In a perspective published in the New England Journal of Medicine on Thursday, two medical device experts argue that real-world evidence (RWE) could be used to answer many questions about the safety and effectiveness of medical devices, but caution that RWE may not be appropriate for initial marketing decisions.
Background: Real World Evidence
In recent years, the US Food and Drug Administration (FDA) has worked to expand the use of RWE, such as electronic health records (EHRs), insurance claims data and information from patient registries, to improve its postmarket surveillance capabilities as well as in its decision-making for medical products.
Last August, the agency finalized guidance on using RWE to support the approval or clearance of medical devices.
Congress has also pressed FDA to incorporate RWE into its oversight of medical products in both the 21st Century Cures Act and the FDA Reauthorization Act.
RWE: Pre- and Postmarket Use
In their article, Frederic Resnic, chairman of the Department of Cardiovascular Medicine at the Lahey Hospital and Medical Center, and Michael Matheny, director of the Center for Population Health Informatics at Vanderbilt University Medical Center, argue that for now, RWE is most useful in the postmarket setting.
"It's particularly in this setting that our current systems are inadequate for ensuring the safety and evaluating the performance of modern medical devices," they write.
In contrast to postapproval studies, which FDA often mandates as a condition of approval for devices, the authors say that RWE gleaned from the healthcare system could provide data about device safety and performance from more patients more quickly and at a lower cost.
Initially, the authors say, the best use for RWE in a premarket setting would be to use electronic data sources to support pragmatic clinical trials conducted using registries or electronic health record (EHR) systems.
"Such approaches could significantly reduce the cost and time required to complete a study as compared with traditional randomized clinical trials," they write.
The authors also applauded FDA's recent decision to expand the use of Edwards Lifesciences' Sapien 3 transcatheter aortic valve replacement (TAVR) to valve-in-valve procedures based on data gathered from the Transcatheter Valve Therapy (TVT) registry.
"By all accounts, this approach cost substantially less and took significantly less time than the studies that would otherwise have been required to demonstrate efficacy in new groups of patients," the authors write.
However, the authors caution that using RWE for initial marketing decisions carries significant risk.
"Even with the most stringent methods of statistical adjustment, RWE-based analyses are observational in nature, with inherent biases in treatment choice coloring the patterns and associations that might be observed," they write.