FDA looks to bolster regulatory decisions using real-world data

The Food and Drug Administration is ramping up its efforts to leverage billing, claims, EHR and registry data to better inform the agency’s regulatory decisions regarding drugs and other medical products.

This real-world evidence (RWE) could help to “answer questions that are relevant to broader patient populations or treatment settings where information may not be captured through traditional clinical trials,” according to FDA Commissioner Scott Gottlieb, MD.

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“We are expanding our ability to use RWE for post-marketing safety surveillance, and exploring its potential to help support expanded label indications,” wrote Gottlieb in an August 29 blog post.

The agency has two active post-market monitoring systems—the National Evaluation System for health Technology (NEST) for medical devices and the Sentinel system for drugs and biologics.

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Gottlieb noted that the FDA Reauthorization Act of 2017 (FDARA), which last year revised and extended the user-fee programs for drugs, medical devices, generic drugs and biosimilar biological products, provided funding to evaluate how RWE can be generated as well as the potential for the use of this data in product evaluations. In particular, he said the FDARA funding included significant new resources to enhance the FDA’s Sentinel system, which so far has used claims data and EHRs to assess safety.

“The FDA is now supporting the first randomized prospective intervention trial that makes use of information in the Sentinel system,” according to Gottlieb. “To take one practical new example of this application, the IMPACT-Afib trial will test an educational intervention to address the important public health problem of underuse of effective medications to reduce the risk of stroke in patients with atrial fibrillation. This proof-of-concept trial can serve as a prototype for future RWE trials.”

In addition, Gottlieb said another agency-funded proof of concept study is examining whether RWE generated using observational data can replicate the results of about 30 randomized controlled clinical trials for drugs.

When it comes to active surveillance for medical devices, he pointed out that the FDA’s Center for Devices and Radiological Health has made “building out” of NEST one of CDRH’s top priorities.

“The goal is for this to ultimately help drive the development of safer, more effective devices and timelier patient access to those devices,” wrote Gottlieb. “It will also increase the value and use of real-world evidence to support the needs of multiple stakeholders in our healthcare system, including the detection of emerging safety signals. NEST may also eventually be used to facilitate reimbursement—the Centers for Medicare and Medicaid Services serves on the NEST Governing Committee—as improved data collection can help encourage coverage with evidence development.”

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