Paper celebrating the 25th Anniversary of Statistics in Medicine
Statistical evaluation of biomarkers as surrogate endpoints: a literature review
Article first published online: 26 OCT 2005
Copyright © 2005 John Wiley & Sons, Ltd.
Statistics in Medicine
Volume 25, Issue 2, pages 183–203, 30 January 2006
How to Cite
Weir, C. J. and Walley, R. J. (2006), Statistical evaluation of biomarkers as surrogate endpoints: a literature review. Statist. Med., 25: 183–203. doi: 10.1002/sim.2319
- Issue published online: 23 DEC 2005
- Article first published online: 26 OCT 2005
- Manuscript Accepted: APR 2005
- Manuscript Received: SEP 2004
- U.K. Medical Research Council Career Development Fellowship. Grant Number: G120/628
- drug development
A valid surrogate endpoint allows correct inference to be drawn regarding the effect of an intervention on the unobserved true clinical endpoint of interest. The perceived practical and ethical advantages of substituting a surrogate endpoint for a clinical endpoint have led to a considerable number of statistical methods being proposed for the evaluation of a biomarker as a surrogate endpoint. We review the main statistical schools of thought which have developed and consider how the validation process might be arranged within the regulatory and practical constraints of the drug development process. We conclude by assessing which of the candidate statistical methods offer the best approach for surrogate endpoint evaluation. Copyright © 2005 John Wiley & Sons, Ltd.