Health economic evaluation of gene replacement therapies: methodological issues and recommendations

Health economic evaluation of gene replacement therapies: methodological issues and recommendations

Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects...

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Main Authors: Samuel Aballéa, Katia Thokagevistk, Rimma Velikanova, Steven Simoens, Lieven Annemans, Fernando Antonanzas, Pascal Auquier, Clément François, Frank-Ulrich Fricke, Daniel Malone, Aurélie Millier, Ulf Persson, Stavros Petrou, Omar Dabbous, Maarten Postma, Mondher Toumi
Format: Article
Language:English
Published: MDPI AG 2020-01-01
Series:Journal of Market Access & Health Policy
Subjects:
gene replacement therapy
guidelines
health economic evaluation
methods
cost-effectiveness analysis
qaly
Online Access:http://dx.doi.org/10.1080/20016689.2020.1822666
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