效率边界分析对我国药物经济学评价方法的启示

Enlightenment of Efficiency Frontier Approach on the Methods of Pharmacoeconomic Evaluation in China

目的:为我国药物经济学研究提供借鉴。方法:梳理介绍效率边界分析(EFA)操作过程中的关键步骤,结合案例演示,比较EFA与成本-效用分析(CUA)、成本-效果分析(CEA)的异同点,并结合我国实际提出EFA对我国药物经济学评价和相关决策的启示。结果与结论:EFA的研究框架大致可以分为成本测算、效益界定、模型建立、增量分析和敏感性分析等5个部分。效益指标是EFA中健康产出的测量指标,包括临床指标和综合指标两种类型,其中综合指标的应用更为广泛。构建效率边界时,一般以干预措施的效益为纵坐标、干预措施的成本为横坐标,以特定疾病领域内各备选干预措施的成本-效益坐标点构成成本-效益平面边界,研究者通过判断各备选干预措施与效率边界的相对位置来进行经济性评价。意愿支付阈值在数值上等于效率边界外延射线斜率的倒数。总体来讲,EFA与CUA、CEA相似,都需要明确成本和健康产出、模型分析、数据来源和不确定性分析等内容;但EFA在干预措施与对照选择、评价结果的表现形式、意愿支付阈值等方面与CUA、CEA不同。建议我国支付方可以考虑利用EFA推算特定疾病领域的意愿支付阈值以保证部分短缺药品的可及性;建立特定疾病医疗保险数据库,在规范数据质量的同时形成一定的评判标准;联用多种药物经济学评价方法以丰富申报资料,既保证干预方案的临床价值又保证其经济性。

OBJECTIVE:To provide reference for pharmacoeconomic study in China.METHODS:The key steps in the operation process of efficiency frontier approach(EFA) were analyzed;the similarities and differences of EFA with cost-utility analysis(CUA)and cost-effectiveness analysis(CEA)were compared on the basis of case demonstration;the enlightenment of EFA to pharmacoeconomic evaluation and related decision-making in China were puts forward combined with the practice of China.RESULTS & CONCLUSIONS:The research framework of EFA could be roughly divided into 5 parts:cost calculation,benefit definition,model establishment,incremental analysis and sensitivity analysis.Benefit indicators were the measurement indicators of health output in EFA,including clinical indicators and comprehensive indicators,among which comprehensive indicators were more widely used.When constructing the efficiency frontier,the benefit of the intervention measures was generally taken as the ordinate and the cost of the intervention measures as the abscissa.The cost-benefit coordinate points of each alternative intervention measure in a specific disease field constituted the cost-benefit plane frontier.Researchers conducted economic evaluation by judging the relative position between each alternative intervention measure and the efficiency frontier.The willingness to pay threshold was numerically equal to the reciprocal of the ray slope outside the efficiency frontier.Generally speaking,EFA was similar to CUA and CEA.All of them needed to clarify the cost and health output,model analysis,data source and uncertainty analysis;however,EFA was different from CUA and CEA in intervention measures and control selection,expression form of evaluation results,willingness to pay threshold,etc.It is suggested that the payers in China can consider using EFA to calculate the willingness to pay threshold of specific disease areas to ensure the accessibility of some drugs in short supply;establish a health insurance database of specific diseases to standardize the data quality and form a clear evaluation standard at the same time,or combine a variety of pharmacoeconomic evaluation methods to enrich the application materials so as to ensure both clinical value and economy of the intervention measure.