Real-world evidence in clinical research: challenges and opportunities


Medical Manager, Prineos, Italy

Randomized controlled trials (RCTs) are considered the scientific gold standard for demonstrating safety and efficacy of an investigational treatment, as well as obtaining its regulatory approval and authorization for marketing. However, the RCT paradigm is neither perfect nor infallible (1, 2).

One pitfall of RCTs is that the study population does often not reflect the broader population managed in routine clinical practice, i.e. the real-world setting. Indeed, participants to RCTs are usually less clinically complex (e.g. they tend to be younger, healthier and on treatment with fewer drugs), and their number and the study duration are very limited (3, 4).

In light of the need to generate evidence in a real-world setting to inform healthcare decisions, with the ultimate goal to improve the efficiency of clinical research, the Real-World Evidence (RWE) i.e. the evidence generated through the analysis of Real-World Data (RWD), has assumed increasingly greater importance over time. It is increasingly recognized that RWE allows to bridge gaps in information that cannot be met by RCTs alone. Moreover, recent advances ...