Application of real world study and human use experience in research and development of new traditional Chinese medicine drugs / 中国中药杂志

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.

Immune checkpoint inhibitors for non-small cell lung cancer: Research progress of real-world studies / 中国肿瘤临床

Several immune checkpoint inhibitors (ICIs) have been approved for use in patients with advanced non-small cell lung cancer (NSCLC) based on the results of randomized controlled trials (RCTs), bringing new hope to patients with such disease. However, the applicability of the RCT results is limited due to their strict inclusion criteria and specific clinical settings. Real-world studies (RWS) can integrate data from real-life practice with long-term clinical observations and follow-up, therefore building up the real-world evidence to complement that provided by conventional clinical trials. ICIs have been used in clinical practice in multiple countries and areas for several years. Here, we aim to provide an overview of the efficacy and safety of ICIs in patients with NSCLC included in the large expanded access program and multicenter retrospective observational studies and review the impact of different populations to provide a reference for ICIs use in China.