抗肺癌药品临床综合评价指标体系的构建及实证研究

Construction of clinical comprehensive evaluation index system of anti-lung cancer drugs and empirical study

目的构建抗肺癌药品临床综合评价指标体系,为药品遴选和临床合理用药提供依据。方法遵循《药品临床综合评价管理指南(2021年版试行)》相关规定,通过文献分析法初步拟定评价指标体系框架及指标预选池,基于德尔菲法对评价指标进行筛选和修改,运用专家直接打分法及相关计算得到各个指标的权重,从而制定抗肺癌药品临床综合评价指标体系;参考《中国医疗机构药品评价与遴选快速指南》制定抗肺癌药品临床综合评价指标评分细则。使用所建临床综合评价指标体系和评分细则对抗肺癌药品帕博利珠单抗开展实证评价。结果所构建的抗肺癌药品临床综合评价指标体系包括6个一级指标、15个二级指标,其中一级指标的权重分别为安全性18、有效性38、经济性15、创新性9、适宜性10、可及性10。初步构建了抗肺癌药品临床综合评价指标评分细则。实证评价结果显示,帕博利珠单抗联合化疗方案的综合评分(61分)高于单纯化疗方案(56分),前者的有效性、创新性、适宜性均优于后者,而后者的安全性、经济性均优于前者。结论所构建的抗肺癌药品临床综合评价指标体系具有可推广落地性,可为临床合理用药提供参考依据。

OBJECTIVE To construct a comprehensive evaluation index system for anti-lung cancer drugs,and to provide a basis for drug selection and rational clinical use.METHODS Based on the Guidelines for the Management of Comprehensive Clinical Evaluation of Drugs(2021 edition for trial use),a preliminary evaluation index system framework and an index preselection pool were initially formulated through literature analysis.The evaluation indices were then selected and modified by using the Delphi method.The weights of each index were determined through expert scoring and relevant calculations,thereby establishing a comprehensive evaluation index system for anti-lung cancer drugs.Referred to Quick Guideline for Drug Evaluation and Selection in Chinese Medical Institutions,scoring detailed rules for clinical comprehensive evaluation indexes of anti-lung cancer drugs were formulated.The constructed comprehensive evaluation index system and scoring detailed rules were applied to conduct empirical evaluation of the anti-lung cancer drug pembrolizumab.RESULTS The constructed comprehensive evaluation index system for anti-lung cancer drugs included 6 primary indicators and 15 secondary indicators.The weights of the primary indicators were as follows:safety(18),efficacy(38),economy(15),innovation(9),suitability(10),and accessibility(10).The scoring detailed rules for the comprehensive evaluation index system for anti-lung cancer drugs had been preliminarily established.The results of empirical evaluation showed that the comprehensive evaluation score(61 points)of pembrolizumab combined with chemotherapy regimen was higher than that of the monotherapy chemotherapy regimen(56 points).The former demonstrated superior efficacy,innovation and suitability compared to the latter,while the latter exhibited better safety and economy.CONCLUSIONS The constructed comprehensive evaluation index system for anti-lung cancer drugs has the potential for generalizability and practical application,providing a reference basis for rational clinical drug use.