Time to review the role of surrogate endpoints in health policy: state of the art and the way forward

Ciani, O., Buyse, M., Drummond, M., Rasi, G., Saad, E. D. and Taylor, R. S. (2017) Time to review the role of surrogate endpoints in health policy: state of the art and the way forward. Value in Health, 20(3), pp. 487-495. (doi: 10.1016/j.jval.2016.10.011) (PMID:28292495)

Abstract

The efficacy of medicines, medical devices, and other health technologies should be proved in trials that assess final patient-relevant outcomes such as survival or morbidity. Market access and coverage decisions are, however, often based on surrogate end points, biomarkers, or intermediate end points, which aim to substitute and predict patient-relevant outcomes that are unavailable because of methodological, financial, or practical constraints. We provide a summary of the present use of surrogate end points in health care policy, discussing the case for and against their adoption and reviewing validation methods. We introduce a three-step framework for policymakers to handle surrogates, which involves establishing the level of evidence, assessing the strength of the association, and quantifying relations between surrogates and final outcomes. Although the use of surrogates can be problematic, they can, when selected and validated appropriately, offer important opportunities for more efficient clinical trials and faster access to new health technologies that benefit patients and health care systems.

• Item Type: Articles
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Taylor, Professor Rod
• Authors: Ciani, O., Buyse, M., Drummond, M., Rasi, G., Saad, E. D., and Taylor, R. S.
• College/School: College of Medical Veterinary and Life Sciences > School of Health & Wellbeing > MRC/CSO SPHSU
• Journal Name: Value in Health
• Publisher: Elsevier
• ISSN: 1098-3015
• ISSN (Online): 1524-4733
• Published Online: 22 December 2016
• Copyright Holders: Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR)
• First Published: First published in Value in Health 20(3):487-495
• Publisher Policy: Reproduced in accordance with the copyright policy of the publisher