我国儿科药物临床试验开展现状分析  

Analysis on the current situation of pediatric drug clinical trials in China

作　　者：樊文香 赖彤彤 王瑞 李春梅[1] 倪韶青[1] Fan Wenxiang;Lai Tongtong;Wang Rui;Li Chunmei;Ni Shaoqing(Clinical Trial Institute Management Office,Children's Hospital,Zhejiang University School of Medicine,National Clinical Research Center for Child Health,Hangzhou 310052,China;College of Pharmacy,Hangzhou Medical College,Hangzhou 310053,China)

机构地区：[1]浙江大学医学院附属儿童医院临床试验机构管理办公室、国家儿童健康与疾病临床医学研究中心,杭州310052 [2]杭州医学院药学院,杭州310053

出　　处：《中华医院管理杂志》2023年第12期945-951,共7页Chinese Journal of Hospital Administration

基　　金：浙江省基础公益研究计划项目(LGF19H310004)。

摘　　要：目的了解我国儿科药物临床试验的开展现状及面临的问题,为促进我国儿科药物临床试验健康开展提供参考。方法以"儿科""儿童""年度报告""儿童用药研发""政策"等为关键词,在药物临床试验机构备案管理信息平台及政府相关部门网站,检索截至2023年10月我国儿科药物研发相关政策法规、儿科药物临床试验机构及儿科药物临床试验专业备案情况、儿科药物临床试验项目注册情况等信息,并对收集到的信息进行描述性分析。结果我国已发布9项儿科药物研发相关政策法规,支持开发符合儿童生理特征的儿童用药品新品种、剂型和规格,并对儿童用药品予以优先审评审批;已发布477个药品技术指导原则,但其中仅有14个是专门针对儿科人群用药的。截至2023年3月20日,共有272家备案儿科药物临床试验专业的药物临床试验机构,占备案总机构数的20.72%;备案机构数量排名前5的省份为广东省(34家)、河南省(21家)、浙江省(20家)、北京市(20家)和江苏省(18家);共对26类儿科药物临床试验专业进行了备案,备案数量较多的为小儿呼吸(143家)、小儿血液病(72家)、儿科-其他(71家)、小儿内分泌(68家)和小儿神经病学专业(64家)。2020—2022年,儿科药物临床试验注册项目在当年登记新药临床试验中的占比分别为8.8%(129/1473)、8.3%(168/2033)和8.3%(164/1974),而仅在儿童人群中开展的临床试验占比分别为2.2%(33/1473)、3.0%(61/2033)和3.2%(64/1974)。结论我国儿科药物研发相关政策法规仍不够完善;儿科药物临床试验机构和儿科专业备案数量均少于成人且存在分布不均衡问题;儿科药物临床试验注册项目尤其是仅在儿童人群中开展的临床试验项目占比较少。建议进一步完善儿科人群药物研发相关政策体系,优化儿科药物临床试验机构及儿科药物临床试验专业的布局。Objective To understand the current situation and problems of pediatric drug clinical trials in China,and provide reference for the healthy development of pediatric drug clinical trials.Methods Such keywords as"pediatrics""children""annual reports""children's drug research and development""policies"were used,to search for information on China's pediatric drug research and development policies and regulations,pediatric drug clinical trial institutions and pediatric drug clinical trial professional registration status,as well as pediatric drug clinical trial project registration status as of October 2023 on the drug clinical trial institution registration management information platforms and relevant government department websites.Then descriptive analysis was made on the collected information.Results China has released 9 policies and regulations on pediatric drug research and development,supporting the development of new varieties,dosage forms,and specifications of pediatric drugs that meet the physiological characteristics of children,and giving priority review and approval to pediatric drugs.477 drug technology guiding principles have been released,but only 14 of them were specifically designed for pediatric populations.As of March 20,2023,there were a total of 272 registered pediatric drug clinical trial institutions,accounting for 20.72%of the total number of registered institutions.The top 5 provinces for their number of registered institutions were Guangdong province(34),Henan province(21),Zhejiang province(20),Beijing(20),and Jiangsu province(18);A total of 26 clinical trial specialties for pediatric drugs have been registered,with the largest number of registrations being pediatric respiratory(143),pediatric hematology(72),pediatrics other(71),pediatric endocrinology(68),and pediatric neurology(64).From 2020 to 2022,the proportion of pediatric drug clinical trial registration projects in newly registered drug clinical trials was 8.8%(129/1473),8.3%(168/2033),and 8.3%(164/1974),respectively,while clinical t

关 键 词：儿科 药物临床试验 药物临床试验机构备案 新药临床试验注册 现状分析 

分 类 号：R95[医药卫生—药学]