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)

• Main Authors: Rebecca Dean (Author), Ivar Jensen (Author), Phil Cyr (Author), Beckley Miller (Author), Benit Maru (Author), Douglas M. Sproule (Author), Douglas E. Feltner (Author), Thomas Wiesner (Author), Daniel C. Malone (Author), Matthias Bischof (Author), Walter Toro (Author), Omar Dabbous (Author)
• Format: Book
• Published: Taylor & Francis Group, 2021-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 Institu...