Endpoints for relative effectiveness assessment (REA) of pharmaceuticals

International Journal of Technology Assessment in Health Care
Mira PavlovicMattias Neyt

Abstract

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. 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. 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. The relevance and hierarchy of the different types of clinical endpoints d...Continue Reading

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Citations

Aug 25, 2015·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Sarah KleijnenWim G Goettsch
Dec 10, 2016·Transplantation Proceedings·B Díaz-MolinaC Morís
Mar 10, 2015·International Journal of Technology Assessment in Health Care·Sarah KleijnenWim G Goettsch
Aug 15, 2017·Journal of Market Access & Health Policy·Krzysztof LachMondher Toumi
Jan 13, 2018·Nature Reviews. Neurology·Carmen TurOlga Ciccarelli
Aug 24, 2019·The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care·Livio Garattini, Anna Padula

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