基于政策工具视角的我国罕见病领域政策文本分析

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。

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

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.