国外罕见病国家政策分析及对我国的启示  

Analysis of foreign national policies for rare diseases and the inspiration for China

作　　者：牟燕[1,2] 范文杰 刘岩 MU Yan;FAN Wenjie;LIU Yan(Medical Intelligence Research Center,Shandong Institute of Medicine and Health Information,Jinan Shandong 250017,China;College of Medical Security,Shandong First Medical University&Shandong Academy of Medical Science,Jinan Shandong 250017,China)

机构地区：[1]山东省医药卫生科技信息研究所医学情报研究中心,山东济南250017 [2]山东第一医科大学/山东省医学科学院医疗保障学院,山东济南250017

出　　处：《卫生软科学》2023年第2期17-22,共6页Soft Science of Health

基　　金：山东省人文社会科学课题(2021-YYGL-28);山东第一医科大学(山东省医学科学院)青年科学基金培育资助计划(202202-001);山东省医药卫生科技发展计划项目(202013041353);山东省药物政策研究重点课题(YZY202202)。

摘　　要：系统梳理了英国、德国、法国、澳大利亚和印度的罕见病国家战略及行动计划的基本框架和内容,结合典型国家的罕见病国家政策和我国罕见病政策现状及存在问题,提出我国需进一步明确罕见病的定义、保障罕见病患者的基本权利、加快创新研究提升罕见病诊疗水平和运用PDCA循环方法对罕见病政策进行优化,为完善我国罕见病相关政策提供借鉴。The basic framework and content of national strategies and action plans for rare diseases in the United Kingdom, Germany, France, Australia and India are systematically reviewed, and the status quo and existing problems of national policies for rare disease in typical countries are combined with those of China. It suggests that we need to further clarify the definition of rare diseases, guarantee the basic rights of patients with rare diseases, accelerate innovative research to improve the level of rare disease diagnosis and treatment, and apply the PDCA cycle approach to optimize rare disease policies, so as to provide reference for completing the relevant policies for rare disease.

关 键 词：罕见病 国家政策 国家行动计划 国家战略 启示 

分 类 号：R199[医药卫生—卫生事业管理]