Applying the win ratio method in clinical trials of orphan drugs: an analysis of data from the COMET trial of avalglucosidase alfa in patients with late-onset Pompe disease.

Matthias BoentertKenneth I BergerJordi Díaz-ManeraMazen M DimachkieAlaa HamedLionel Riou FrançaNathan ThibaultPragya ShuklaJack IshakJ Jaime Caro

Published in: Orphanet journal of rare diseases (2024)

The win ratio approach can be used in clinical trials of rare diseases to provide meaningful insight on treatment benefits from multiple endpoints and across disease domains.

Keyphrases

late onset
clinical trial
early onset
phase ii
phase iii
replacement therapy
study protocol
open label
electronic health record
double blind
machine learning