基于疗效指标的药物临床试验现场核查关注点分析  

Analysis on Concerns of Efficacy Indicators in Drug Clinical Trial On-site Inspection

作　　者：樊红延 胡敬峰 赵婷婷 张建永 冯巧巧 Fan Hongyan;Hu Jingfeng;Zhao Tingting;Zhang Jianyong;Feng Qiaoqiao(Shandong Center for Food and Drug Evaluation&Inspection,Jinan 250014,China)

机构地区：[1]山东省食品药品审评查验中心,济南250014

出　　处：《中国药事》2023年第8期864-869,共6页Chinese Pharmaceutical Affairs

摘　　要：目的:分析药物临床试验现场核查过程中疗效指标相关问题的关注点,为药物临床试验现场核查工作提供参考。方法:参考国内外药物临床试验实施和检查相关指导性文件,对疗效指标相关核查要点进行总结;并结合近年来现场核查经典案例,梳理核查过程中不同试验设计的药物临床试验常用的疗效评价指标及对应的常见问题,分析现场核查的关注点。结果:通过案例分析,明确了疾病临床终点或相关重要临床事件类疗效指标、量表类疗效指标、实验室检测结果类疗效指标、影像学结果类疗效指标4类常用疗效评价指标的现场核查关注点。结论:不同类型的疗效指标具有不同的评估要求、评估标准和评估流程,现场核查过程中应有针对性地考虑适当的关注点,才能使核查工作更加高质高效地开展。Objective:To analyze the focus points of efficacy indicators related issues during the on-site inspections of drug clinical trials,and provide references for such relevant inspections.Methods:On the basis of domestic and international guidance documents related to the implementation and inspections of drug clinical trials,this article summarizes the key points of inspections related to efficacy indicators.Combined with the classical cases of on-site inspections in recent years,the efficacy evaluation indexes and corresponding problems in drug clinical trials with different trial designs are sorted out and the focus of on-site inspections are analyzed.Results:Through the case analysis,this article further clarified the focus of on-site inspections related to four common types of evaluation indexes,including clinical endpoint of disease or related important clinical events efficacy indicators,scale efficacy indicators,laboratory test results efficacy indicators,and imaging results efficacy indicators.Conclusion:Different types of efficacy indicators have different evaluation requirements,standards and processes.In the process of on-site inspections,the specific focus should be taken into account properly in order to make the work more high-quality and efficient.

关 键 词：药物临床试验 疗效指标 现场核查 疗效评估 

分 类 号：R95[医药卫生—药学]