Luo Yashuang, Cheng Wendi, Fu Yuyan, Wang Haode, Wang Haiyin
Shanghai Health Development Research Center (Shanghai Medical Information Center), Shanghai, China.
School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, United Kingdom.
Intractable Rare Dis Res. 2024 Aug 31;13(3):157-164. doi: 10.5582/irdr.2024.01027.
This study aimed to assess the cost-utility of romiplostim (ROMI) compared to eltrombopag (EPAG) as a second-line treatment for chronic primary immune thrombocytopenia (cITP) in Chinese adults. A decision tree-embedded Markov model with a lifetime horizon was used to estimate the quality-adjusted life years (QALYs) and costs for ROMI versus EPAG from the perspective of the Chinese health care system. The model was driven by platelet response with a 4-week cycle. Both QALYs and costs were discounted 5% per year. Clinical data comparing ROMI and EPAG were obtained by matching-adjusted indirect comparison (MAIC), utilizing individual patient data on ROMI and published Chinese Phase III trial data on EPAG. Costs were reported in 2022 US dollars and included drug acquisition costs, monitoring costs, bleeding-related costs, and costs associated with adverse events. Deterministic and probabilistic sensitivity analyses were performed. The CEA model indicated that treatment with ROMI resulted in an average of $4,344.4 higher costs for 0.004 QALYs. One-way sensitivity analysis (OSA) indicated that the model was most sensitive to the high bleeding rate in response (Markov stage) for EPAG and ROMI. Probabilistic sensitivity analysis (PSA) indicated that ROMI was likely to be cost effective in 0.16% cases at a willingness-to-pay threshold of $12039.1 (China per capita GDP in 2022) per QALY. If the price of ROMI is either lower than or equal to that of EPAG, ROMI could likely be considered cost-effective as a second-line treatment for Chinese adults with cITP.
本研究旨在评估与艾曲泊帕（EPAG）相比，罗米司亭（ROMI）作为中国成年慢性原发性免疫性血小板减少症（cITP）二线治疗药物的成本效益。采用嵌入决策树的马尔可夫模型，从中国医疗保健系统的角度，估算罗米司亭与艾曲泊帕的质量调整生命年（QALY）和成本。该模型以4周为周期，以血小板反应为驱动因素。QALY和成本均按每年5%进行贴现。通过匹配调整间接比较（MAIC）获得比较罗米司亭和艾曲泊帕的临床数据，利用罗米司亭的个体患者数据和已发表的关于艾曲泊帕的中国III期试验数据。成本以2022年美元报告，包括药物购置成本、监测成本、出血相关成本以及与不良事件相关的成本。进行了确定性和概率敏感性分析。成本效益分析（CEA）模型表明，使用罗米司亭治疗导致每0.004个QALY的成本平均高出4344.4美元。单向敏感性分析（OSA）表明，该模型对艾曲泊帕和罗米司亭在反应（马尔可夫阶段）中的高出血率最为敏感。概率敏感性分析（PSA）表明，在每QALY支付意愿阈值为12039.1美元（2022年中国人均国内生产总值）时，罗米司亭在0.16%的情况下可能具有成本效益。如果罗米司亭的价格低于或等于艾曲泊帕，罗米司亭作为中国成年cITP患者的二线治疗药物可能被认为具有成本效益。
