Blinded sample size recalculation for clinical trials with normal data and baseline adjusted analysis

Pharmaceutical Statistics
T Friede, M Kieser

Abstract

Baseline adjusted analyses are commonly encountered in practice, and regulatory guidelines endorse this practice. Sample size calculations for this kind of analyses require knowledge of the magnitude of nuisance parameters that are usually not given when the results of clinical trials are reported in the literature. It is therefore quite natural to start with a preliminary calculated sample size based on the sparse information available in the planning phase and to re-estimate the value of the nuisance parameters (and with it the sample size) when a portion of the planned number of patients have completed the study. We investigate the characteristics of this internal pilot study design when an analysis of covariance with normally distributed outcome and one random covariate is applied. For this purpose we first assess the accuracy of four approximate sample size formulae within the fixed sample size design. Then the performance of the recalculation procedure with respect to its actual Type I error rate and power characteristics is examined. The results of simulation studies show that this approach has favorable properties with respect to the Type I error rate and power. Together with its simplicity, these features should make i...Continue Reading

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Citations

Aug 25, 2012·Trials·John A KairallaKeith E Muller
Apr 14, 2015·International Journal of Stroke : Official Journal of the International Stroke Society·Silvia SchönenbergerJulian Bösel
Jun 14, 2017·Statistics in Medicine·Tobias Mütze, Tim Friede
Feb 11, 2010·Statistics in Medicine·Tim Friede, Heinz Schmidli
Jan 5, 2018·Statistical Methods in Medical Research·Marius Placzek, Tim Friede
Jul 1, 2013·Therapeutic Innovation & Regulatory Science·Daniel Wachtlin, Meinhard Kieser
Jul 23, 2019·Journal of Biopharmaceutical Statistics·Georg ZimmermannArne C Bathke
Nov 18, 2021·Hip International : the Journal of Clinical and Experimental Research on Hip Pathology and Therapy·Catriona HeaverRobin D Banerjee

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