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

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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.

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Most cited references 22

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Improved curve fits to summary survival data: application to economic evaluation of health technologies

Martin W Hoyle, William Edward Henley (2011)

Background Mean costs and quality-adjusted-life-years are central to the cost-effectiveness of health technologies. They are often calculated from time to event curves such as for overall survival and progression-free survival. Ideally, estimates should be obtained from fitting an appropriate parametric model to individual patient data. However, such data are usually not available to independent researchers. Instead, it is common to fit curves to summary Kaplan-Meier graphs, either by regression or by least squares. Here, a more accurate method of fitting survival curves to summary survival data is described. Methods First, the underlying individual patient data are estimated from the numbers of patients at risk (or other published information) and from the Kaplan-Meier graph. The survival curve can then be fit by maximum likelihood estimation or other suitable approach applied to the estimated individual patient data. The accuracy of the proposed method was compared against that of the regression and least squares methods and the use of the actual individual patient data by simulating the survival of patients in many thousands of trials. The cost-effectiveness of sunitinib versus interferon-alpha for metastatic renal cell carcinoma, as recently calculated for NICE in the UK, is reassessed under several methods, including the proposed method. Results Simulation shows that the proposed method gives more accurate curve fits than the traditional methods under realistic scenarios. Furthermore, the proposed method achieves similar bias and mean square error when estimating the mean survival time to that achieved by analysis of the complete underlying individual patient data. The proposed method also naturally yields estimates of the uncertainty in curve fits, which are not available using the traditional methods. The cost-effectiveness of sunitinib versus interferon-alpha is substantially altered when the proposed method is used. Conclusions The method is recommended for cost-effectiveness analysis when only summary survival data are available. An easy-to-use Excel spreadsheet to implement the method is provided.

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A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients.

Klaus Zerres, Sabine Rudnik-Schöneborn, Eric Forrest … (1997)

We analyzed clinical data of 569 patients in two combined series with childhood and juvenile proximal SMA. This cohort included only patients who had achieved the ability to sit unaided (type II and III SMA). The survival rate among 240 type II patients (who sat but never walked) was 98.5% at 5 years and 68.5% at 25 years. SMA III (n = 329) (those who walked and had symptoms before age 30 years) was subdivided into those with an onset before and after age 3 years (type IIIa, n = 195; SMA IIIb, n = 134). In patients with SMA III, life expectancy is not significantly less than a normal population. The probabilities of being able to walk at 10 years after onset was 70.3%, and at 40 years, 22.0% in SMA IIa. For SMA IIIb, 96.7% were walking 10 years after onset and 58.7% at 40 years. The subdivision of type III SMA was justified by the probability of being ambulatory depending on age at onset; the prognosis differed for those with onset before or after age 3 years. The data provide a reliable basis of the natural history of proximal SMA and support a classification system that is based primarily on age at onset and the achievement of motor milestones.

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Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain

Julio López-Bastida, Luz Peña-Longobardo, Isaac Aranda-Reneo … (2017)

Background The aim of this study was to determine the economic burden and health-related quality of life (HRQOL) of patients with Spinal Muscular Atrophy (SMA) and their caregivers in Spain. Methods This was a cross-sectional and retrospective study of patients diagnosed with SMA in Spain. We adopted a bottom up, prevalence approach design to study patients with SMA. The patient’s caregivers completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and non-healthcare services. Costs were estimated from a societal perspective (including healthcare costs and non-healthcare costs), and health-related quality of life (HRQOL) was assessed using the EQ-5D questionnaire. The main caregivers also answered a questionnaire on their characteristics and on their HRQOL. Results A total of 81 caregivers of patients with different subtypes of SMA completed the questionnaire. Based on the reference unitary prices for 2014, the average annual costs per patient were € 33,721. Direct healthcare costs were € 10,882 (representing around 32.3% of the total cost) and the direct non-healthcare costs were € 22,839 (67.7% of the total cost). The mean EQ-5D social tariff score for patients was 0.16, and the mean score of the EQ-5D visual analogue scale was 54. The mean EQ-5D social tariff score for caregivers was 0.49 and their mean score on the EQ-5D visual analogue scale was 69. Conclusion The results highlight the burden that SMA has in terms of costs and decreased HRQOL, not only for patients but also for their caregivers. In particular, the substantial social/economic burden is mostly attributable to the high direct non-healthcare costs.

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Author and article information

Journal

Journal ID (nlm-ta): J Mark Access Health Policy

Journal ID (iso-abbrev): J Mark Access Health Policy

Journal ID (publisher-id): ZJMA

Journal ID (publisher-id): zjma20

Title: Journal of Market Access & Health Policy

Publisher: Routledge

ISSN (Electronic): 2001-6689

Publication date Collection: 2019

Publication date (Electronic): 08 May 2019

Volume: 7

Issue: 1

Electronic Location Identifier: 1601484

Affiliations

[a ]College of Pharmacy, University of Arizona , Tucson, AZ, USA

[b ]Precision Xtract , Boston, MA, USA

[c ]AveXis, Inc. , Bannockburn, IL, USA

[d ]SSI Strategy , Parsippany, NJ, USA

Author notes

CONTACT Ramesh Arjunji RArjunji444@ 123456avexis.com AveXis, Inc , Bannockburn, IL, USA

Article

Publisher ID: 1601484

DOI: 10.1080/20016689.2019.1601484

PMC ID: 6508058

PubMed ID: 31105909

SO-VID: f35217eb-4838-4550-ac9b-5ff6d1f5c4fe

License:

This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License ( http://creativecommons.org/licenses/by-nc/4.0/), which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.

History

Date received : 21 February 2019

Date revision received : 25 March 2019

Date accepted : 27 March 2019

Page count

Figures: 4, Tables: 10, References: 49, Pages: 15

Funding

Funded by: AveXis, Inc

This work was supported by AveXis, Inc., a Novartis company.

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

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Most cited references 22

Improved curve fits to summary survival data: application to economic evaluation of health technologies

A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients.

Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain

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