Title | A novel test to compare two treatments based on endpoints involving both nonfatal and fatal events. |
Publication Type | Journal Article |
Year of Publication | 2015 |
Authors | Potthoff, Richard F., and Susan Halabi |
Journal | Pharm Stat |
Volume | 14 |
Issue | 4 |
Pagination | 273-83 |
Date Published | 2015 Jul-Aug |
ISSN | 1539-1612 |
Keywords | Carcinoma, Renal Cell, Clinical Trials, Phase III as Topic, Computer Simulation, Data Interpretation, Statistical, Endpoint Determination, Humans, Kidney Neoplasms, Models, Statistical, Randomized Controlled Trials as Topic, Research Design, Statistics, Nonparametric, Time Factors, Treatment Outcome |
Abstract | In a clinical trial comparing two treatment groups, one commonly-used endpoint is time to death. Another is time until the first nonfatal event (if there is one) or until death (if not). Both endpoints have drawbacks. The wrong choice may adversely affect the value of the study by impairing power if deaths are too few (with the first endpoint) or by lessening the role of mortality if not (with the second endpoint). We propose a compromise that provides a simple test based on the time to death if the patient has died or time since randomization augmented by an increment otherwise. The test applies the ordinary two-sample Wilcoxon statistic to these values. The formula for the increment (the same for experimental and control patients) must be specified before the trial starts. In the simplest (and perhaps most useful) case, the increment assumes only two values, according to whether or not the (surviving) patient had a nonfatal event. More generally, the increment depends on the time of the first nonfatal event, if any, and the time since randomization. The test has correct Type I error even though it does not handle censoring in a customary way. For conditions where investigators would face no easy (advance) choice between the two older tests, simulation results favor the new test. An example using a renal-cancer trial is presented. |
DOI | 10.1002/pst.1683 |
Alternate Journal | Pharm Stat |
Original Publication | A novel test to compare two treatments based on endpoints involving both nonfatal and fatal events. |
PubMed ID | 25894200 |
PubMed Central ID | PMC4503508 |
Grant List | U01 CA157703 / CA / NCI NIH HHS / United States P01 CA142538 / CA / NCI NIH HHS / United States R01 CA155296 / CA / NCI NIH HHS / United States CA142538 / CA / NCI NIH HHS / United States CA155296 / CA / NCI NIH HHS / United States |
A novel test to compare two treatments based on endpoints involving both nonfatal and fatal events.
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