Yang Zhong-Qi, Tang Hui-Min, Tang Ya-Qin, DU Yan-Ping, Gao Rui, Hu Si-Yuan, Yuan Wei-An, Zou Chong, Ding Hong, Zhao Yan-Ling
the First Affiliated Hospital of Guangzhou University of Chinese Medicine Guangzhou 510407, China.
Xiyuan Hospital, China Academy of Chinese Medical Sciences Beijing 100091, China.
Zhongguo Zhong Yao Za Zhi. 2021 Nov;46(22):5987-5991. doi: 10.19540/j.cnki.cjcmm.20210517.501.
Real world study(RWS) refers to the process of collecting real world data related to the health of research subjects in the real world environment for pre-set clinical problems and obtaining the status of drug use and potential benefits/risks through analysis. The data are derived from the hospital information system(HIS), medical insurance system, disease registration system, adverse drug reaction monitoring system, etc. Human use experience of traditional Chinese medicine(TCM) is a new concept put forward by experts after summarizing the problems existing in clinical trials of new TCM drugs. The data come partially from the real world, and more importantly, such key elements as the formulated prescriptions of new TCM drugs, principles and methods, and clinical applications should be covered. RWS is mainly used for adverse drug reaction monitoring after marketing, benefit evaluation of listed drugs, decision-making of medical treatment and medical insurance, as well as supervision and approval of special medical devices and special drugs. It is complementary to randomized controlled clinical trials. Human use experience is suitable for the research and development of Chinese medicinal compound preparations and the expansion of functions and indications. There are no special provisions for clinical indications and target population. There exists a sequential relationship between the human use experience and clinical trials. Specifi-cally, the summarization of human use experience provides good support for the design and implementation of clinical trials, which is an important segment in the research and development of new TCM drugs. The correlation between real-world data and research results and their reliability should be ensured in RWS, and the unreality should be avoided. The key to summarizing the human use experience is to identify the clinical orientation, target population, course of treatment, usage and dosage of new TCM drugs, and it should be noted that human use experience does not only mean clinical experience. Experimental clinical trial(PCT), a type of study in the real world, has been commonly employed for the summary of human use experience. RWS and human use experience are different research designs targeting different clinical questions in the research and development of new TCM drugs, which can be flexibly selected depending on the actual situation.
真实世界研究（RWS）是指在真实世界环境中，针对预设的临床问题收集与研究对象健康相关的真实世界数据，并通过分析获得药物使用状况及潜在获益/风险的过程。这些数据来源于医院信息系统（HIS）、医疗保险系统、疾病登记系统、药品不良反应监测系统等。中药人用经验是专家在总结中药新药临床试验存在的问题后提出的新概念。数据部分来源于真实世界，更重要的是，应涵盖中药新药的组方、原则和方法以及临床应用等关键要素。RWS主要用于上市后药品不良反应监测、上市药品获益评估、医疗及医保决策，以及特殊医疗器械和特殊药品的监管审批。它是对随机对照临床试验的补充。人用经验适用于中药复方制剂的研发以及功能主治的拓展。对临床适应证和目标人群没有特殊规定。人用经验与临床试验之间存在先后顺序关系。具体而言，人用经验的总结为临床试验的设计和实施提供了良好支撑，是中药新药研发的重要环节。在RWS中应确保真实世界数据与研究结果之间的相关性及其可靠性，避免不真实性。总结人用经验的关键在于明确中药新药的临床定位、目标人群、疗程、用法用量，需要注意的是，人用经验并不单纯指临床经验。试验性临床试验（PCT）作为一种真实世界中的研究类型，已普遍用于人用经验的总结。RWS与人用经验是中药新药研发中针对不同临床问题的不同研究设计，可根据实际情况灵活选用。
