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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| Format: | Article |
| Language: | English |
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MDPI AG
2019-01-01
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| Series: | Journal of Market Access & Health Policy |
| Subjects: | |
| Online Access: | http://dx.doi.org/10.1080/20016689.2019.1601484 |
| _version_ | 1797229462314549248 |
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| author | Daniel C. Malone Rebecca Dean Ramesh Arjunji Ivar Jensen Phil Cyr Beckley Miller Benit Maru Douglas M. Sproule Douglas E. Feltner Omar Dabbous |
| author_facet | Daniel C. Malone Rebecca Dean Ramesh Arjunji Ivar Jensen Phil Cyr Beckley Miller Benit Maru Douglas M. Sproule Douglas E. Feltner Omar Dabbous |
| author_sort | Daniel C. Malone |
| collection | DOAJ |
| description | 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 ≤$5M. Conclusion: Single-dose AVXS-101 was cost-effective compared to chronic nusinersen for SMA1 patients. |
| first_indexed | 2024-04-24T15:12:58Z |
| format | Article |
| id | doaj.art-930a9177e655400d90828ef1a4362fd9 |
| institution | Directory Open Access Journal |
| issn | 2001-6689 |
| language | English |
| last_indexed | 2024-04-24T15:12:58Z |
| publishDate | 2019-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj.art-930a9177e655400d90828ef1a4362fd92024-04-02T09:58:13ZengMDPI AGJournal of Market Access & Health Policy2001-66892019-01-017110.1080/20016689.2019.16014841601484Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patientsDaniel C. Malone0Rebecca Dean1Ramesh Arjunji2Ivar Jensen3Phil Cyr4Beckley Miller5Benit Maru6Douglas M. Sproule7Douglas E. Feltner8Omar Dabbous9University of ArizonaPrecision XtractAveXis, Inc.Precision XtractPrecision XtractPrecision XtractSSI StrategyAveXis, Inc.AveXis, Inc.AveXis, Inc.Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy for SMA1. Study design: A Markov model was used to estimate the incremental cost-effectiveness ratio (ICER), expressed as cost/quality-adjusted life year ($/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.http://dx.doi.org/10.1080/20016689.2019.1601484avxs-101cost-effectivenessgene-replacement therapynusinersenonasemnogene abeparvovecspinal muscular atrophy type 1 |
| spellingShingle | Daniel C. Malone Rebecca Dean Ramesh Arjunji Ivar Jensen Phil Cyr Beckley Miller Benit Maru Douglas M. Sproule Douglas E. Feltner Omar Dabbous Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients Journal of Market Access & Health Policy avxs-101 cost-effectiveness gene-replacement therapy nusinersen onasemnogene abeparvovec spinal muscular atrophy type 1 |
| title | Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients |
| title_full | Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients |
| title_fullStr | Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients |
| title_full_unstemmed | Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients |
| title_short | Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients |
| title_sort | cost effectiveness analysis of using onasemnogene abeparvocec avxs 101 in spinal muscular atrophy type 1 patients |
| topic | avxs-101 cost-effectiveness gene-replacement therapy nusinersen onasemnogene abeparvovec spinal muscular atrophy type 1 |
| url | http://dx.doi.org/10.1080/20016689.2019.1601484 |
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