Robust statistical inference for the matched net benefit and the matched win ratio.

Seminar Series

Friday, February 12, 2021 - 01:30
Zoom Conference
Roland Matsouaka, PhD

Abstract: As alternatives to the time-to-first-event analysis of composite endpoints, the net benefit (NB) and the win ratio (WR) -- which assess treatment effects using prioritized component outcomes based on clinical importance -- have been proposed. However, statistical inference of NB and WR relies on a large-sample assumptions, which can lead to an invalid test statistic and inadequate, unsatisfactory confidence intervals, especially when the sample size is small or the proportion of wins is near 0 or 1.

For this talk, we will show how to address these limitations in a paired-sample design. We first introduce a new test statistic under the null hypothesis of no treatment difference. Then, present new ways to estimate the confidence intervals of these estimands. The confidence interval estimations use the method of variance estimates recovery (MOVER). The MOVER combines two separate individual-proportion confidence intervals into a hybrid interval for each estimand of interest. We assess the performance of the proposed test statistic and MOVER confidence interval estimations through simulation studies.

We demonstrate that the MOVER confidence intervals are as good as the large-sample confidence intervals when the sample is large and when the proportions of wins is bounded away from 0 and 1. Moreover, the MOVER intervals outperform their competitors when the sample is small or the proportions are at or near the boundaries 0 and 1. We illustrate the method (and its competitors) using three examples from randomized clinical studies.

Speaker: Roland Matsouaka, PhD
Assistant Professor
Department of Biostatistics & Bioinformatics
Duke University School of Medicine


Mtg ID:     988 1883 7919  

Passcode:       978442