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ENDPOINTS FOR RELATIVE EFFECTIVENESS ASSESSMENT (REA) OF PHARMACEUTICALS

Published online by Cambridge University Press:  06 March 2015

Mira Pavlovic ,
Conor Teljeur ,
Beate Wieseler ,
Marianne Klemp ,
Irina Cleemput  and
Mattias Neyt
Mira Pavlovic
Affiliation:
La Haute Autorité de Santé [email protected]
Conor Teljeur
Affiliation:
Health Information and Quality Authority
Beate Wieseler
Affiliation:
Institute for Quality and Efficiency in Health Care
Marianne Klemp
Affiliation:
Norwegian Knowledge Centre for the Health Services
Irina Cleemput
Affiliation:
Belgian Health Care Knowledge Centre; Hasselt University
Mattias Neyt
Affiliation:
Belgian Health Care Knowledge Centre
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Abstract

Objectives: Clinical endpoints are defined as valid measures of clinical benefit or harm due to treatment, that describe the impact of treatment on how a patient feels, functions, and survives. The choice of endpoints and the manner in which they are reported have a major impact on the relative effectiveness assessment (REA) of pharmaceuticals. The aim of this article is to describe the guideline development process and the key findings that set a framework for appropriate use of endpoints in REAs in Europe.

Methods: A multi-health technology assessment (HTA)-agency collaborative process in EUnetHTA JA1 was used to scope, draft, and finalize methodological guidelines for REA in Europe.

Results: Patient-relevant clinical endpoints can be broadly categorized into: mortality, morbidity and health-related quality of life. A clinical endpoint is a main symptom or sign of a disease that is clinically relevant, valid, reproducible and responsive to change. Preference is for long-term or final endpoints whenever possible. Surrogate endpoints may be used when there is compelling evidence of a clear and consistent correlation of treatment effects on the surrogate and final outcome of interest.

Conclusions: The relevance and hierarchy of the different types of clinical endpoints depend on the research question, disease, and the treatment investigated. Not only the primary endpoint, but also other relevant endpoints are assessed in comparison to adequate comparator(s). This simultaneous assessment of all relevant endpoints is a hallmark of REA.

Keywords

Relative effectiveness assessmentClinical endpointsPatient-relevant endpoints
Type
Theme Submissions
Information
International Journal of Technology Assessment in Health Care , Volume 30 , Issue 5 , November 2014 , pp. 508 - 513
Copyright
Copyright © Cambridge University Press 2015 

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