Dehbi, Hakim‐Moulay;
Embleton‐Thirsk, Andrew;
McCaw, Zachary Ryan;
(2022)
Sample size calculation for randomized selection trials with a time‐to‐event endpoint and a margin of practical equivalence.
Statistics in Medicine
10.1002/sim.9490.
(In press).
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Abstract
Selection trials are used to compare potentially active experimental treatments without a control arm. While sample size calculation methods exist for binary endpoints, no such methods are available for time-to-event endpoints, even though these are ubiquitous in clinical trials. Recent selection trials have begun using progression-free survival as their primary endpoint, but have dichotomized it at a specific time point for sample size calculation and analysis. This changes the clinical question and may reduce power to detect a difference between the arms. In this article, we develop the theory for sample size calculation in selection trials where the time-to-event endpoint is assumed to follow an exponential or Weilbull distribution. We provide a free web application for sample size calculation, as well as an R package, that researchers can use in the design of their studies.
| Type: | Article |
|---|---|
| Title: | Sample size calculation for randomized selection trials with a time‐to‐event endpoint and a margin of practical equivalence |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1002/sim.9490 |
| Publisher version: | https://doi.org/10.1002/sim.9490 |
| Language: | English |
| Additional information: | © 2022 The Authors. Statistics in Medicine published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
| Keywords: | early phase trials, randomization, sample size calculation, selection trials |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > Comprehensive CTU at UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10150133 |
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