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在高氏病1型和3b型中评估酶替代疗法的经济价值:扩大覆盖范围是否合理?

Waranya Rattanavipapong¹, Thunyarat Anothaisintawee², Wanrudee Isaranuwatchai^3,4

¹Health Intervention and Technology Assessment Program, Department of Health, Ministry of Public Health, 6th Floor, 6th Building, Tiwanon Rd., Mueang, Nonthaburi, 11000, Thailand. waranya.r@hitap.net.

PharmacoEconomics - open

|April 29, 2025

概括

在泰国扩大对高氏病1型和3b型的酶替代疗法 (ERT) 并不具有成本效益. 降低成本或增加造血干细胞移植 (HSCT) 可能会提高经济可行性.

科学领域:

卫生经济学卫生经济学
药物经济学药物经济学
罕见疾病管理罕见疾病管理

背景情况:

氏病是一种罕见的遗传性疾病,需要昂贵的治疗方法,如酶替代疗法 (ERT).
泰国目前的政策仅针对高氏病1型的ERT (imiglucerase) 进行治疗.
对类型1和3b的ERT扩展进行评估对于公共卫生系统规划至关重要.

研究的目的:

对于高氏病1型和3b型的ERT进行成本效益和预算影响分析.
为在泰国公共卫生系统中扩大ERT覆盖范围的决策提供信息.
为了比较当前与拟议的ERT政策.

主要方法:

决策树和马尔科夫模型被用于对整个生命周期的成本效益分析.
在成本效益分析中采用了社会视角.
根据泰国卫生技术评估指南,进行了为期五年的预算影响分析.

主要成果:

在高氏病1型和3b型的ERT中,增加性成本效益比率 (ICER) 显著超过泰国的成本效益值.
扩大ERT覆盖范围将导致相当大的额外预算负担.
增加血造干细胞移植 (HSCT) 率可以提高成本效益.

结论:

在泰国,以当前价格扩大高氏病类型1和3b的ERT并非具有成本效益.
通过降低药品价格约60%的成本效益可以实现.
对于符合条件的患者来说,通用HSCT为成本效益提供了一个替代途径.

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