Real-World Evidence

Real-world evidence (RWE) can help health care decision-makers understand how a treatment, service, or method of delivering care works when applied in real-world, clinical practice environments. But high-quality methods and standards must be used to conduct real-world research and decision-makers need tools to evaluate and use RWE. 

What is real-world evidence? 

Real-world studies investigate what treatments work best in the real world under usual care settings, instead of highly controlled environments like randomized controlled trials (RCTs). Real-world evidence (RWE) comes from real-world data (RWD) – information that is obtained during routine clinical practice – and can be gleaned from the data in electronic health records, administrative claims databases and patient registries. It can complement existing research, help fill gaps in knowledge, and support decision-making in clinical practice, treatment coverage and reimbursement.

RWE is used in governmental, nonprofit and private industries by health care decision-makers, insurance providers, academia, policymakers, biopharmaceutical companies, employers and payers. The National Pharmaceutical Council (NPC) has been playing a leading role in encouraging the use of high-quality methodology and standards to conduct real-world research and help decision-makers understand how to evaluate and use RWE.

Why does RWE matter?  

RWE is growing both in availability and in its ability to capture the real-world experience of living with a health condition.  One in every two Food and Drug Administration (FDA) approvals in 2020 included RWE studies in the regulatory submissions, a trend that’s expected to grow. RWE helps health care decision-makers answer an important question: does a treatment, service, or method of delivering care work when applied in real world, clinical practice environments?

What’s the difference between RWE and evidence from RCTs?  

Patients have characteristics and treatment experiences that often differ from RCTs, which are conducted in the controlled environment of clinical studies. RCTs alone are not able to answer every question about how drugs will perform in the real world. 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. RWE from well-designed real-world studies helps researchers and other stakeholders draw meaningful conclusions about which interventions are the most effective treatment options for specific conditions and patients. Typical RCTs 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 that patients receive and the real benefits over usual care are more difficult to assess. 


Despite this movement toward the use of RWE, some stakeholders remain reluctant to rely on real-world studies. Part of the reason for this is because there are multiple standards and methods for conducting analyses with real-world clinical experience evidence. When studies are done correctly and adhere to good practices, RWE 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 used by the FDA, may encourage others to accept high-quality studies and disregard poor-quality studies.

In a study, over 50% of surveyed health plan pharmacy and medical directors said that a pivotal barrier to RWE use was their personal or their organization’s lack of experience in using online tools that help to interpret RWE studies.



digital toolbox

How can available tools help stakeholders evaluate real-world evidence?

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