摘要
目的比较2018-2022年中国和美国孤儿药的上市情况,进一步了解两国孤儿药发展的趋势和差异形成原因。方法检索国家药品监督管理局、美国食品药品监督管理局获取中国和美国上市的孤儿药信息,并对中美上市孤儿药的数量、类型、药品上市许可持有人、目标疾病和创新药进行分类比较。结果2018-2022年,美国上市426种孤儿药,我国上市64种孤儿药,平均环比增长率分别为-4.81%和8.38%。中国和美国上市孤儿药数量最多的类型均为抗肿瘤药和免疫调节剂类药品,其中美国235种(55.16%),我国15种(23.44%)。在上市孤儿药的目标治疗罕见病类型中,美国最多的是罕见的肿瘤疾病197种(46.24%);我国则是罕见的神经和精神疾病22种(34.38%)。美国上市36种具有突破性治疗价值的首创孤儿药,我国上市40种罕见病治疗的创新药。结论美国的孤儿药上市数量远超我国,但略微呈现下降趋势,而我国则呈现出上升趋势;美国本土孤儿药产业集中度较高,我国孤儿药仍需依赖进口;我国的国产罕见病创新药品数量取得实质性进步,但是与美国仍存在巨大差距,特别是在先进技术创新药研发方面。因此,我国在罕见病新药的研发上还需加大力度,积极参与全球同步研发实现合作共赢。
Objective To compare the approval status of orphan drugs in China and the United States(the U.S.)from 2018 to 2022,and to further understand the trends and reasons for the differences in the development of orphan drugs between the two countries.Methods Data on orphan drug approvals in China and the U.S.were retrieved from the National Medical Products Administration of China and the U.S.Food and Drug Administration(FDA),and the number,types,Marketing Authorisation Holder(MAH),target diseases,and new drugs marketed in China and the U.S.were classified and compared.Results From 2018 to 2022,426 approvals of orphan drugs in the U.S.and 64 approvals in China.Average annual growth rates of drug approval numbers in the U.S.and China were-4.81%and 8.38%,respectively.The most common types of orphan drugs approved in both countries were antineoplastic drugs and immunomodulators,of which 235(55.16%)were in the U.S.versus 15 types(23.44%)in China.Among the types of rare diseases targeted by these orphan drugs,the U.S.had the most rare tumor diseases(197 types,46.24%),while China had the most rare neurologrical and psychiatric diseases(22 types,34.38%).The U.S.marketed 36 first-in-class orphan drugs with breakthrough therapeutic value.And China marketed 40 innovative new drugs for the treatment of rare diseases.Conclusion The number of orphan drugs marketed in the U.S.has far surpassed that in China,but has shown a slight downward trend,while an increasing trend in China.The concentration of the domestic orphan drug industry in the U.S.is high,while China's orphan drugs still rely on imports.China has made substantial progress in making domestic innovative drugs for rare diseases,but there is a huge gap between China and the U.S.,especially in terms of advanced technology and innovative drugs.Therefore,intensify efforts should be made in the research and development(R&D)of new drugs for rare diseases in China,and actively participate in global synchronized research and development to achieve win-win cooperation.
作者
蒋维鑫
龚时薇
JIANG Weixin;GONG Shiwei(School of Pharmacy,Tongji Medical College,Huazhong University of Science and Technology,Wuhan 430030,China)
出处
《医药导报》
CAS
北大核心
2024年第6期885-890,共6页
Herald of Medicine
基金
国家自然科学基金面上项目(70903025,71373089)。
关键词
孤儿药
罕见病
药品数量
药品类型
创新药
Orphan drug
Rare disease
Drug quantity
Drug type
Innovative drug
