Composite outcomes are common in clinical trials, especially for multiple time-to-event outcomes (endpoints). The standard approach that uses the time to the first outcome event has important limitations. Several alternative approaches have been proposed to compare treatment versus control, including the proportion in favor of treatment and the win ratio. Herein, we construct tests of significance and confidence intervals in the context of composite outcomes based on prioritized components using the large sample distribution of certain multivariate multi-sample U-statistics. This non-parametric approach provides a general inference for both the proportion in favor of treatment and the win ratio, and can be extended to stratified analyses and the comparison of more than two groups. The proposed methods are illustrated with time-to-event outcomes data from a clinical trial.
Reference Type
Journal Article
Periodical Full
Biostatistics (Oxford, England)
Publication Year
2016
Volume
17
Issue
1
Start Page
178
Other Pages
187
Publisher
Oxford University Press (OUP)
Place of Publication
England
ISSN/ISBN
1468-4357
Document Object Index
10.1093/biostatistics/kxv032
URL
https://search.datacite.org/works/10.1093/biostatistics/kxv032
PMID
26353896