急性髓系白血病治疗药物经济学评价研究的系统评价

Economic evaluation of different medicines for acute myeloid leukemia:a systematic review

目的系统评价急性髓系白血病(AML)治疗的卫生经济学评价研究。方法计算机检索PubMed、EMbase、Cochrane Library、CBM、CNKI和WanFang Data数据库,以及CRD database卫生经济学专业数据库,并手工检索卫生经济学领域的相关杂志和各国HTA机构网站,全面搜集关于AML治疗药物的经济学评价研究。检索时限均从建库至2022年6月。参考CHEERS清单对文献质量进行评价。总结目前国内外已发表AML治疗卫生经济学评价的基本特征,分析评价模型结构及方法学差异,并比较药物的经济性。结果共纳入17个研究,分别从卫生体系、患者、全社会和医疗保险支付方角度出发进行成本-效果分析,评价模型比较统一,但在方法和结果报告上存在差异,质量有待加强。研究对象主要是去甲基化药、靶向药与传统化疗方案进行比较,以及不同化疗组合方案、不同药物剂量之间的比较。结论基于真实世界数据的研究主要聚焦于传统化疗方案,而马尔可夫等经济学评价模型,更多应用于近年研发的靶向药和去甲基化药物。与对照方案相比,基于阿糖胞苷的化疗方案、米哚妥林以及地西他滨更具有成本-效果。

Objective To systematically review the health economic evaluation studies of medicines for the treatment of acute myeloid leukemia(AML).Methods The PubMed,EMbase,Cochrane Library,CBM,CNKI,and WanFang Data,as well as the CRD database specifically for health economics were electronically searched from inception to June 2022,and related journals in the field of health economics and the websites of HTA institutions in various countries were manually searched.The quality of the studies was assessed using the CHEERS checklist.The basic characteristics of health economics evaluation publications were summarized,the quality of model structures and methodologies was assessed and economic evaluation results were compared among different treatments.Results A total of 17 studies were included,and cost-effectiveness analyses were conducted from the perspectives of the health system,patients,the whole society,and medical insurance payers.The economic evaluation models were relatively unified,but there were differences in methods and results reporting,and the quality needed to be improved.The research objects were mainly the comparison of hypomethylating agents,targeted medicine and traditional chemotherapy regimens,as well as the comparison of different chemotherapy combinations and different drug dosages.Conclusion Real-world studies are mainly focused on traditional chemotherapy regimens,and model-based health economic evaluations,such as Markov models,are more frequently applied to newly developed targeted drugs and demethylation drugs.Among all treatments,the chemotherapy regimens including cytarabine,midostaurin,and decitabine are found to be more cost-effective.