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Does the FDA Really Accept Real World Data?
(Thursday, May 16, 2024) Over the last decade, the FDA has repeatedly expressed strong interest in real world evidence (RWE) from real world data (RWD) in support of regulatory decisions but very few market approval applications significantly depended on RWE over the same period as shown in an independent review. It is critical to understand the reason for this disconnect and address them before allocating resources for RWD and RWE. FDA defines Real World Data as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” such as medical records, medical claims data, data from product and disease registries, patient-generated data including in-home use settings, and data gathered from other sources that can inform on health status, such as mobile devices. When RWD is used to support the claims of the safety and benefits of a product, it becomes RWE. FDA has published six Guidance Documents and numerous other publications and presentations describing its expectations from RWD. Despite its much-touted support for RWD and RWE, an independent review of FDA decisions showed that the FDA accepted RWE in only 90 out of 26,121 medical device applications (0.34 percent) approved between 2012 and 2019. The review highlights several cases where the FDA not only did not accept RWD but “even raised the evidentiary bar by requiring a clinical study as the only adequate means.” The authors express their deep frustration with the FDA where “despite FDA leadership’s public efforts to recognize and encourage the use of RWD and RWE, reviewers have not fully embraced RWD/RWE.” The potential of RWD/RWE “will never be realized unless reviewers are willing to accept RWD/RWE despite their limitations.” This is not a unique opinion. There is ample evidence, from all branches of the FDA, that the FDA has not trained its own staff about how to review and use RWD and RWE. RWD has several limitations; comparing and equating it with clinical trial data is not only unfair but sets a regulatory bar that can never be met. Such disregard for RWE in support of medical applications is evident when the FDA cherry-picks data from the real world to make negative decisions about products such as it did recently with Lab Developed Tests (LDTs) where more than three decades of real world experience has shown that such tests can be used which was completely ignored by the FDA is creating a new rule. Such experience discourages developers from allocating resources towards RWD/RWE. But it won’t be fair to blame the FDA alone. The industry also needs to do a better job of collecting good quality RWD. The FDA guidance documents describe the matrices for acceptable RWD. The data from RWD should be collected with the same vigor as that for clinical trial data with well planned real-world protocol, well designed case report forms, and comprehensive RW clinical reports to demonstrate reliable RWD and RWE. That said, so far, RWD and RWE are still in their early years. The hype over their use did not match the reality of their acceptability. It is still a theoretical possibility in terms of its used for FDA approval. It is important for developers collecting RWD and generating RWE to have a clear understanding of the limitations of using such data with FDA. 
AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: [email protected] Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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