New designs for the selection of treatments to be tested in randomized clinical trials
Article first published online: 15 OCT 2006
Copyright © 1994 John Wiley & Sons, Ltd.
Statistics in Medicine
Volume 13, Issue 5-7, pages 417–429, 15 March - 15 April 1994
How to Cite
Simon, R., Thall, P. F. and Ellenberg, S. S. (1994), New designs for the selection of treatments to be tested in randomized clinical trials. Statist. Med., 13: 417–429. doi: 10.1002/sim.4780130506
- Issue published online: 15 OCT 2006
- Article first published online: 15 OCT 2006
The most important aspect of phase III randomized clinical trials is the selection of the experimental treatments to be tested. Often this decision is based on uncontrolled phase II trials. Substantial statistical attention has been focused on the design of phase III trials and for simple phase II trials, which determine whether a new drug has any anti-disease activity. Much less statistical effort has been devoted to the design and analysis of phase II trials for screening active experimental treatments to determine whether they are sufficiently active, relative to standard treatments, to warrant the conduct of a large randomized phase III trial. This problem is particularly acute in the development of drug combinations where many regimens are possible. We review several designs for such screening trials which we have developed.