Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients. Issue 1 (1st January 2019)

• Record Type: Journal Article
• Authors: Malone, Daniel C.; Dean, Rebecca; Arjunji, Ramesh; Jensen, Ivar; Cyr, Phil; Miller, Beckley; Maru, Benit; Sproule, Douglas M.; Feltner, Douglas E.; Dabbous, Omar
• Is Part Of: Journal of market access & health policy. Volume 7:Issue 1(2019)
• Publication Date: 2019-01-01
• Subjects: AVXS-101 -- cost-effectiveness -- gene-replacement therapy -- nusinersen -- onasemnogene abeparvovec -- spinal muscular atrophy type 1; Medical policy -- Periodicals; Medical policy -- Economic aspects -- Periodicals; International trade -- Environmental aspects -- Periodicals; Health Policy -- economics; Technology Assessment, Biomedical; Marketing of Health Services; International trade -- Environmental aspects; Medical policy; Periodicals; Periodicals; Fulltext; Internet Resources; Periodicals; 362.1
• DOI: 10.1080/20016689.2019.1601484
• Languages: English
• ISSNs: 2001-6689

Abstract

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.