SA26 Win Ratio Analyses of Multiple Endpoints in Rare Disease Trials: A Case-Study Based on a Trial of Avaglucosidase Alfa in Late-Onset Pompe Disease (LOPD)

Abstract

The win ratio approach aims to improve analyses of composite endpoints in clinical trials by accounting for the relative importance of components of the composite endpoint and enhancing interpretability of the treatment effect measure. We propose a new application as an alternative to hierarchical testing of endpoints to improve statistical power in small trials (e.g., in rare diseases). This is illustrated with a reanalysis of the COMET trial (NCT02782741) comparing avalglucosidase alfa (AVA) and alglucosidase alfa (ALGLU) in patients with LOPD, in which statistical noninferiority was achieved, but superiority was not met.