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The inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic: an unbiased estimator in the presence of independent censoring

Gaohong Dong, Lu Mao, Bo Huang, Margaret Gamalo, Jiuzhou Wang, Guanglei Yu, David C. Hoaglin

2020Journal of Biopharmaceutical Statistics76 citationsDOIOpen Access PDF

Abstract

The win ratio method has received much attention in methodological research, ad hoc analyses, and designs of prospective studies. As the primary analysis, it supported the approval of tafamidis for the treatment of cardiomyopathy to reduce cardiovascular mortality and cardiovascular-related hospitalization. However, its dependence on censoring is a potential shortcoming. In this article, we propose the inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic (i.e., the IPCW-adjusted win ratio statistic) to overcome censoring issues. We consider independent censoring, common censoring across endpoints, and right censoring. We develop an asymptotic variance estimator for the logarithm of the IPCW-adjusted win ratio statistic and evaluate it via simulation. Our simulation studies show that, as the amount of censoring increases, the unadjusted win proportions may decrease greatly. Consequently, the bias of the unadjusted win ratio estimate may increase greatly, producing either an overestimate or an underestimate. We demonstrate theoretically and through simulation that the IPCW-adjusted win ratio statistic gives an unbiased estimate of treatment effect.

Topics & Concepts

Censoring (clinical trials)StatisticsMathematicsEstimatorStatisticWeightingInverse probability weightingInverse probabilityU-statisticMinimum-variance unbiased estimatorEconometricsMedicineBayesian probabilityPosterior probabilityRadiologyStatistical Distribution Estimation and ApplicationsAmyloidosis: Diagnosis, Treatment, OutcomesGenetic Associations and Epidemiology
The inverse-probability-of-censoring weighting (IPCW) adjusted win ratio statistic: an unbiased estimator in the presence of independent censoring | Litcius