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. 2019 May 8;7(1):1601484.
doi: 10.1080/20016689.2019.1601484. eCollection 2019.

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

Daniel C Malone  1 , Rebecca Dean  2 , Ramesh Arjunji  3 , Ivar Jensen  2 , Phil Cyr  2 , Beckley Miller  2 , Benit Maru  4 , Douglas M Sproule  3 , Douglas E Feltner  3 , Omar Dabbous  3
Affiliations

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

Daniel C Malone et al. J Mark Access Health Policy. .

Abstract

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.

Keywords: AVXS-101; cost-effectiveness; gene-replacement therapy; nusinersen; onasemnogene abeparvovec; spinal muscular atrophy type 1.

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Figures

Figure 1.
Figure 1.
Model health states for SMA1. Five functional health states (A-E) and death are shown for SMA1.
Figure 2.
Figure 2.
Patient movement over time through health states. Proportion of patients treated with (A) AVXS-101 or (B) nusinersen over time is shown by health states and cycle.
Figure 3.
Figure 3.
Tornado diagram showing deterministic sensitivity analysis for the top 14 parameters for incremental cost-effectiveness ratio (ICER) of AVXS-101 vs nusinersen. * Parameters with >2% variation are not presented.
Figure 4.
Figure 4.
Probabilistic sensitivity analysis for model parameters relating to cost and utilities using 1,000 simulation runs.
See this image and copyright information in PMC

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