Randomized Trials vs Real-World Evidence

Noting that randomized clinical trials (RCTs) “generally yield the strongest inferences about the effects of medical treatments,”(p984) the AMA Manual of Style instructs that “randomized trials may use terms such as effect and causal relationship,”(p983) In contrast, the manual claims that observational studies “cannot lead to causal inferences”(p994) and thus “should be described in terms of association or correlation and should avoid cause-and-effect wording.”(p983) Nevertheless, analyses of observational data—often consisting of real-world evidence from databases of medical claims and electronic health records—play an increasingly prominent role in health care. Given the well-recognized limitations of observational studies, their growing use to estimate treatment effects is the subject of considerable controversy. Should real-world evidence have a meaningful role in decision-making? In this issue of JAMA, investigators from the RCT DUPLICATE Initiative address this controversy by directly comparing the results of published RCTs to analyses of 3 national claims databases that are carefully designed to emulate the original RCTs. The results of their productive line of research warrant careful consideration.

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Aprile 2023