Testing for Trend in Benefit-Risk Analysis with Prioritized Multiple Outcomes
Ziqiao Wang
Jie Chen
10.6084/m9.figshare.10277660.v2
https://tandf.figshare.com/articles/dataset/Testing_for_Trend_in_Benefit-Risk_Analysis_with_Prioritized_Multiple_Outcomes/10277660
<p>Benefit-risk analysis using prioritized multiple outcomes has been proposed for use in randomized controlled trials with two treatment groups. This research extends the two-group comparison to testing for a trend over multiple treatment groups, for example, multiple doses, in terms of a composite outcome that is derived from pairwise comparisons of subjects between groups or by ranking subjects of pooled groups, according to their prioritized outcomes. Tukey’s trend test, Wilcoxon rank-sum test and Jonkheere-Terpstra (JT) test and their permutation-based trend tests are investigated for detection of an increasing trend over doses with respect to the composite benefit-risk endpoint. Simulation studies show that the permutation-based Tukey’s and JT tests outperform the others in terms of Type I error control and power under various simulation settings. For illustrative purpose, the six approaches are applied to a migraine example data to determine whether an increasing trend exists among four dose groups in terms of a composite benefit-risk endpoint that are measured by four prioritized outcomes.</p>
2020-01-04 02:11:59
Benefit-risk analysis
Pairwise comparisons
Prioritized outcomes
Ranking
Trend test