Igarashi Ataru, Inoue Shun, Onishi Yasushi
Department of Health Policy and Public Health, Graduate School of Pharmaceutical Sciences, The University of Tokyo, Tokyo, Japan.
Asahi Kasei Pharma Corporation, Tokyo, Japan.
J Med Econ. 2025 Dec;28(1):460-470. doi: 10.1080/13696998.2025.2483098. Epub 2025 Mar 27.
This study aimed to evaluate the cost-effectiveness of isavuconazole compared with voriconazole as a first-line therapy for patients with invasive aspergillosis prior to differential pathogen diagnosis.
Using a state-transition model, a cost-utility analysis of isavuconazole compared with voriconazole was conducted in patients with presumptive invasive aspergillosis. The study population consisted of patients with hematological malignancies undergoing hematopoietic stem cell transplantation (HSCT) or chemotherapy who developed invasive fungal infections. The incremental cost-effectiveness ratio (ICER) was analyzed from the perspective of public healthcare. In patients with presumptive invasive aspergillosis, 6.6% were assumed to have mucormycosis. Efficacy data were sourced from the SECURE and VITAL trials, which included patients with invasive aspergillosis and mucormycosis. Expected survival was based on data for acute myeloid leukemia. The cost of voriconazole was based on its generic price. Different parameters were set for quality of life, expected survival period, and hospitalization costs in the HSCT and chemotherapy models, and the robustness of the model was evaluated using probabilistic and deterministic sensitivity analyses.
In the HSCT model, the base case showed an incremental quality-adjusted life-years (QALYs) of 0.37 and an incremental cost of JPY 918,682 for isavuconazole compared with voriconazole, with an ICER of JPY 2,515,813. In the chemotherapy model, the incremental QALYs was 0.16, and the incremental cost was JPY 723,111, with an ICER of JPY 4,411,564. The probability sensitivity analysis showed that the proportion of ICERs below JPY 5 million was 100.0% in the HSCT model and 79.1% in the chemotherapy model.
Reference efficacy data were obtained from non-Japanese clinical trials.
Assuming a willingness-to-pay threshold of JPY 5 million for additional QALYs, isavuconazole was shown to be cost-effective compared with voriconazole in both the HSCT and chemotherapy models as a first-line therapy for patients with presumptive invasive aspergillosis.
本研究旨在评估在侵袭性曲霉病患者进行病原菌鉴别诊断之前，与伏立康唑相比，艾沙康唑作为一线治疗药物的成本效益。
采用状态转换模型，对疑似侵袭性曲霉病患者进行了艾沙康唑与伏立康唑的成本效用分析。研究人群包括接受造血干细胞移植（HSCT）或化疗且发生侵袭性真菌感染的血液系统恶性肿瘤患者。从公共卫生保健角度分析增量成本效益比（ICER）。在疑似侵袭性曲霉病患者中，假定6.6%患有毛霉病。疗效数据来源于SECURE和VITAL试验，这些试验纳入了侵袭性曲霉病和毛霉病患者。预期生存期基于急性髓系白血病的数据。伏立康唑的成本基于其仿制药价格。在HSCT和化疗模型中，对生活质量、预期生存期和住院费用设置了不同参数，并使用概率和确定性敏感性分析评估模型的稳健性。
在HSCT模型中，基础病例显示，与伏立康唑相比，艾沙康唑的增量质量调整生命年（QALY）为0.37，增量成本为918,682日元，ICER为2,515,813日元。在化疗模型中，增量QALY为0.16，增量成本为723,111日元，ICER为4,411,564日元。概率敏感性分析显示，在HSCT模型中，ICER低于500万日元的比例为100.0%，在化疗模型中为79.1%。
参考疗效数据来自非日本的临床试验。
假设每增加一个QALY的支付意愿阈值为500万日元，在HSCT和化疗模型中，对于疑似侵袭性曲霉病患者，与伏立康唑相比，艾沙康唑作为一线治疗药物具有成本效益。
