基于政策工具视角的我国罕见病领域政策文本分析  

Policy text analysis of rare disease in China based on the perspective of policy tools

作　　者：牟燕 宋奎勐[1,2] 吴敏 何有琴[1,2] 甄天民 MU Yan;SONG Kui-meng;WU Min;HE You-qin;ZHEN Tian-min(Shandong First Medical University&Shandong Academy of Medical Sciences,School of Healthcare Security&Shandong Healthcare Security Research Institute,Jinan Shandong 250062,China;Shandong Institute of Medicine and health Information,Jinan Shandong 250062,China)

机构地区：[1]山东第一医科大学(山东省医学科学院)医疗保障学院(山东省医疗保障研究院),山东济南250062 [2]山东省医药卫生科技信息研究所,山东济南250062

出　　处：《中国卫生政策研究》2021年第1期17-23,共7页Chinese Journal of Health Policy

基　　金：山东第一医科大学学术提升计划;山东省医学科学院医药卫生科技创新工程;山东省医药卫生科技发展计划项目(2019WS191)。

摘　　要：目的:总结我国罕见病领域相关政策的关键要素及不足,为我国罕见病领域后续政策的制定与完善提供参考。方法:采用内容分析法,构建“政策目标—政策工具”分析框架对我国32份国家层面的罕见病领域相关政策进行量化分析。结果:我国罕见病领域政策目标为提升罕见病药品可及性、提升罕见病诊疗水平和提高罕见病患者保障水平,应用于上述目标的政策工具比例分别为50%、44.87%和5.13%。讨论:我国罕见病领域政策目标集中在罕见病药品可及性及诊疗水平提升;相关政策工具的选择和使用存在一定失衡或缺失,环境型政策工具使用较多,需求型政策工具使用较少。建议:今后在政策设计上应加强罕见病防治与管理,促进罕见病药品的研发、上市与使用,健全罕见病保障机制,完善罕见病政策体系。Objective:To summarize the key factors and deficiencies of current rare disease policies of China,and to provide references for the formulation and consummation of follow-up policies.Methods:Content analysis method was used to build an analysis framework of“policy objectives and policy tools”for quantitative analysis of 32 current Chinese rare disease policies at national level.Results:The policy objectives were to improve the accessibility of rare disease drugs,improve the diagnosis and treatment of rare diseases.Policy tools used to them accounted for 50%,44.87%and 5.13%.Discussion:The objectives of rare disease policies focus on the access to rare disease drugs and the improvement of diagnosis and treatment of rare diseases.There is a certain imbalance or deficiency in the selection and use of policy tools.Environment-oriented policy tools are used more and demand-oriented policy tools are seldom used.Suggestions:Future policy design strengthen the prevention and management of rare diseases,promote the R&D,commercialization and use of rare disease drugs,consummate the rare disease security mechanism and further improve the rare disease policy system.

关 键 词：罕见病 政策分析 政策工具 文本分析 

分 类 号：R197[医药卫生—卫生事业管理]