Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy...

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Bibliographic Details
Published in:Journal of market access & health policy 2019, Vol.7 (1), p.1601484-1601484
Main Authors: Malone, Daniel C., Dean, Rebecca, Arjunji, Ramesh, Jensen, Ivar, Cyr, Phil, Miller, Beckley, Maru, Benit, Sproule, Douglas M., Feltner, Douglas E., Dabbous, Omar
Format: Article
Language:English
Subjects:
AVXS-101
Cost analysis
cost-effectiveness
Costs
Effectiveness
gene-replacement therapy
Health care expenditures
Infants
Medical treatment
Neuromuscular diseases
nusinersen
onasemnogene abeparvovec
Original
Patients
Quality of life
spinal muscular atrophy type 1
Walking
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Summary:Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy for SMA1. Study design: A Markov model was used to estimate the incremental cost-effectiveness ratio (ICER), expressed as cost/quality-adjusted life year ($/QALY), of AVXS-101 versus nusinersen over a lifetime. Survival, healthcare costs and QALYs were estimated using natural history data for SMA patients who achieved motor milestones (sitting/walking). Health utility weights were obtained from the CHERISH trial. Setting: USA; commercial payer perspective Participants: SMA1 infants Interventions: AVXS-101 was compared to nusinersen. Main outcome measure: The primary outcome was the ICER. Results: Expected survival (undiscounted) over a lifetime predicted by the model was 37.20 life years for AVXS-101 and 9.68 for nusinersen (discounted QALYs, 15.65 and 5.29, respectively). Using a potential AVXS-101 price range ($2.5-5.0M/treatment), the average lifetime cost/patient was $4.2-6.6M for AVXS-101 and $6.3M for nusinersen. The ICER range was (-$203,072) to $31,379 per QALY gained for AVXS-101 versus nusinersen, indicating that AVXS-101 was cost-effective with prices of ≤$5M. Conclusion: Single-dose AVXS-101 was cost-effective compared to chronic nusinersen for SMA1 patients.
ISSN:2001-6689
2001-6689
DOI:10.1080/20016689.2019.1601484