Real-World Evidence — What Is It and What Can It Tell Us?

Randomized Controlled Trials (RCTs) can be problematic - expensive, large with difficulties in enrolment, and arguably lack external validity. Nonrandomized observational analyses are been promoted as alternatives to RCTs. But is RWE the solution?

While RWE can be viewed as a means of incorporating diverse types of evidence into decision-making, the confluence of data from uncertain quality and origin will only generate incorrect or unreliable conclusions. In order to realize the full promise of RWE, we must be clear about what it is and how it can be used most effectively.

This important 2016 article via the NEJM distinguishes two key dimensions of RWE: the setting in which evidence is generated; and the methodological approach used to conduct the surveillance or research.

The authors conclude that 'real-world' research and the concepts of a 'planned intervention and randomization' are entirely compatible. Indeed, one of the most important advances in clinical trial methodology may be the broadening of the application of randomization outside the more typical venues for clinical trials.