A Model-Based Economic Evaluation of Hypothetical Treatments for Amyotrophic Lateral Sclerosis in the UK: Implications for Pricing of New and Emerging Health Technologies

Paul Tappenden (), Orla Hardiman, Sun-Hong Kwon, Mon Mon-Yee, Miriam Galvin and Christopher McDermott
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Paul Tappenden: SCHARR, University of Sheffield
Orla Hardiman: Trinity College Dublin
Sun-Hong Kwon: SCHARR, University of Sheffield
Mon Mon-Yee: SCHARR, University of Sheffield
Miriam Galvin: Trinity College Dublin
Christopher McDermott: University of Sheffield

PharmacoEconomics, 2024, vol. 42, issue 9, No 5, 1003-1016

Abstract: Abstract Background Amyotrophic lateral sclerosis (ALS) is a devastating disease which leads to loss of muscle function and paralysis. Historically, clinical drug development has been unsuccessful, but promising disease-modifying therapies (DMTs) may be on the horizon. Objectives The aims of this study were to estimate survival, quality-adjusted life-years (QALYs) and costs under current care, and to explore the conditions under which new therapies might be considered cost effective. Methods We developed a health economic model to evaluate the cost effectiveness of future ALS treatments from a UK National Health Service and Personal Social Services perspective over a lifetime horizon using data from the ALS-CarE study. Costs were valued at 2021/22 prices. Two hypothetical interventions were evaluated: a DMT which delays progression and mortality, and a symptomatic therapy which improves utility only. Sensitivity analysis was conducted to identify key drivers of cost effectiveness. Results Starting from King’s stage 2, patients receiving current care accrue an estimated 2.27 life-years, 0.75 QALYs and lifetime costs of £68,047. Assuming a 50% reduction in progression rates and a UK-converted estimate of the price of edaravone, the incremental cost-effectiveness ratio for a new DMT versus current care is likely to exceed £735,000 per QALY gained. Symptomatic therapies may be more likely to achieve acceptable levels of cost effectiveness. Conclusions Regardless of efficacy, DMTs may struggle to demonstrate cost effectiveness, even at a low price. The cost effectiveness of DMTs is likely to be strongly influenced by drug price, the magnitude and durability of relative treatment effects, treatment starting/stopping rules and any additional utility benefits over current care.

Date: 2024
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DOI: 10.1007/s40273-024-01395-7

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