Pragmatic Clinical Trials for Real-World Evidence: Concept and Implementation

The importance of real-world evidence (RWE) has been highlighted in recent years, and the limitations of the classical randomized controlled trials, also known as explanatory clinical trials (ECTs), have been emphasized. Post-marketing observational studies have several problems, such as biases and incomparability between patient groups, and RWE can only be obtained after a certain period. Therefore, pragmatic clinical trials (PCTs) have garnered attention as an alternative to obtaining scientifically robust RWE in a relatively short time. PCTs are clinical trials that have a pragmatic concept, i.e., the opposite of ECTs and are intended to help decision makers by evaluating the effectiveness of interventions in routine clinical practice. The characteristics of PCTs are the inclusion of various patients in clinical practice, recruitment of patients in heterogeneous settings, and comparison with actual clinical treatments rather than a placebo. Thus, the results of PCTs are likely to be generalized and can have a direct impact on clinical and policy decision-making. This study aimed to describe the characteristics and definitions of PCTs compared with those of ECTs and to highlight the important considerations in the planning process of PCTs. To perform PCTs for the purpose of obtaining RWE, the contents covered in this study will be helpful.