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泰国严重眼睑痉挛患者中A型肉毒杆菌毒素与口服药物治疗的成本效益分析

Cost-utility analysis of botulinum toxin type A versus oral drug treatment in patients with severe blepharospasm in Thailand.

作者信息

Hirunwiwatkul Parima, Permsuwan Unchalee, Ngamkiatphaisan Sureerat, Chirapapaisan Niphon, Sriratanaban Jiruth

机构信息

Department of Ophthalmology, Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand.

Department of Ophthalmology, King Chulalongkorn Memorial Hospital, Thai Red Cross Society, Bangkok, Thailand.

出版信息

PLoS One. 2025 Apr 21;20(4):e0319926. doi: 10.1371/journal.pone.0319926. eCollection 2025.

Abstract

BACKGROUND

Blepharospasm is a chronic facial movement disorder affecting a person's ability to work, causing depression, pain, and a reduced quality of life (QoL). Botulinum toxin type A (BoNT-A) treatment can improve these conditions; however, its cost remains a significant barrier for inclusion of this indication into the Thai National List of Essential Medicine.

OBJECTIVES

This study aimed to assess the cost-effectiveness of onabotulinumtoxinA (onaBoNT-A) and abobotulinumtoxinA (aboBoNT-A) treatment compared to oral medication treatment in patients with severe blepharospasm from a societal perspective.

METHODS

A cost-utility analysis using a two-part model was conducted to analyze lifetime costs and quality-adjusted life-years (QALYs). Inputs were mainly obtained from real-world evidence of 159 Thai patients with blepharospasm. Costs and outcomes were discounted at 3% annually and presented as 2023 value. Incremental cost-effectiveness ratios (ICERs) were estimated. Deterministic and probabilistic sensitivity analyses were also conducted.

RESULTS

In comparison to standard oral medication, both onaBoNT-A and aboBoNT-A incurred greater lifetime cost (3,055 USD, 2,889 USD vs 1,926 USD) while gaining additional QALYs (6.94 years, 6.94 years vs 6.53 years). The estimated ICERs were 2,722 USD/QALY for onaBoNT-A and 2,323 USD/QALY for aboBoNT-A. Utility and cost of BoNT-A were important determinants in the sensitivity analysis.

CONCLUSION

Among patients with severe blepharospasm, both onaBoNT-A and aboBoNT-A were considered a cost-effective strategy under the Thai willingness to pay threshold of 4,613 USD/QALY. Having aboBoNT-A was slightly more favorable due to lower cost, using a conversion ratio of 1U of onaBoNT-A: 3U of aboBoNT-A.

摘要

背景

眼睑痉挛是一种慢性面部运动障碍,会影响患者的工作能力,导致抑郁、疼痛,并降低生活质量(QoL)。A型肉毒杆菌毒素(BoNT-A)治疗可改善这些状况;然而,其成本仍是将该适应症纳入泰国国家基本药物清单的重大障碍。

目的

本研究旨在从社会角度评估与口服药物治疗相比,注射用A型肉毒毒素(onaBoNT-A)和A型肉毒杆菌毒素(aboBoNT-A)治疗重度眼睑痉挛患者的成本效益。

方法

采用两部分模型进行成本效用分析,以分析终身成本和质量调整生命年(QALYs)。数据主要来自159名泰国眼睑痉挛患者的真实世界证据。成本和结果按每年3%进行贴现,并以2023年的价值呈现。估计了增量成本效益比(ICERs)。还进行了确定性和概率性敏感性分析。

结果

与标准口服药物相比,onaBoNT-A和aboBoNT-A的终身成本更高(分别为3055美元、2889美元和1926美元),但获得了更多的QALYs(分别为6.94年、6.94年和6.53年)。估计onaBoNT-A的ICER为2722美元/QALY,aboBoNT-A为2323美元/QALY。在敏感性分析中,BoNT-A的效用和成本是重要的决定因素。

结论

在重度眼睑痉挛患者中,根据泰国每QALY支付意愿阈值4613美元,onaBoNT-A和aboBoNT-A均被认为是具有成本效益的策略。由于成本较低,使用1单位onaBoNT-A:3单位aboBoNT-A的换算比例,aboBoNT-A略占优势。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1514/12011269/b6be8f2527ca/pone.0319926.g001.jpg

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