我国矮身材儿童药物临床试验的开展状况  

Development of drug clinical trials in short stature children in China

作　　者：张冠东[1] 杨钰 赵瑞玲[1] ZHANG Guan-dong;YANG Yu;ZHAO Rui-ling(Office of Drug Clinical Trial Institution,Children's Hospital of Shanxi,Taiyuan 030013,Shanxi Province,China)

机构地区：[1]山西省儿童医院药物临床试验机构办公室,山西太原030013

出　　处：《中国临床药理学杂志》2024年第16期2405-2408,共4页The Chinese Journal of Clinical Pharmacology

摘　　要：目的 分析2013—2023年国内矮身材儿童药物临床试验的登记情况及生长激素类药品在中国的上市情况。方法 检索国家药品监督管理局“药物临床试验登记与信息公示平台”和“药品查询”数据库,获得矮身材儿童药物临床试验注册信息及生长激素类药品的上市信息,进行分类统计。结果 平台共登记了31项包含矮身材儿童的药物临床试验,项目所处试验分期以Ⅱ、Ⅲ期为主,合计21项(67.74%,21项/31项),组长单位呈集中趋势,与主要研究者的学术水平、临床试验经验、专业权威等密切相关。基于矮身材治疗的疗程需要,注册项目的临床试验疗程≥52周的项目占比达83.87%(26项/31项)。国内外制药企业的研发趋势为长效生长激素(64.52%,20项/31项),申办者、研究者对于临床试验的保险意识及投保积极性很高,重视临床试验的风险控制,特别是受试者的保障。目前有7家国内外制药企业的生长激素产品在中国获准上市,是迄今已上市的生物技术药物中品种最丰富的一类。结论 期望未来有更便捷给药方式、更优释放特性的长效生长激素产品问世,降低治疗成本,使矮身材儿童获得理想的健康身高。Objective To analyze the registration status of domestic drug clinical trials for short stature children and the marketing status of growth hormone drugs in China during 2013-2023. Methods The website of National Medical Products Administration was searched to abtain the date and statistical analysis was conducted. Results A total of 31 drug clinical trials involving short stature children were registered in the platform, and the trial stages were mainly on phase Ⅱ and Ⅲ, with a total of 21(67.74%, 21/31). The group leader was relatively focused on specialized hospital, which was closely related to the academic level, clinical trial experience and professional authority of the main investigators. Based on the duration of treatment for short stature, 83.87%(26/31) of registered projects had clinical trials lasting for more than 52 weeks. The research trend was long-acting growth hormone(64.52%, 20/31).Sponsors and researchers had high insurance awareness and enthusiasm for clinical trials, especially the protection of subjects.At present, growth hormone products from seven domestic and foreign enterprises had been approved for marketing in China.The growth hormone products were the most abundant category of biotechnology drugs that had been marketed so far. Conclusion It is expected that there will be more convenient ways of administration and better release characteristics of long-acting growth hormone products in the future,which will reduce treatment costs and enable short stature children to obtain an ideal healthy height.

关 键 词：生长障碍 生长缓慢 临床试验 上市药品 

分 类 号：R97[医药卫生—药品]