Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) Treatment for Spinal Muscular Atrophy I in The Netherlands With Relapse Scenarios
Objectives: Onasemnogene Abeparvovec-xioi (AVXS-101) is a gene therapy intended for curative treatment of spinal muscular atrophy (SMA) with an expected price of around €2 000 000. The goal of this study is to perform a cost-effectiveness analysis of treatment of SMA I patients with AVXS-101 in The Netherlands including relapse scenarios. Methods: An individual-based state-transition model was used to model treatment effect and survival of SMA I patients treated with AVXS-101, nusinersen and best supportive care (BSC). The model included five health states: three health states according to SMA... Mehr ...
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Dokumenttyp: | Artikel |
Erscheinungsdatum: | 2021 |
Schlagwörter: | advanced therapy medicinal product / AVXS-101 / cost-effectiveness / gene therapy / health technology assessment / microsimulation / relapse / spinal muscular atrophy / Zolgensma / Health Policy / Public Health / Environmental and Occupational Health |
Sprache: | Englisch |
Permalink: | https://search.fid-benelux.de/Record/base-26836470 |
Datenquelle: | BASE; Originalkatalog |
Powered By: | BASE |
Link(s) : | https://dspace.library.uu.nl/handle/1874/412690 |