The plethora of data available from electronic health records, claims systems, registries and from patients themselves is an unprecedented opportunity to modernize and augment clinical trials, while improving the current drug development process through the use of real-world evidence.
That’s the conclusion of a new report released Thursday by the Bipartisan Policy Center, which contends that real-world evidence can only be derived from real-world data gathered from sources outside of randomized controlled trials. The best data can come from sources such as EHRs, because it reflects the actual experiences of patients during routine care.
“Bringing safe and effective cures and treatments to patients in a timely and cost-effective manner will require new processes and new policies to support them,” according to Bill Frist, MD, former Senate majority leader, and Bart Gordon, former Congressman, who were co-chairs of BPC’s initiative, entitled, “FDA: Advancing Medical Innovation” initiative.
“Using real-world evidence—data that reflects the actual experience of patients during real-world situations—in addition to relying on data derived from conventional randomized controlled trials—will enhance the nation’s ability to advance the safety and effectiveness of drugs in a wider population,” Frist and Gordon wrote in the report.
According to the authors, other sources of valuable real-world data include patient-generated data captured on home-based and wearable monitoring devices, as well as patient information-sharing networks and social media.
“The significant increase in the use of EHRs within the clinical setting, combined with the surge in the number of Americans recording health information through devices and information-sharing applications, is producing unprecedented amounts of information useful for regulatory decision-making,” states the BPC report. “All of these are sources of potentially useful health data that can complement data generated through controlled trials.”
“We’ve used unreal evidence,” says Andrew von Eschenbach, MD, president of Samaritan Health Initiatives, a former FDA commissioner, and a member of BPC’s advisory committee. “But what we’re finding increasingly is that those clinical trials don’t reflect necessarily the real world, which is the general population when you can’t control all those variables. If we’re going to continue to be confident and comfortable about our decisions as to the value of drugs, their effectiveness and relative risk, we need to expand the information that we are gathering.”
While the U.S. Food and Drug Administration is already using real-world data to support post-market surveillance efforts, the authors note that leveraging real-world data is less common when generating evidence through clinical trials to support drug approval.
However, the report suggests that “pragmatic” randomized clinical trials—ones that take place in settings where routine care occurs, such as community clinics, hospitals, and health systems and they involve diverse, representative populations—could begin to build experience with the use of real-world evidence within clinical trials.
“Real-world evidence can enable more efficient and targeted recruitment of patients for clinical trials,” concludes BPC. “It can also reduce the burden of data collection and reporting and enable the collection of patient-reported outcomes.”
Nonetheless, to effectively use real-world evidence, the authors argue that several difficult—but surmountable—data challenges exist, including lack of data completeness for clinical research, lack of adoption of standards to support effective interoperability and use of the data across disparate systems, lack of agreement on methods for accurate patient matching, and lack of traceability of data or data provenance.
“Work is currently underway to help address many of these challenges, but more action will be needed to support the generation and use of real-world evidence,” warns the report.
Toward that end, BPC calls on the FDA to establish a program to promote the sharing and evaluation of methods used in the evaluation of real-world evidence for regulatory decision-making. In addition, the authors urge the Department of Health and Human Services to “continue its efforts to advance the adoption of standards and the interoperability of EHRs”—efforts that should extend to other clinical systems beyond the EHR.
“HHS should develop and publish standards that will improve the accuracy of matching of patient data across health information technology systems,” states the report, as well as “continue its efforts to harmonize standards related to data used for clinical research with standards related to data within EHRs and other systems used within healthcare.”
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