Martin Schoen, MD, PhD, on the role of real-world evidence in ARPI selection

In this video, Martin Schoen, MD, PhD, discusses how real-world evidence can inform understanding of androgen receptor pathway inhibitors (ARPIs) and emphasizes the importance of study methodology. Schoen is a medical oncologist at the VA Medical Center and a professor at Saint Louis University in St. Louis, Missouri.

Schoen explains that real-world evidence can help address questions that have not been answered by randomized trials, particularly in the absence of head-to-head comparisons between ARPIs. Data from large practice networks, including the Veterans Health Administration, may provide opportunities to compare treatments in populations with similar clinical characteristics while accounting for baseline differences.

“We should be thinking about this not that real-world evidence is much different than prospective trials,” Schoen says, emphasizing that the same principles used to design and interpret prospective studies should guide real-world analyses.

He notes that patient selection and analytical methods are critical to the strength of comparative real-world evidence. Ideally, analyses should emulate the design of a randomized trial by applying similar inclusion and exclusion criteria, defining comparable clinical scenarios at baseline, and evaluating outcomes such as overall survival or progression-free survival. Schoen also distinguishes between indirect treatment comparisons, which generally rely on cross-trial comparisons, and matching-adjusted indirect comparisons, which account for differences in baseline characteristics between study populations.

When interpreting these analyses, Schoen emphasizes that clinicians should consider how closely the methodology reflects the clinical question being addressed. More rigorous approaches can help reduce the influence of differences between patient populations and provide a more informative comparison when direct randomized evidence is unavailable, while analyses with less careful patient selection or methodology may warrant greater caution in interpretation.