In the world of scientific exploration, researchers work very hard to control as many variables as possible to ensure that what they’re studying is not impacted by extraneous variables that might taint the results.
In the “real world,” though, those variables aren’t controlled and the insights gleaned can be very different from those attained through controlled studies. Researchers, clinicians, and patients are beginning to recognize and value the different results or insights that they may attain through real-world evidence (RWE).
Data vs. Evidence
There’s an important distinction to be made between data and evidence. Data represents the numbers and statistics that are gathered through electronic medical records, patient records, wearable/electronic devices, and so on. Evidence is what is generated through actual use cases — what are the impacts when real people use drugs in real-world settings and situations?
Between the two extremes of carefully controlled research and lived patient experience lies the hybrid of real-world evidence, which can provide a higher level of reliability and validity. This requires gathering, synthesizing and analyzing data from a wide range of sources.
The Inherent Limitations of Clinical Trials
Brad Rodu, professor of medicine at the University of Louisville has conducted and published research in both areas. Clinical trials, says Rodu, “are powerful tools using sophisticated scientific methods to precisely determine the effectiveness of treatments for specific diseases.” They are, though, “ill-suited to the measurement of behavior and/or consumers’ preferences.” For such measurement, he says, RWE, which may consist of surveys and post-market surveillance, is a better tool.
David Handelsman, vice president for the non-profit Project Data Sphere, a digital library-laboratory platform, says that clinical trials are beneficial because they allow for the collection of high-quality, targeted information in response to some hypotheses posted as part of a clinical trial protocol. The result is an idealized sample of a “true patient population.” But, he adds, “real patients exist in the real world, and have real and complex health care needs.
“RWE and/or case studies can serve to exemplify behaviors that are typical problems in the area of patient/client/customer satisfaction. They often mimic large studies without the extended time and large financial investment required, and so serve to launch a cost-cutting or service-oriented change project,” says Candy Campbell, CNP, assistant professor, MSN-CNL Maternity Lead at the University of San Francisco School of Nursing. In contrast, clinical research is time-consuming — and expensive, requiring a team of experts to gather and review data, obtain permissions, and analyze data and, in the case of experimental research, years of review.
If your house is on fire, says Campbell, “you don’t need a research study to justify your call for help!”
Applications of Real-World Evidence
Using RWE provides a more agile research process, meaning patients may have access to promising drugs sooner than in the past. “The traditional process used to study new drugs before they go to market, the classic ‘interventional trial’, is quite rigid and relies on systems and processes that are remarkably disjointed from the flow of routine clinical care,” says Chris Kish, the VP of data strategy for Patient iP, a clinical research firm that uses RWE to inform its recruiting strategies for clinical trials.
There is a wide degree of variability among patients, Handelsman notes. That means, he says, that they “may have confounding health conditions, may be using additional treatments and the data collected from them may vary considerably in quality.” Real-world data, while much more difficult to analyze because of its variability and lack of standardization and structure, “can provide the necessary information to understand how patients respond in the real world,” he says. “This provides a unique opportunity to compare the effectiveness of treatments that compete in the marketplace, something rarely addressed in clinical trials.”
Today, with so many ways to collect and share reliable data, new opportunities are emerging. Electronic medical records, for instance, now provide a way to capture data from a much broader range of subjects than in the past, even if data extraction still is problematic. Even still, what has proven effective is to collect thepublished and presented results of real world research; not only the EMRs, but the cohort studies, retrospective and prospective studies, research from global registries, investigator initiated research, etc – and analyze those records to get a complete picture of what is occurring in the real world.
Additional Benefits of Real-World Evidence
In addition to providing insight into how the effects of medications are experienced by people in uncontrolled settings, RWE allows for much broader research. Clinical studies are inherently small in order to control for as many variables as possible and ensure the reliability of the data gathered. In RWE the controls are removed, and a much wider net can be cast to capture information.
RWE also allows for better understanding of rare diseases where few patients are available for clinical trials. “To truly understand the effectiveness of treatments for rare diseases, the larger amount of patients — and data — available provides unique opportunities to study outcomes in different patient subgroups, and over a longer term,” says Handelsman. In addition, he says, rarely occurring adverse events, or side effects, also can be best studied through RWE. “With larger pools of patient data, either by aggregating patient-level data from multiple manufacturers, or by collecting real-world data, it should be possible to develop new diagnostic tools and treatment protocols that help to minimize the risk of these toxicities that may exist,” says Handelsman.
While there will certainly always be a place for scientific clinical research, scientists, clinicians, payers and even the FDA, are increasingly recognizing the value that RWE holds for understanding the impact of pharmaceuticals among various populations.