What is Real World Evidence?

Real world evidence (RWE) refers to evidence from data sources with greater generalizability than that which is typically gained from phase I-III clinical trials. The most common form of RWE is observational studies of retrospective data such as electronic health records (EHR) or administrative claims data (such as Truven Marketscan or Optum Clinformatics). Randomized forms of RWE include pragmatic clinical trials which typically have more relaxed inclusion and exclusion criteria compared to registration trials and thus include a more heterogeneous population.

Real world evidence is of growing importance for the industry and the scientific community as robust data sources continue to grow in terms of size and quality of data and allow for studies to be completed in shorter time periods and for much lower cost than RCTs. The downside of RWE is that the lack of randomization often means less certainty with regards to potential confounding and potential for abuse through p-hacking or similar tactics due to the ease of running analyses.

Real World Evidence for Regulatory Decision-making

With the proliferation of real world data, there is growing interest for utilizing real world evidence to guide regulatory decision-making include potentially label updates. Several initiatives are currently active at the FDA and other regulatory bodies to test various options with the aim of ultimately providing a means or guidance for industry to be able to utilize RWE for regulatory purposes. In some limited instances, RWE has led to drug approval or label updates, primarily in rare diseases.

“Real World Evidence” can also be the name of an organization, often synonymous with the “outcomes research” part of HEOR.