李柯欣, 赵琨, 郑佳音, 杨玉琪, 李林康. 《第二批罕见病目录》收录病种的药物研发和落地现状[J]. 罕见病研究, 2023, 2(4): 596-601. DOI: 10.12376/j.issn.2097-0501.2023.04.015
引用本文: 李柯欣, 赵琨, 郑佳音, 杨玉琪, 李林康. 《第二批罕见病目录》收录病种的药物研发和落地现状[J]. 罕见病研究, 2023, 2(4): 596-601. DOI: 10.12376/j.issn.2097-0501.2023.04.015
LI Kexin, ZHAO Kun, ZHENG Jiayin, YANG Yuqi, LI Linkang. Current Status of Drug Development and Implementation for Diseases Included in the Second Catalog of Rare Diseases[J]. Journal of Rare Diseases, 2023, 2(4): 596-601. DOI: 10.12376/j.issn.2097-0501.2023.04.015
Citation: LI Kexin, ZHAO Kun, ZHENG Jiayin, YANG Yuqi, LI Linkang. Current Status of Drug Development and Implementation for Diseases Included in the Second Catalog of Rare Diseases[J]. Journal of Rare Diseases, 2023, 2(4): 596-601. DOI: 10.12376/j.issn.2097-0501.2023.04.015

《第二批罕见病目录》收录病种的药物研发和落地现状

Current Status of Drug Development and Implementation for Diseases Included in the Second Catalog of Rare Diseases

  • 摘要: 中华人民共和国国家卫生健康委员会等六部门共同发布了《第二批罕见病目录》,增扩了中国的罕见病定义。目录的扩容意味着国家层面对罕见病的更大关注,也有望能够推动相关罕见病药物的加速上市,让更多的罕见病患者获益。新的罕见病目录发布,就需要快速的梳理治疗药物的可及情况,摸清基数,以供后续相关措施的推进。本文检索了国家药品监督管理局、国家药品监督管理局药品审评中心以及国外药品监管机构等官方医药信息网站和医药信息数据库,梳理和汇总《第二批罕见病目录》收录的疾病归属学科、对应的药物全球研发和落地情况,以及在中国的医保准入情况,阐述了国内外孤儿药可及性的差异。在中国罕见病防治与保障事业来到了新时点的情形下,对未来相关政策出台,以及促进研发和保障等重点工作做出规划和展望。

     

    Abstract: The National Health Commission of the People′s Republic of China and six other departments jointly released the Second Catalog of Rare Diseases, which expanded the definition of rare diseases in China. The expansion of the catalog means greater attention to rare diseases at the national level, and is also expected to accelerate the launch of relevant rare disease drugs and benefit more rare disease patients. When the new rare disease catalog released, it is necessary to quickly sort out the availability of drugs and find out the base number for the advancement of subsequent relevant measures. This paper searched official medical information websites and medical databases such as the National Medical Products Administration (NMPA), the Center for Drug Evaluation (CDE), and foreign drug regulatory agencies, and sorted out and summarized the subject categories of diseases included in the Second Catalog of Rare Diseases, the corresponding global research and development and implementation of drugs, as well as the medical insurance access situation in China, elaborates on the differences in the accessibility of orphan drugs at home and abroad. As China′s prevention, treatment and protection of rare diseases has reached a new point, plans and prospects are made for the introduction of relevant policies in the future, as well as the promotion of research and development and protection and other key tasks.

     

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