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伊朗crizanlizumab治疗镰状细胞病的成本效益分析。

Cost-Effectiveness Analysis of Crizanlizumab in Sickle Cell Disease in Iran.

作者信息

Nosrati Marzieh, Izadidehkordi Shadi, Nikfar Shekoufeh, Heydari Fatemeh Sadat

机构信息

Personalized Medicine Research Center, Endocrinology and Metabolism Research Institute, Tehran University of Medical Sciences, Tehran, Iran.

Department of Pharmacoeconomics and Pharmaceutical Administration, School of Pharmacy, Tehran University of Medical Sciences, Tehran, Iran.

出版信息

Iran J Med Sci. 2025 Aug 1;50(8):548-555. doi: 10.30476/ijms.2025.102567.3573. eCollection 2025 Aug.

Abstract

BACKGROUND

Sickle cell disease (SCD) prevalence is predicted to rise dramatically in the upcoming years. Although several medications have received Food and Drug Administration (FDA) approval in recent years, low- and middle-income countries (LMICs) still struggle to access these medications due to their remarkably high prices. Crizanlizumab, owing to its clinical and economic privileges, appears to be the most suitable option for addition to pharmacotherapy guidelines. However, no study has yet investigated its cost-effectiveness in Iran's healthcare system.

METHODS

This cost-effectiveness evaluation was conducted in 2022 at the Pharmacoeconomic and Pharmaceutical Administration Department of the Faculty of Pharmacy at Tehran University of Medical Sciences, Tehran, Iran. A decision-tree model was designed, followed by a cost-utility analysis for crizanlizumab in two separate scenarios, targeting not only monotherapy with crizanlizumab in SCD compared with placebo, but also crizanlizumab's concomitant use with hydroxyurea compared with hydroxyurea. The study reports the outcomes from Iran's healthcare system perspective. Direct medical costs, quality-adjusted life years related to vaso-occlusive crisis, hospitalizations, and adverse effects were calculated. Incremental cost-effectiveness ratios were compared. SUSTAIN trial was the main clinical source for modeling crizanlizumab's effectiveness in SCD. A sensitivity analysis was performed to measure the sensitivity of outcomes to changes in medication costs. Microsoft Excel 2020 was utilized for calculations and modeling.

RESULTS

Concomitant therapy with low-dose crizanlizumab added to hydroxyurea led to the lowest Incremental Cost Effectiveness Ratio (ICER) of 398,881 United States dollars (USD), exceeding Iran's accepted cost-effectiveness threshold. Sensitivity analysis results demonstrate that even a 20% reduction in the price of crizanlizumab does not lead to its cost-effectiveness in Iran.

CONCLUSION

Crizanlizumab administration in sickle cell disease is not found cost-effective in Iran, neither as a monotherapy nor added to hydroxyurea.

摘要

背景

预计未来几年镰状细胞病(SCD)的患病率将大幅上升。尽管近年来有几种药物已获得美国食品药品监督管理局(FDA)的批准,但由于价格极高,低收入和中等收入国家(LMICs)在获取这些药物方面仍面临困难。由于其临床和经济优势,克立硐珠单抗似乎是添加到药物治疗指南中的最合适选择。然而,尚无研究调查其在伊朗医疗保健系统中的成本效益。

方法

2022年在伊朗德黑兰医科大学药学院药物经济学和药物管理系进行了这项成本效益评估。设计了一个决策树模型,随后在两种不同情况下对克立硐珠单抗进行成本效用分析,不仅针对SCD中克立硐珠单抗单药治疗与安慰剂的比较,还针对克立硐珠单抗与羟基脲联合使用与羟基脲的比较。该研究从伊朗医疗保健系统的角度报告结果。计算了直接医疗成本、与血管闭塞性危机、住院和不良反应相关的质量调整生命年。比较了增量成本效益比。SUSTAIN试验是模拟克立硐珠单抗在SCD中有效性的主要临床来源。进行了敏感性分析,以衡量结果对药物成本变化的敏感性。使用Microsoft Excel 2020进行计算和建模。

结果

低剂量克立硐珠单抗与羟基脲联合治疗导致最低的增量成本效益比(ICER)为398,881美元,超过了伊朗认可的成本效益阈值。敏感性分析结果表明,即使克立硐珠单抗价格降低20%,在伊朗也不具有成本效益。

结论

在伊朗,无论是单药治疗还是与羟基脲联合使用,克立硐珠单抗治疗镰状细胞病均未显示出成本效益。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fc98/12374056/b7cfef391b5c/IJMS-50-8-548-g001.jpg

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