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. 2022 Dec;269(12):6544-6554.
doi: 10.1007/s00415-022-11319-0. Epub 2022 Aug 18.

Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type I in Australia

Tianjiao Wang  1   2 , Paul Scuffham  3   4 , Joshua Byrnes  3   4 , Martin Downes  3   4
Affiliations

Cost-effectiveness analysis of gene-based therapies for patients with spinal muscular atrophy type I in Australia

Tianjiao Wang et al. J Neurol. 2022 Dec.

Abstract

Introduction: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder and regarded as one of the most frequent genetic causes of infant mortality. The aim of this study is to develop a cost-effectiveness analysis of AVXS-101 (Onasemnogene Abeparvovec/Zolgensma®) and nusinersen (Spinraza®) for SMA to inform decision-making on reimbursement policies in Australia.

Methods: A Markov model was developed with five health states to evaluate the costs and effects for patients with SMA Type I from a healthcare system perspective over a time-horizon of 100 years. The model parameters were based on clinical trials, parametric distributions, published literature, and Australian registries. One-way and probabilistic sensitivity analysis were performed to appraise the uncertainties of the parameters in the model. A threshold analysis was conducted to estimate the cost of AVXS-101 of being cost-effective.

Results: The incremental cost-effectiveness ratio (ICER) of AVXS-101 was $1,808,471 per quality-adjusted life year (QALY) and that of nusinersen was $2,772,798 per QALY, compared to standard of care, respectively. The ICER of AVXS-101 was $1,238,288 per QALY compared to nusinersen. The key drivers influencing on ICERs were costs of using treatments and utility values of sitting and walking independently.

Conclusion: Both nusinersen and AVXS-101 resulted in health benefits, but they were not cost-effective with a commonly used willingness-to-pay (WTP) threshold of $50,000 per QALY. Developing high-quality clinical data and exploring appropriate WTP thresholds are critical for decision-making on reimbursement policies in the treatment of rare diseases.

Keywords: Cost-effectiveness analysis; Genetic therapy; Nusinersen; Onasemnogene abeparvovec; Rare disease; Spinal muscular atrophy.

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Conflict of interest statement

The authors declare that they have no conflict of interest.

Figures

Fig. 1
Fig. 1
Structure of Markov model in the analysis
Fig. 2
Fig. 2
Cost-effectiveness scatterplot of AVXS-101, nusinersen and SOC
Fig. 3
Fig. 3
Cost-effectiveness acceptability curve of AVXS-101, nusinersen and SOC
See this image and copyright information in PMC

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