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Conditionally unbiased estimation in phase II/III clinical trials with early stopping for futility
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Kimani, Peter K., Todd, Susan C. and Stallard, Nigel (2013) Conditionally unbiased estimation in phase II/III clinical trials with early stopping for futility. Statistics in Medicine, Volume 32 (Number 17). pp. 2893-2901. doi:10.1002/sim.5757
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WRAP_Kimani_Conditionally_unbiased_estimation_sim5757.pdf - Published Version Available under License Creative Commons Attribution. Download (410Kb) | Preview |
Official URL: http://dx.doi.org/10.1002/sim.5757
Abstract
Seamless phase II/III clinical trials combine traditional phases II and III into a single trial that is conducted in two stages, with stage 1 used to answer phase II objectives such as treatment selection and stage 2 used for the confirmatory analysis, which is a phase III objective. Although seamless phase II/III clinical trials are efficient because the confirmatory analysis includes phase II data from stage 1, inference can pose statistical challenges. In this paper, we consider point estimation following seamless phase II/III clinical trials in which stage 1 is used to select the most effective experimental treatment and to decide if, compared with a control, the trial should stop at stage 1 for futility. If the trial is not stopped, then the phase III confirmatory part of the trial involves evaluation of the selected most effective experimental treatment and the control. We have developed two new estimators for the treatment difference between these two treatments with the aim of reducing bias conditional on the treatment selection made and on the fact that the trial continues to stage 2. We have demonstrated the properties of these estimators using simulations. Copyright © 2013 John Wiley & Sons, Ltd.
| Item Type: | Journal Article | ||||
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| Subjects: | Q Science > QA Mathematics R Medicine > RS Pharmacy and materia medica |
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| Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School | ||||
| Library of Congress Subject Headings (LCSH): | Drugs -- Testing , Clinical trials, Clinical pharmacology, Medical statistics, Medicine -- Mathematics | ||||
| Journal or Publication Title: | Statistics in Medicine | ||||
| Publisher: | John Wiley & Sons Ltd. | ||||
| ISSN: | 0277-6715 | ||||
| Official Date: | 30 July 2013 | ||||
| Dates: |
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| Volume: | Volume 32 | ||||
| Number: | Number 17 | ||||
| Page Range: | pp. 2893-2901 | ||||
| DOI: | 10.1002/sim.5757 | ||||
| Status: | Peer Reviewed | ||||
| Publication Status: | Published | ||||
| Access rights to Published version: | Open Access |
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