MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design.

Abstract

A non-inferiority trial is usually conducted to investigate whether a new drug/treatment is no worse than a reference drug/treatment by a small, pre-specified, non-inferiority margin. This study aimed to assess the non-inferiority of the difference between two binary-outcome treatments in a matched-pairs design based on the method of variance of estimates recovery (MOVER). The processes for estimating the confidence interval of a single proportion included in the MOVER are the Wilson score interval, Agresti - Coull interval, Jeffreys interval, modified Jeffreys interval, score method with continuity correction, and arcsin interval. The performance of the six MOVER tests, the fiducial test, and the restricted maximum likelihood estimation test were evaluated by comparing their type I error rates and power at different pre-assigned levels and with varying combinations of parameters. The evaluation results showed that the modified Jeffreys MOVER test can be a competitive alternative to the other recommended tests. It can control type I errors well, and its power is not inferior to other methods. The proposed tests were illustrated with three real-world examples.