Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis

Abstract Background Edaravone has been widely used in amyotrophic lateral sclerosis (ALS) treatment, and a sublingual (SL) tablet has been developed to offer a more convenient alternative for injection. We present a cost-utility analysis to comprehensively evaluate the costs and health outcomes of o...

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Main Authors: Chang Liu, Yao Wu, Fangxu Wang, Shuang Sun, Jiayin Wei, Libo Tao
Format: Article
Language:English
Published: BMC 2024-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Online Access:https://doi.org/10.1186/s13023-024-03381-w
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author Chang Liu
Yao Wu
Fangxu Wang
Shuang Sun
Jiayin Wei
Libo Tao
author_facet Chang Liu
Yao Wu
Fangxu Wang
Shuang Sun
Jiayin Wei
Libo Tao
author_sort Chang Liu
collection DOAJ
description Abstract Background Edaravone has been widely used in amyotrophic lateral sclerosis (ALS) treatment, and a sublingual (SL) tablet has been developed to offer a more convenient alternative for injection. We present a cost-utility analysis to comprehensively evaluate the costs and health outcomes of oral and intravenous edaravone for the treatment of ALS in Chinese medical context. Methods Cost-utility analysis of SL tablets of edaravone versus intravenous edaravone at home was performed by constructing a 20-year Markov model of ALS stage 1–4 and death. The data were extracted from the literature with model assumptions. Typical sensitivity analysis and scenario analysis for administering SL tablets at home versus intravenous tablets at the hospital were performed. Results In the base case analysis, with SL tablets and intravenous injections both at home, the model estimated an additional cost of ¥12,670.04 and an additional 0.034 QALYs over 20 years (life time) of modeling analysis, and the ICER was ¥372,648.24 per QALY. However, in the scenario of intravenous administration at the hospital, SL tablet was demonstrated dominance to intravenous injection. Conclusions Using 3 times the GDP per capita of China in 2023 as the threshold, the SL tablet edaravone was not cost-effective in the context of home treatment for both formulationst, but was dominance to intravenous injection in hospital treatment. The results highlighted the importance of treatment context for health economic analysis.
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spelling doaj-art-4004723ccda440c4a08e67a9d2245caf2025-08-20T02:18:33ZengBMCOrphanet Journal of Rare Diseases1750-11722024-10-011911810.1186/s13023-024-03381-wCost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosisChang Liu0Yao Wu1Fangxu Wang2Shuang Sun3Jiayin Wei4Libo Tao5Center for Health Policy and Technology Evaluation, Peking University Health Science CenterCenter for Health Policy and Technology Evaluation, Peking University Health Science CenterCenter for Health Policy and Technology Evaluation, Peking University Health Science CenterCenter for Health Policy and Technology Evaluation, Peking University Health Science CenterCenter for Health Policy and Technology Evaluation, Peking University Health Science CenterCenter for Health Policy and Technology Evaluation, Peking University Health Science CenterAbstract Background Edaravone has been widely used in amyotrophic lateral sclerosis (ALS) treatment, and a sublingual (SL) tablet has been developed to offer a more convenient alternative for injection. We present a cost-utility analysis to comprehensively evaluate the costs and health outcomes of oral and intravenous edaravone for the treatment of ALS in Chinese medical context. Methods Cost-utility analysis of SL tablets of edaravone versus intravenous edaravone at home was performed by constructing a 20-year Markov model of ALS stage 1–4 and death. The data were extracted from the literature with model assumptions. Typical sensitivity analysis and scenario analysis for administering SL tablets at home versus intravenous tablets at the hospital were performed. Results In the base case analysis, with SL tablets and intravenous injections both at home, the model estimated an additional cost of ¥12,670.04 and an additional 0.034 QALYs over 20 years (life time) of modeling analysis, and the ICER was ¥372,648.24 per QALY. However, in the scenario of intravenous administration at the hospital, SL tablet was demonstrated dominance to intravenous injection. Conclusions Using 3 times the GDP per capita of China in 2023 as the threshold, the SL tablet edaravone was not cost-effective in the context of home treatment for both formulationst, but was dominance to intravenous injection in hospital treatment. The results highlighted the importance of treatment context for health economic analysis.https://doi.org/10.1186/s13023-024-03381-wEdaravoneSublingual tabletAmyotrophic lateral sclerosisCost-utility analysis
spellingShingle Chang Liu
Yao Wu
Fangxu Wang
Shuang Sun
Jiayin Wei
Libo Tao
Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
Orphanet Journal of Rare Diseases
Edaravone
Sublingual tablet
Amyotrophic lateral sclerosis
Cost-utility analysis
title Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
title_full Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
title_fullStr Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
title_full_unstemmed Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
title_short Cost-utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
title_sort cost utility analysis for sublingual versus intravenous edaravone in the treatment of amyotrophic lateral sclerosis
topic Edaravone
Sublingual tablet
Amyotrophic lateral sclerosis
Cost-utility analysis
url https://doi.org/10.1186/s13023-024-03381-w
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