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

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 poin...

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Bibliographic Details
Published in:Value in health Vol. 20; no. 3; pp. 487 - 495
Main Authors: Ciani, Oriana, Buyse, Marc, Drummond, Michael, Rasi, Guido, Saad, Everardo D., Taylor, Rod S.
Format: Journal Article
Language:English
Published: United States Elsevier Inc 01.03.2017
Elsevier Science Ltd
Subjects:
Adoption of innovations
Biological markers
Biomarkers
clinical outcome assessment
Clinical outcomes
Clinical research
Clinical trials
Clinical Trials as Topic
Disease-Free Survival
Drugs
Efficacy
Evidence-Based Medicine
Health care policy
Health Policy
Health services
health technology assessment
Humans
Internal Medicine
Medical equipment
Medical Oncology
Medical supplies
Medical technology
Morbidity
Patient communication
Patients
Policy making
Quality-Adjusted Life Years
surrogate end points
Technology Assessment, Biomedical
Treatment Outcome
United States
United States Food and Drug Administration
validation
Validity
health technology assessment
clinical outcome assessment
surrogate end points
validation
ISSN:1098-3015, 1524-4733, 1524-4733
Online Access:Get full text
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Summary: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.
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ISSN:1098-3015
1524-4733
1524-4733
DOI:10.1016/j.jval.2016.10.011