The Library
Flexible selection of a single treatment incorporating short-term endpoint information in a phase II/III clinical trial
Tools
Stallard, Nigel, Kunz, Cornelia, Todd, Susan, Parsons, Nicholas R. and Friede, Tim (2015) Flexible selection of a single treatment incorporating short-term endpoint information in a phase II/III clinical trial. Statistics in Medicine, 34 (23). pp. 3104-3115. doi:10.1002/sim.6567 ISSN 0277-6715.
|
PDF
WRAP_Stallard et al 2015 SiM.pdf - Published Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (394Kb) | Preview |
Official URL: http://dx.doi.org/10.1002/sim.6567
Abstract
Seamless phase II/III clinical trials in which an experimental treatment is selected at an interim analysis have been the focus of much recent research interest. Many of the methods proposed are based on the group sequential approach. This paper considers designs of this type in which the treatment selection can be based on short-term endpoint information for more patients than have primary endpoint data available. We show that in such a case, the familywise type I error rate may be inflated if previously proposed group sequential methods are used and the treatment selection rule is not specified in advance. A method is proposed to avoid this inflation by considering the treatment selection that maximises the conditional error given the data available at the interim analysis. A simulation study is reported that illustrates the type I error rate inflation and compares the power of the new approach with two other methods: a combination testing approach and a group sequential method that does not use the short-term endpoint data, both of which also strongly control the type I error rate. The new method is also illustrated through application to a study in Alzheimer's disease. © 2015 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd.
Item Type: | Journal Article | ||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|
Subjects: | R Medicine > RA Public aspects of medicine | ||||||||||
Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
||||||||||
Library of Congress Subject Headings (LCSH): | Clinical trials -- Methodology | ||||||||||
Journal or Publication Title: | Statistics in Medicine | ||||||||||
Publisher: | John Wiley & Sons Ltd. | ||||||||||
ISSN: | 0277-6715 | ||||||||||
Official Date: | 15 October 2015 | ||||||||||
Dates: |
|
||||||||||
Volume: | 34 | ||||||||||
Number: | 23 | ||||||||||
Number of Pages: | 12 | ||||||||||
Page Range: | pp. 3104-3115 | ||||||||||
DOI: | 10.1002/sim.6567 | ||||||||||
Status: | Peer Reviewed | ||||||||||
Publication Status: | Published | ||||||||||
Access rights to Published version: | Open Access (Creative Commons) | ||||||||||
Date of first compliant deposit: | 31 December 2015 | ||||||||||
Date of first compliant Open Access: | 31 December 2015 | ||||||||||
Funder: | Medical Research Council (Great Britain) (MRC) | ||||||||||
Grant number: | G1001344 (MRC) |
Request changes or add full text files to a record
Repository staff actions (login required)
View Item |
Downloads
Downloads per month over past year