Some conclusions drawn from explanatory randomized controlled trial (ERCT) lack practical value in application under real-world clinical settings, and there are also some limitations on the generalization of pragmatic randomized controlled trial (PRCT) results. In some countries, real-world studies (RWSs) have been conducted to explore the effectiveness and safety of clinical interventions or postmarketing drugs, which is a new tendency in clinical researches. By reviewing some RWSs carried out in Western countries, we sum up the basic characteristics of the research design, and discuss the differences and connections between PRCT and RWS. The design ideas of RWS are different from PRCT. Researchers in RWS tend to perform long-term evaluation based on large quantities of subjects and quite large sample and focus on outcome measures which are clinically meaningful. Strict control on data collection, management and analysis are very important to RWS and PRCT. Owing to the complexity of clinical interventions and evaluation, PRCT is not completely suitable for clinical researches in traditional Chinese medicine (TCM). It is an inevitable tendency to apply RWS in clinical trials of TCM. We can explore the efficacy of TCM interventions through PRCT, and better understand the effectiveness through RWS. RWS will become a powerful approach to TCM clinical trials and postmarketing evaluation of Chinese medicines.

Key words: Real-world study, Randomized controlled trial, Chinese herbal drug, Investigational new drug, Drug evaluation