罕见病医疗保障现状及问题研究——以某医院收治的3个罕见病种为例  

Research on current situation and problems of medical security for rare diseases——Take three rare diseases in a hospital as an example

作　　者：程俊[1] 高华斌[2] 何梅 CHENG Jun;GAO Hua-bin;HE Mei(School of Public Administration,Nanjing Agricultural University,Nanjing Jiangsu 210095,China;The First Affiliated Hospital of Kunming Medical University,Kunming Yunnan 650500,China;School of Humanities and Management,Kunming Medical University,Kunming Yunnan 650500,China)

机构地区：[1]南京农业大学公共管理学院,江苏南京210095 [2]昆明医科大学第一附属医院,云南昆明650500 [3]昆明医科大学人文与管理学院,云南昆明650500

出　　处：《卫生软科学》2021年第9期21-26,共6页Soft Science of Health

基　　金：昆明医科大学“十三五”重点扶持学科-社会保障建设项目(2017HP013)。

摘　　要：梳理了国内外学者对罕见病医疗保障制度的态度和评价,并以昆明医科大学第一附属医院5年内收治的罕见病中的3个病种为例分析我国罕见病医疗保障的现状,发现我国罕见病医疗保障工作仍然存在如下问题:基本定义缺失和诊断标准不明晰,病例登记系统不完善,基本的罕见病用药(罕用药)短缺、罕见病的诊疗水平和可治性低、基本医疗保障力度不够、商业保险中重疾险涉及罕见病的条款很少,组织机构不健全等。国家应制定罕见病相关法律法规,加快推进流行病学调查,确定罕见病治疗药物研发激励机制,提高罕见病诊疗水平,完善罕见病预防体系,加强国际合作,以提高我国罕见病的医疗保障水平。This study mainly sorts out the attitudes and evaluations of domestic and foreign scholars on rare disease medical security system,and analyzes current situation of rare disease medical security by taking 3 rare diseases treated in a hospital within 5 years as examples.It is found that there are still some problems of rare disease medical security work in our country,such as lack of basic definitions and unclear diagnostic criteria,imperfect case registration system,shortage of basic orphan drugs,low level and curability of diagnosis and treatment of rare diseases,insufficient security efforts of basic medical insurance,less items involved rare diseases in commercial insurance,unsound of organization,etc.In order to promote the development of rare disease medical security,it is recommended that government should formulate relevant laws and regulations for rare diseases.Accelerate the advancement of epidemiological investigations.Determine incentive mechanism of orphan drug research and development.Improve the diagnosis and treatment of rare diseases.Improve rare disease prevention system.Strengthen international cooperation to improve the medical security level of orphan drugs in our country.

关 键 词：罕见病 罕用药 医疗保障 药物治疗 现状 

分 类 号：R197.1[医药卫生—卫生事业管理]