Body of Evidence for Onasemnogene Abeparvovec in Spinal Muscular Atrophy Supports Long-Term Duration of Effect Without Relapse
Nov 1, 2022, 00:00
10.1016/j.jval.2021.10.019
https://www.valueinhealthjournal.com/article/S1098-3015(22)00042-0/fulltext
Title :
Body of Evidence for Onasemnogene Abeparvovec in Spinal Muscular Atrophy Supports Long-Term Duration of Effect Without Relapse
Citation :
https://www.valueinhealthjournal.com/action/showCitFormats?pii=S1098-3015(22)00042-0&doi=10.1016/j.jval.2021.10.019
First page :
1922
Section Title :
LETTER TO THE EDITOR
Open access? :
No
Section Order :
1922
In June 2021, Broekhoff et al published a cost-effectiveness model of onasemnogene abeparvovec, a single-dose gene replacement therapy, for spinal muscular atrophy (SMA) type 1. We would like to provide feedback on the relapse scenarios,
which we consider implausible, even as hypothetical scenarios, and contradictive to published clinical evidence.
Categories :
- Cost-comparison, Effectiveness, Utility, Benefit Analysis
- Economic Evaluation
- Musculoskeletal Disorders
- Specific Diseases & Conditions