Journal Article

Progression-free survival as a surrogate for overall survival in oncology trials: a methodological systematic review

De Rycke, Yann, Dechartres, Agnès, Belin, Lisa, Tan, Aidan

2020

Overview

Background Progression-free survival (PFS) is a surrogate endpoint widely used for overall survival (OS) in oncology. Validation of PFS as a surrogate must be done for each indication and each intervention. We aimed to identify all studies evaluating the validity of PFS as a surrogate for OS in oncology, and to describe their methodological characteristics. Methods We conducted a systematic review by searching MEDLINE via PubMed and the Cochrane Library with no limitation on time, selected relevant studies and extracted data in duplicate on how surrogacy was evaluated (meta-analytic approach, assessment of correlation and level of evaluation). Results We identified 91 studies evaluating the validity of PFS as a surrogate for OS in 24 cancer localisations. Although a meta-analytic approach was used in 83 (91%) studies, the methods used to validate PFS as a surrogate of OS were heterogeneous across studies. Of the 47 studies concluding that PFS is a good surrogate for OS, for 15 (32%), there was no quantitative argument for surrogacy. Conclusions Although most studies used a meta-analytic approach as recommended, our methodological review highlights heterogeneity in methods and reporting, which stresses the importance of developing and applying clear recommendations in this area.

• Publisher: Nature Publishing Group UK,Nature Publishing Group,Cancer Research UK
• Subject: 692/308/409 / 692/4028/67 / Biomedical and Life Sciences / Biomedicine / Cancer Research / Clinical Trials as Topic - methods / Clinical Trials as Topic - standards / Drug Resistance / Epidemiology / Humans / Life Sciences / Medical Oncology - methods / Medical Oncology - standards / Molecular Medicine / Neoplasms - mortality / Oncology / Progression-Free Survival / Reviews / Survival / Systematic review