Format

Send to

Choose Destination
Value Health. 2017 Mar;20(3):487-495. doi: 10.1016/j.jval.2016.10.011. Epub 2016 Dec 22.

Time to Review the Role of Surrogate End Points in Health Policy: State of the Art and the Way Forward.

Author information

1
Evidence Synthesis and Modelling for Health Improvement, Institute of Health Research, University of Exeter Medical School, Exeter, UK; Centre for Research on Health and Social Care Management, Bocconi University, Milan, Italy. Electronic address: o.ciani@exeter.ac.uk.
2
International Drug Development Institute, Louvain-la-Neuve, Belgium; CluePoints, Inc., Cambridge, MA, USA.
3
Centre for Health Economics, University of York, York, UK.
4
European Medicines Agency, London, UK; University "Tor Vergata," Rome, Italy.
5
Dendrix Research, Sao Paulo, Brazil.
6
Evidence Synthesis and Modelling for Health Improvement, Institute of Health Research, University of Exeter Medical School, Exeter, UK.

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.

KEYWORDS:

clinical outcome assessment; health technology assessment; surrogate end points; validation

PMID:
28292495
DOI:
10.1016/j.jval.2016.10.011
[Indexed for MEDLINE]
Free full text

Supplemental Content

Full text links

Icon for Elsevier Science
Loading ...
Support Center