基于人用经验的中药新药安全性评价  

Safety evaluation of new drugs of traditional Chinese medicine based on human use experience

作　　者：杨忠奇[1,2] 唐雅琴 汤慧敏[1,2] 凌燕 杜彦萍 YANG Zhong-qi;TANG Ya-qin;TANG Hui-min;LING Yan;DU Yan-ping(the First Afiliated Hospital of Guangzhou University of Chinese Medicine,Guangzhou 510405,China;Guangdong Clinical Research Academy of Chinese Medicine,Guangzhou 510405,China)

机构地区：[1]广州中医药大学第一附属医院,广东广州510405 [2]广东省中医临床研究院,广东广州510405

出　　处：《中国中药杂志》2025年第3期812-816,共5页China Journal of Chinese Materia Medica

基　　金：广东省中药监管科学研究基地项目(2020JDB07);广东省中药人用经验临床评价技术重点实验室项目(2022ZDB06);广东省药品监督管理局科研项目(粤药监2023950);2024年广州市科技计划项目(2024B03J1298)。

摘　　要：中药新药有效物质、代谢途径和作用靶点均不清晰,非临床安全性评价往往无法实现毒性靶器官和靶组织准确定位。在中药人用经验中蕴含着重要的安全性信息,但以往人用经验临床安全性数据很少且没有得到有效应用。通过开展规范的人用经验前瞻性研究收集临床安全性数据,选择合适的理化指标(包括血/尿/大便常规、肝脏/肾脏生化指标、心血管安全性评价等),科学设计检测时点和观察的样本量。重视对患者不良事件/反应的症状与体征观察,关注老人、儿童、孕妇等特殊人群安全性信息,基于中医理论与中医诊疗模式对某些中药特有的不良事件进行观察、判断与处理;全面收集中药新药人用经验临床安全性信息,弥补中药新药毒理实验结果外推至人类可靠性较差的不足。充分发挥中药新药“源于临床”的独特优势,将毒理研究结果与人用经验安全性评价结论相互参照,开展基于人用经验的获益-风险评估,构建符合中医药特点的“宏观微观结合全景式”中药新药安全性评价技术体系,以提高中药新药研发的成功率。Because of the unclear active substances,metabolic pathways,and targets of new drugs of traditional Chinese medicine(TCM),non-clinical safety evaluation often fails to accurately locate the target organs and tissue exposed to medicinal toxicity.The human use experience(HUE)contains important safety information of TCM,while the clinical safety data in the past HUE are few and have not been effectively applied.Standardized prospective HUE studies should be carried out to collect the clinical safety data,in which appropriate physical and chemical indicators(including blood,urine,and stool routine),liver biochemical indicators,kidney biochemical indicators,and cardiovascular biochemical indicators should be selected for safety evaluation,and the detection time point and sample size should be rationally designed.Importance should be attached to the observation of symptoms and signs of adverse events/reactions in patients as well as the safety information of special groups such as the elderly,children,and pregnant women.The adverse events of TCM should be observed,judged,and treated according to the theory and the diagnosis and treatment mode of TCM.The clinical safety information about the HUE should be comprehensively collected for new drugs of TCM to make up for the lack of extrapolation of toxicological test results to humans.The unique advantages of clinical origin of new drugs of TCM should be given full play for cross-reference of the results of toxicological research and the conclusions of HUE safety evaluation.In addition,benefit-risk assessment should be conducted based on HUE,and a panoramic safety evaluation system characterized by macro and micro combination and in line with the characteristics of TCM should be established to improve the success rate in the research and development of new drugs of TCM.

关 键 词：中药新药 人用经验 安全性评价 

分 类 号：R285[医药卫生—中药学]