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Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
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Article

Cost-Effectiveness Analysis of Using Onasemnogene Abeparvocec (AVXS-101) in Spinal Muscular Atrophy Type 1 Patients

by
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 and
Omar Dabbous
3
1
College of Pharmacy, University of Arizona, Tucson, AZ, USA
2
Precision Xtract, Boston, MA, USA
3
AveXis, Inc., Bannockburn, IL, USA
4
SSI Strategy, Parsippany, NJ, USA
J. Mark. Access Health Policy 2019, 7(1), 1601484; https://doi.org/10.1080/20016689.2019.1601484
Submission received: 21 February 2019 / Revised: 25 March 2019 / Accepted: 27 March 2019 / Published: 8 May 2019

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

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MDPI and ACS Style

Malone, D.C.; Dean, R.; Arjunji, R.; Jensen, I.; Cyr, P.; Miller, B.; Maru, B.; Sproule, D.M.; Feltner, D.E.; Dabbous, O. Cost-Effectiveness Analysis of Using Onasemnogene Abeparvocec (AVXS-101) in Spinal Muscular Atrophy Type 1 Patients. J. Mark. Access Health Policy 2019, 7, 1601484. https://doi.org/10.1080/20016689.2019.1601484

AMA Style

Malone DC, Dean R, Arjunji R, Jensen I, Cyr P, Miller B, Maru B, Sproule DM, Feltner DE, Dabbous O. Cost-Effectiveness Analysis of Using Onasemnogene Abeparvocec (AVXS-101) in Spinal Muscular Atrophy Type 1 Patients. Journal of Market Access & Health Policy. 2019; 7(1):1601484. https://doi.org/10.1080/20016689.2019.1601484

Chicago/Turabian Style

Malone, Daniel C., Rebecca Dean, Ramesh Arjunji, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, and Omar Dabbous. 2019. "Cost-Effectiveness Analysis of Using Onasemnogene Abeparvocec (AVXS-101) in Spinal Muscular Atrophy Type 1 Patients" Journal of Market Access & Health Policy 7, no. 1: 1601484. https://doi.org/10.1080/20016689.2019.1601484

APA Style

Malone, D. C., Dean, R., Arjunji, R., Jensen, I., Cyr, P., Miller, B., Maru, B., Sproule, D. M., Feltner, D. E., & Dabbous, O. (2019). Cost-Effectiveness Analysis of Using Onasemnogene Abeparvocec (AVXS-101) in Spinal Muscular Atrophy Type 1 Patients. Journal of Market Access & Health Policy, 7(1), 1601484. https://doi.org/10.1080/20016689.2019.1601484

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