罕见病药品保障模式的国际经验与启示——以酶替代疗法药品为例

International Experience and Enlightenment of Drug Security Model for Rare Diseases——Taking Enzyme Replacement Therapy Drugs as an Example

目的:以酶替代疗法药品为例,分析我国罕见病药品保障现状,在借鉴国外经验的基础上,提出完善我国罕见病药品保障政策的建议。方法:通过政策梳理及文献分析,了解我国酶替代疗法药品保障现状,总结澳大利亚、俄罗斯、新西兰对酶替代疗法药品的基本保障模式。结果:澳大利亚、俄罗斯、新西兰的典型保障模式对罕见病患者酶替代疗法用药均有不同程度的补助,具有国家财政参与、药品衔接保障和多部门协同的共同特点。结论:我国可借鉴国际经验,通过设立国家专项基金、建立疾病与药品间政策衔接机制、加强信息网络建设等举措,完善罕见病药品保障体系。

Objective Taking enzyme replacement therapy drugs as an example,to analyze the current situation of drug security for rare disease in China,and to propose suggestions for improving drug security policy for rare disease in China based on international experience.Methods Policy review and literature analysis was used to understand the current situation of drug security for enzyme replacement therapy in China,and the basic security models for enzyme replacement therapy drugs in Australia,Russia,and New Zealand were summarized.Results The typical security models of Australia,Russia,and New Zealand provided varying degrees of subsidies for enzyme replacement therapy drugs for patients with rare disease,with common characteristics of national financial participation,drug connection guarantee,and multi departmental collaboration.Conclusion China can learn from international experience and improve drug security system for rare diseases by establishing a national special fund,building a policy connection mechanism between diseases and drugs,and strengthening information network construction.