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Use of Meta-Analysis for the Validation of Surrogate Endpoints and Biomarkers in Cancer Trials

Buyse, Marc ScD

doi: 10.1097/PPO.0b013e3181b9c602
Special Issue on Monitoring of Therapeutic Responses to Cancer Treatment: Review Article
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This article discusses statistical approaches to the validation of surrogate biomarkers and endpoints. One approach that has been successfully used in oncology consists of estimating associations at two levels: the association between the surrogate and the clinical endpoint, called the individual-level association, and the association between the effects of treatment on the surrogate and the clinical endpoint, called the trial-level association. This approach requires data to be available from multiple randomized trials, such as in a meta-analysis of trials based on individual patient data. The approach is illustrated using randomized trials of first-line treatments for advanced tumors of the colon, breast, ovary, and prostate. Data from several meta-analyses suggest that progression-free survival is an acceptable surrogate in advanced colorectal and ovarian cancer, but not in breast and prostate cancer.

From the International Drug Development Institute, Louvain-la-Neuve, Belgium; and Center for Statistics, I-BioStat, Hasselt University, Diepenbeek, Belgium.

Reprints: Marc Buyse, ScD, IDDI, 30 Avenue Provinciale, Louvain-la-Neuve 1340, Belgium. E-mail: marc.buyse@iddi.com.

© 2009 Lippincott Williams & Wilkins, Inc.