Early Cost-Effectiveness of Onasemnogene Abeparvovec-xioi (Zolgensma) and Nusinersen (Spinraza) Treatment for Spinal Muscular Atrophy I in The Netherlands With Relapse Scenarios
Broekhoff, Thomas F.; Sweegers, Carly C.G.; Krijkamp, Eline M.; Mantel-Teeuwisse, Aukje K.; Leufkens, Hubert G.M.; Goettsch, Wim G.; Vreman, Rick A.
(2021) Value in Health, volume 24, issue 6, pp. 759 - 769
(Article)
Abstract
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
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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 types, one for permanent ventilation and one for death. Deterministic and probabilistic sensitivity analyses were performed. Effects of relapsing to lower health states in the years following treatment was explored. Results: The base-case incremental cost-effectiveness ratio (ICER) for AVXS-101 versus BSC is €138 875/QALY, and €53 447/QALY for AVXS-101 versus nusinersen. If patients relapse within 10 years after treatment with AVXS-101, the ICER can increase up to 6-fold, with effects diminishing thereafter. Only relapses occurring later than 50 years after treatment have a negligible effect on the ICER. To comply with Dutch willingness-to-pay reference values, the price of AVXS-101 must decrease to €680 000. Conclusions: Based on this model, treatment with AVXS-101 is unlikely to be cost-effective under Dutch willingness-to-pay reference values. Uncertainty regarding the long-term curative properties of AVXS-101 can result in multiplication of the ICER. Decision-makers are advised to appropriately balance these uncertainties against the price they are willing to pay now.
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Keywords: 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
ISSN: 1098-3015
Publisher: Elsevier Ltd
Note: Funding Information: Author Contributions: Concept and design: Mantel-Teeuwisse, Leufkens, Goettsch, Vreman, Acquisition of data: Broekhoff, Analysis and interpretation of data: Broekhoff, Krijkamp, Vreman, Drafting of the manuscript: Broekhoff, Sweegers, Krijkamp, Mantel-Teeuwisse, Leufkens, Goettsch, Vreman, Critical revision of the paper for important intellectual content: Broekhoff, Sweegers, Krijkamp, Mantel-Teeuwisse, Leufkens, Goettsch, Vreman, Statistical analysis: Broekhoff, Vreman, Supervision: Mantel-Teeuwisse, Leufkens, Goettsch, Vreman, Other (critical revision of the paper from an HTA perspective): Sweegers, Conflict of Interest Disclosures: Dr Leufkens is a member of the Lygature Leadership Team. No other disclosures were reported. Funding/Support: Ms Krijkamp was supported by the Society for Medical Decision Making fellowship through a grant by the Gordon and Betty Moore Foundation, United States (GBMF7853). Role of the Funder/Sponsor: The funder had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication. Funding Information: Funding/Support: Ms Krijkamp was supported by the Society for Medical Decision Making fellowship through a grant by the Gordon and Betty Moore Foundation, United States ( GBMF7853 ). Publisher Copyright: © 2021 ISPOR–The Professional Society for Health Economics and Outcomes Research
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