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A Model-Based Economic Evaluation of Hypothetical Treatments for Amyotrophic Lateral Sclerosis in the UK: Implications for Pricing of New and Emerging Health Technologies.
- Source :
-
PharmacoEconomics [Pharmacoeconomics] 2024 Sep; Vol. 42 (9), pp. 1003-1016. Date of Electronic Publication: 2024 May 31. - Publication Year :
- 2024
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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.<br />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.<br />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.<br />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.<br />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.<br /> (© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)
- Subjects :
- Humans
United Kingdom
Disease Progression
Biomedical Technology economics
Technology Assessment, Biomedical
Amyotrophic Lateral Sclerosis economics
Amyotrophic Lateral Sclerosis therapy
Amyotrophic Lateral Sclerosis drug therapy
Cost-Benefit Analysis
Quality-Adjusted Life Years
Models, Economic
Subjects
Details
- Language :
- English
- ISSN :
- 1179-2027
- Volume :
- 42
- Issue :
- 9
- Database :
- MEDLINE
- Journal :
- PharmacoEconomics
- Publication Type :
- Academic Journal
- Accession number :
- 38819717
- Full Text :
- https://doi.org/10.1007/s40273-024-01395-7