Why methodology matters in real-world evidence studies

In this video, Martin Schoen, MD, PhD, discusses key methodological considerations when interpreting comparative real-world evidence on androgen receptor pathway inhibitors (ARPIs) in prostate cancer. Schoen is a medical oncologist at the VA Medical Center and a professor at Saint Louis University in St. Louis, Missouri.

Schoen explains how patient selection, outcome definitions, and analytic approaches can influence the conclusions of real-world evidence analyses, emphasizing the value of using methods that emulate the design of a prospective randomized trial. He also highlights the importance of ensuring that patients have comparable clinical characteristics at baseline when evaluating outcomes such as progression-free survival and overall survival.

“In real-world analysis, it's the same thing; we have to use patients that have a very similar clinical scenario, and then think about it as though we were doing a randomized trial, with appropriate inclusion and exclusion [criteria],” Schoen says.

He notes that statistical adjustment can address measured confounders, but important factors such as frailty, performance status, or disease characteristics may be missing or inconsistently captured in real-world data. As a result, unmeasured or incompletely documented differences between patient groups can limit the strength of conclusions drawn from observational analyses.