1. Reference curve sampling variability in one–sample log–rank tests
- Author
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Danzer, Moritz Fabian, Feld, Jannik, Faldum, Andreas, Schmidt, René, and Universitäts- und Landesbibliothek Münster
- Subjects
610 Medicine and health ,Medicine and health ,ddc:610 ,Research errors ,Test statistics ,Random variables ,Normal distribution ,Distribution curves ,Statistical distributions ,Stochastic processes ,Probability theory - Abstract
The one–sample log–rank test is the method of choice for single–arm Phase II trials with time–to–event endpoint. It allows to compare the survival of patients to a reference survival curve that typically represents the expected survival under standard of care. The one–sample log–rank test, however, assumes that the reference survival curve is known. This ignores that the reference curve is commonly estimated from historic data and thus prone to sampling error. Ignoring sampling variability of the reference curve results in type I error rate inflation. We study this inflation in type I error rate analytically and by simulation. Moreover we derive the actual distribution of the one–sample log–rank test statistic, when the sampling variability of the reference curve is taken into account. In particular, we provide a consistent estimate of the factor by which the true variance of the one-sample log–rank statistic is underestimated when reference curve sampling variability is ignored. Our results are further substantiated by a case study using a real world data example in which we demonstrate how to estimate the error rate inflation in the planning stage of a trial., Finanziert durch den Open-Access-Publikationsfonds der Westfälischen Wilhelms-Universität Münster (WWU Münster)
- Published
- 2022
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