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

• Main Authors: Samuel Aballéa (Author), Katia Thokagevistk (Author), Rimma Velikanova (Author), Steven Simoens (Author), Lieven Annemans (Author), Fernando Antonanzas (Author), Pascal Auquier (Author), Clément François (Author), Frank-Ulrich Fricke (Author), Daniel Malone (Author), Aurélie Millier (Author), Ulf Persson (Author), Stavros Petrou (Author), Omar Dabbous (Author), Maarten Postma (Author), Mondher Toumi (Author)
• Format: Book
• Published: Taylor & Francis Group, 2020-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...