Opinion: Real-world evidence is changing the way we study drug safety and effectiveness

Randomized controlled clinical trials are a great way to test the safety and effectiveness of a new drug. But when the trial is over and the drug approved, it’s used by patients and health care practitioners in settings that are quite different from the rarified clinical trial setting.

Interesting, important, and sometimes surprising findings can emerge when the narrow constraints of clinical trial eligibility and intent-to-treat analyses are set aside. That’s why it’s time for biotech and pharmaceutical companies to pay more attention to understanding, and undertaking, real-world studies, an approach backed by Food and Drug Administration Commissioner Scott Gottlieb in a talk Monday on using new data sources as evidence for both regulatory evaluation and value-based payment programs.

Just what is doesn’t offer much help there. It defines real-world evidence as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials.”