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Joint testing of mortality and a non‐fatal outcome in clinical trials
Author(s) -
McMahon Robert P.,
Harrell Frank E.
Publication year - 2001
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.814
Subject(s) - bonferroni correction , medicine , outcome (game theory) , mortality rate , clinical trial , rank (graph theory) , statistics , mathematics , mathematical economics , combinatorics
The following procedures are proposed for clinical trials of potentially lethal diseases with a non‐fatal primary outcome: (i) comparison of treatments on worst‐rank scores combining mortality and the non‐fatal outcome; (ii) testing first for mortality differences, and then for differences on the worst‐rank scoring if there is no significant evidence for mortality differences. Power and control of type I error rate while testing mortality and worst‐rank scores by Bonferroni or closed testing procedures are compared. Used together these procedures improve power to detect treatments with favourable effects on both mortality and the designated non‐fatal outcome, while reducing the likelihood of declaring that a treatment benefits patients when undetected adverse effects on mortality are present. Copyright © 2001 John Wiley & Sons, Ltd.