Using Real-World Evidence

Understanding how treatments work in the real world is an important and complex task to guide care in real-world, clinical practice environments. That’s because patients have characteristics and treatment experiences that often differ from the controlled environment of clinical studies. To improve patient care, more research is being targeted toward understanding how a treatment, service, or method of delivering care works when applied in real world, clinical practice environments.

By conducting “real-world” studies, health care stakeholders are thinking beyond traditional randomized controlled trials (RCTs), which have long been considered the gold standard of scientific studies. They are making use of real-world databases that record the processes of care patients receive, such as electronic medical record (EMR) and medical claims databases. They also are working with patient registries that focus on patients with a specific disease or who receive a particular treatment or device and record the medical outcome for those patients; or these registries could be based on a specific population so that researchers can see what happens within that population.

Another way of looking at the real world is via practical clinical trials that relax some of the constraints of a normal trial and better reflect the real world conditions of clinical practice. Participants in routine RCTs usually must meet very precise clinical criteria, and thus the information gained from the trial may not be “generalized” to a broader group of patients. Typical RCTs also define the frequency of visits and tests to be performed, and compliance with treatments is closely monitored and controlled. These characteristics are not typical of usual care patients receive and thus the real benefits over usual care are more difficult to assess. In practical trials, patients may be randomized to treatments under study, but subsequent care may be determined by the doctor based on his or her clinical judgment. Practical trials often include a comparison group receiving usual care rather than a placebo comparison.

Despite this movement toward the use of real-world evidence, some stakeholders remain reluctant to rely on real-world studies. Payers, for example, prefer to use RCTs instead of external observational data to inform drug coverage decisions.

Part of the reason for this is because there are multiple standards and methods for conducting analyses with real-world clinical experience evidence. When done studies are done correctly and adhere to good practices, real-world evidence can provide meaningful information; when done poorly, misinterpretations and poor health outcomes for patients are possible. A set of methods and standards for collecting real-world clinical experience evidence developed in consultation with multiple stakeholders and utilized by the Food and Drug Administration may encourage others to accept high-quality studies and disregard poor-quality studies.