首页|我国121种罕见病的治疗药品及其医疗保障现状分析

我国121种罕见病的治疗药品及其医疗保障现状分析

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目的:建立中国121种罕见病国内外治疗药品目录并分析这些药品在中国的医疗保障现状。方法:通过国家药品监督管理局、美国食品药品监督管理局、欧盟Orphanet孤儿药数据库和药品说明书以及相关文献确定中国121种罕见病的治疗药品目录,通过药智数据和国家医疗保障局获得在中国上市罕见病治疗药品的价格和医疗保险覆盖数据。结果:截至2022年10月,中国上市治疗109个罕见病的194种通用名共219个剂型药品,国产率72。60%。然而,仍有12种罕见病在中国没有治疗药品,国外上市的治疗55种罕见病的93个药品在中国尚未上市。205个有价格信息的治疗药品的平均单元包装价格为1 363。58元,其中有132个药品(占比64。39%)的平均单元包装价格低于50元,165个(占比80。49%)药品被国家基本医疗保险目录覆盖。平均单元包装价格低于50元的药品的医疗保险目录覆盖率为89。39%,高于200元的药品的医疗保险目录覆盖率为54。72%。结论:目前中国121种罕见病大部分有治疗药品和基本医疗保险覆盖,但罕见病用药的持续保障能力仍然十分脆弱。因此,急需依据罕见病药品的特点建立供应、利用和保险支付的分类保障体系。
Analysis of treatment drugs for 121 rare diseases in China and their medical coverage status
OBJECTIVE To identify domestic and foreign therapeutic drugs for 121 rare diseases and explore the current sta-tus of medical coverage of these drugs in China.METHODS The list of therapeutic drugs for 121 rare diseases was established through the databases of National Medical Products Administration of China,US Food and Drug Administration,Orphanet of European Commission,package inserts and the relevant literatures.Drug prices and medical insurance coverage data were retrieved through the Yaozhi database and the official website of National Healthcare Security Administration of China.RESULTS As of October 2022,China had marketed 194 drugs with a total of 219 dosage forms for treating 109 rare diseases.However,12 rare diseases lacked therapeutic drugs in China and 93 foreign therapeutic drugs for 55 rare diseases have not been marketed domestically.Bidding price information was available for 205 dosage forms of therapeutic drugs with an average unit packaging price of 1363.58 RMB.Among them,132 drugs(64.39%)had an average unit package price under 50 RMB and 165 drugs(80.49%)were covered by National Basic Medical Insurance Catalog(NBMIC).The coverage proportion of drugs in NBMIC with an average unit packaging price under 50 RMB was 89.39%.And the coverage proportion of drugs in NBMIC with an average unit packaging price of above 200 RMB was 54.72%.CONCLUSION Most therapeutic drugs of 121 rare diseases in China are currently covered by NBMIC.However,the capacity for sustained supply of drugs for rare diseases remains quite frag-ile.Therefore it is imperative to establish a categorical guarantee system for supply,utilization and insurance payment according to the characteristics of these drugs for rare diseases in China.

rare diseasestherapeutic drugsmarketing statusNational Basic Medical Insurance Cataloguecoverage proportion

罗仕俊、朱玉琳、丁玉峰、龚时薇

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华中科技大学同济医学院药学院,湖北武汉 430030

武汉市第五医院药学部,湖北武汉 430050

华中科技大学同济医学院附属同济医院药学部,湖北武汉 430030

罕见病 治疗药品 上市状态 国家基本医疗保险目录 覆盖率

国家自然科学基金国家自然科学基金

7090302571373089

2024

中国医院药学杂志
中国药学会

中国医院药学杂志

CSTPCD北大核心
影响因子:1.198
ISSN:1001-5213
年,卷(期):2024.44(10)
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