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Current Situation and Prospect of Pharmacoeconomic Evaluation of Hemophilia in China
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摘要: 2018年血友病被纳入中国《第一批罕见病目录》，药物可及性与可负担性是血友病患者面临的最大困难，因此，全面评估血友病药物的成本与疗效是促进药物合理使用的重要手段。本研究在梳理血友病药品审评审批与医保准入现状的基础上，系统检索国内外公开发表的血友病药物经济学评价研究，全面分析国内血友病药物经济学研究的文献质量、评价内容及面临的挑战，最后从开发血友病专用模型、多途径获取患者健康效用值、制订罕见病特有意愿支付阈值等三个方面提出今后研究展望，以期为国内开展血友病药物经济学评价研究提供参考与借鉴。Abstract: In 2018, hemophilia, one of the first ones, entered into the catalogue of Rare Disease in China. However, drug accessibility and affordability have been the challenges for hemophilia patients. Therefore, a comprehensive evaluation of the cost and efficacy of hemophilia drugs is essential to push forward the appropriate application of hemophilia drugs. In the article, we have teased out the existing process of the review and approval of hemophilia drugs; have examined the procedures for the drug entry into the medical insurance policy; have systematically searched the pharmacoeconomic evaluation studies of hemophilia in published literature in both China and overseas; and have comprehensively analyzed the quality, the content of evaluation, and challenges of pharmacoeconomic studies of hemophilia in China. At last, we discuss the prospect for the future based on the following three aspects: developing a special model of hemophilia, obtaining the health utility from various sources, and establishing the threshold of willingness to pay for rare diseases, hoping to provide reference and examples to the research into pharmacoeconomic evaluation of hemophilia in China.
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Key words:
- hemophilia /
- pharmacoeconomics /
- evaluation
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表 1 中国已上市的血友病替代治疗药物
Table 1. Hemophilia drugs that have been listed in China
患者类型 药物类型 血友病A 血浆源性人凝血因子Ⅷ 基因重组人凝血因子Ⅷ 血友病B 血浆源性人凝血因子Ⅸ 基因重组人凝血因子Ⅸ 凝血酶原复合物 血友病伴抑制物 基因重组活化人凝血因子Ⅶ 艾美赛珠单抗 凝血酶原复合物* *因各种原因无法使用上述两种药物情况下，再用凝血酶原复合物 
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