Methods for Real-World Evidence on Therapeutics

While randomized clinical trials are crucial for generating evidence about the efficacy and safety of therapeutic agents (i.e., drugs), they have limitations. Their limitations include infeasibility in certain settings, limited generalizability, and limited power to identify rare adverse effects. Real-world evidence (RWE) is evidence derived from the review and/or analysis of real-world data, which can be defined as data derived from the provision of healthcare outside of clinical trials. There is significant overlap between RWE and pharmacoepidemiology, which is the study of the health effects of drugs and other medical products in populations, and the application of this knowledge to improve health. The purpose of this course is to explore and integrate concepts essential to planning, conducting, and interpreting pharmacoepidemiologic studies to produce RWE. Methods to produce RWE are advancing rapidly. This course will provide students with: 1) a broad-based appreciation of current approaches to generating RWE; and 2) proficiency and confidence in learning and applying new pharmacoepidemiologic methods. Exemplar topics covered include: confounding control in causal inference, propensity scores, the prevalent new user design, instrumental variables, emulated trial designs, self-controlled study designs, and the use of RWE for regulatory decision making.