全球药物价值评价工具的循证评价

Evidence-based evaluation of global value assessment tools

目的循证评价全球所有药物价值评价工具的基本特征、主要内容及关键要素、技术等,以抗肿瘤药物价值评价为重点,为创建我国首个抗肿瘤药物价值评价工具提供借鉴与参考。方法计算机检索PubMed、EMbase、CBM、CNKI、VIP、WanFang Data和19个相关机构与学会网站,纳入全球所有药物价值评价工具,检索时间从建库至2018年12月31日。由2位评价员按照纳入排除标准独立筛选文献、提取资料并交叉核对,采用定性分析描述其发布机构、年份、国家、适用疾病类型与药物类别、结果形式等基本特征;重点比较价值评价维度、证据来源/分级、工具形成方法与过程等关键要素和技术。结果①共纳入12个英文药物价值评价工具,发布于2010~2018年,均由欧洲、北美洲制订,适用疾病类型与药物类别不完全相同;②各工具的"目标使用人群"略有差异;③"价值评价维度"、"证据来源/分级"与"形成方法与过程"属药物价值评价的关键问题。结论①现有药物价值评价工具的结构基本一致,但价值定义、评价维度、证据来源、结果形式不同;②工具形成方法与过程欠清晰,亟待研发价值导向清楚、重点明确、可操作性强的抗肿瘤药物价值评价工具,以满足以价值为导向的药物上市后再评价需求。

Objectives To evaluate the characteristics,main contents,key elements and techniques of global drug value assessment tools,especially for those developed for antineoplastic agents,and to provide reference for the establishment of the first value assessment tool for antineoplastic agents in China.Methods Databases including MEDLINE,EMbase,CBM,CNKI,VIP,WanFang Data and 19 relevant websites of institutions and societies were searched from inception to October 31st,2018 to identify all the drug value assessment tools worldwide.Two independent reviewers screened the literatures,extracted the data and cross-checked them according to the inclusion and exclusion criteria.A qualitative analysis was conducted to describe the characteristics of these drug value assessment tools,including the publishing organization,year of publication,country,applicable type of disease and drug category,result display,and etc.Key elements and techniques in terms of evaluation dimensions,sources and levels of evidence,methods and procedures to form the tool were compared.Results A total of 12 English drug value assessment tools were included,which were published in 2010 to 2018 exclusively from Europe and North America.The applicable types of diseases and drug categories are not identical.The target users and stakeholders of each tool were slightly different.Evaluation dimensions,sources and levels of evidence,methods and procedures to form the tool were vital issues in value evaluation for drugs.Conclusions The structures of existing drug value assessment tools were almost the identical.However,there is no consensus on value definitions,evaluation dimensions,sources of evidence and result display.Methods and procedures to form the tool are not well described.It is urgent to explore and develop a value-oriented,focused and feasible drug assessment tool for antineoplastic agents in order to satisfy the strategic requirements of value-based postmarketing drug reevaluation.