2011—2023年我国各省级行政区罕见病及其药物相关政策差异性分析  

Differential analysis of rare disease and related drug policies across provincial-level administrative regions in China from 2011 to 2023

作　　者：王雅群 周娜 朱炜楷 罗蓟[5] 袁妮[1,2] WANG Ya-qun;ZHOU Na;ZHU Wei-kai;LUO Ji;YUAN Ni(School of Public Health,Dalian Medical University,Dalian LIAONING 116044,China;Innovative Drug Policy and Medical Insurance Research Center,Dalian Medical University,Dalian LIAONING 116044,China;Department of Health Policy and Management,School of Public Health,Peking University,BEIJING 100191,China;The First Affiliated Hospital of Dalian Medical University,Dalian LIAONING 116011,China;The First Hospital of China Medical University,Shenyang LIAONING 110001,China)

机构地区：[1]大连医科大学公共卫生学院,辽宁大连116044 [2]大连医科大学创新药品政策与医疗保障研究中心,辽宁大连116044 [3]北京大学公共卫生学院卫生政策与管理学系,北京100191 [4]大连医科大学附属第一医院,辽宁大连116011 [5]中国医科大学附属第一医院,辽宁沈阳110001

出　　处：《中国新药与临床杂志》2025年第3期228-235,共8页Chinese Journal of New Drugs and Clinical Remedies

摘　　要：目的梳理我国各省级行政区出台的罕见病及其药物相关政策,为政策的完善提供参考。方法收集地方政府等颁布的罕见病及其药物相关政策文本,总计432条,并采用文本分析、描述性统计和相关性分析等方法剖析当前政策的现状、不同地区之间的政策差异及造成差异的影响因素。结果2011—2023年期间,各省级行政区发布的罕见病及其药物相关政策数量总体上呈逐年增长的趋势,2016年之后的发文数量占发文总数的93.75%。其中,用药保障和研发注册是数量最多的政策类型,分别有130条(30%)和122条(28%);东部、中部、西部和东北地区平均每省政策发文数量分别为19.5、14.3、9和14.3条,东部地区对罕见病及其药物相关政策的关注度最高;地区分布、居民人均可支配收入、居民人均消费支出及卫生总费用等因素与政策数量之间显著相关。结论各省级行政区对罕见病问题的关注程度在不断提高,但仍面临一些困难与挑战。建议出台更多的地方罕见病专项政策,加强地区间的合作与交流等,从而促进全国范围内罕见病政策的均衡发展。AIM To review the rare disease and related drug policies enacted by provincial-level administrative regions in China,providing references for policy improvement.METHODS A total of 432 policy texts related to rare diseases and their drugs were collected from local governments.Text analysis,descriptive statistics,and correlation analysis were used to analyze the current status of policies,regional differences,and factors influencing these differences.RESULTS Between 2011 and 2023,the number of policies related to rare diseases and their medications issued by provincial-level administrative regions showed an overall increasing trend year by year.The number of publications since 2016 accounted for 93.75%of the total.Among them,the most prevalent policy types were drug coverage and research registration,with 130(30%)and 122(28%)policies,respectively.The average number of policies issued per province in the eastern,central,western,and northeastern regions was 19.5,14.3,9 and 14.3,respectively,with the highest focus on rare diseases and their drug-related policies observed in the eastern region.Significant correlations were found between policy quantity and factors such as regional distribution,per capita disposable income,per capita consumption expenditure,and total healthcare expenditure.CONCLUSION The degree of attention paid by various provincial-level administrative regions to rare disease issues is constantly increasing,but they still face some difficulties and challenges.It is recommended to enact more specialized policies for rare diseases at the local level and strengthen cooperation and communication among regions to promote balanced development of rare disease policies nationwide.

关 键 词：罕见病 政策 地区差异 影响因素 

分 类 号：R95[医药卫生—药学]