An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. Objective: To compare the manufacturer’s proprietary CUA model to the model published by the Institute fo...

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Bibliographic Details
Main Authors: Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:Journal of Market Access & Health Policy
Subjects:
cost-effectiveness analysis
gene therapy
spinal muscular atrophy
onasemnogene abeparvovec
cost-utility analysis
nusinersen
Online Access:http://dx.doi.org/10.1080/20016689.2021.1889841
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http://dx.doi.org/10.1080/20016689.2021.1889841

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