar 26.

Abstract

To analyze prioritized outcomes, Buyse (2010) and Pocock et al. (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 © 2017 John Wiley & Sons, Ltd.

Keywords: clinical trials; composite end points; contribution index; prioritized outcomes; variance estimation; weighted win ratio.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Acute Coronary Syndrome / drug therapy
  • Angiotensin-Converting Enzyme Inhibitors / therapeutic use
  • Biostatistics
  • Clinical Trials as Topic / statistics & numerical data*
  • Computer Simulation
  • Coronary Artery Disease / drug therapy
  • Data Interpretation, Statistical
  • Endpoint Determination / statistics & numerical data
  • Factor Xa Inhibitors / therapeutic use
  • Humans
  • Models, Statistical
  • Proportional Hazards Models
  • Randomized Controlled Trials as Topic / statistics & numerical data
  • Rivaroxaban / therapeutic use
  • Survival Analysis
  • Treatment Outcome

Substances

  • Angiotensin-Converting Enzyme Inhibitors
  • Factor Xa Inhibitors
  • Rivaroxaban