More ‘Real-World’ Data Likely to Support FDA Device Reviews

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By Jeannie Baumann

Medical device makers who want to mobilize existing data sources for their FDA applications now have greater insight into how the agency decides which data to accept.

The Food and Drug Administration released its final guidance, Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices, on Aug. 30. The guidance clarifies how device companies can use electronic health records, medical claims databases, and other sources instead of relying solely on the costlier, lengthier process of conducting randomized, controlled trials.

“We think, particularly in the device space, the use of real-world evidence is really going to take off in the coming years,” Owen Faris, who oversees the FDA’s clinical trials program for devices, , told Bloomberg BNA in an Aug. 30 interview. “And we need to, as much as possible, stay ahead of that and provide information to our stakeholders that helps them stay informed.”

There’s nothing that prohibits device companies from submitting real-world data currently, Faris said, noting real-world evidence contributed to FDA approval of a heart valve. But the agency has been fielding questions from stakeholders every day about how to submit these data well ahead of designing a study.

Growing Dramatically

Janet Trunzo, senior executive vice president of technology and regulatory affairs for the Advanced Medical Technology Association, told Bloomberg BNA the device trade group is still reviewing the guidance. “We are pleased the final guidance provides additional information concerning the potential benefits of [real-world evidence] for premarket uses, such as reducing premarket burdens and supporting expanded indications of use.”

The terms “real-world data” and “real-world evidence” aren’t interchangeable terms in the guidance; “data” refers to the raw health information, whereas “evidence” refers to the use of those data.

The guidance comes out as the National Evaluation System for health Technology (NEST), a network led in part by the agency to build a national system for generating better medical device evidence, has started to take off. The 21st Century Cures law ( Pub. L. 114-255) to spur new drugs and devices also directs the FDA to establish a program to evaluate the potential use of real-world evidence.

“There is a lot happening in this space,” Faris said. “It’s moving quickly and in order to be able to provide the guidance that folks need and bring this forward, we felt like it needed to move relatively quickly.”

The FDA released the draft version a little more than a year ago, which is a quick turnaround by the agency’s standards. Faris, whose official title is the clinical trials director for the Office of Device Evaluation in the FDA’s Center for Devices and Radiological Health, said the agency is trying to improve its turnaround time overall.

Clinical Investigation Requirements

Most of the changes from the draft to final versions were minor clarifications, Faris said. But in response to comments, the agency added more robust language on when the collection of real-world data would trigger an investigational device exemption, the FDA authorization needed to conduct a clinical investigation on a device.

Real-world activity that occurs during the normal course of clinical practice wouldn’t require an IDE, Faris said, but any real-world data-gathering activity that influences how patients are treated may require one.

“If you’re treating patients under the normal course of care, we don’t want to make it harder for you to learn from that information,” Faris said. “We wanted to provide some direction and some assurance that that’s something we want to encourage doing, so long as appropriate protections are in place when you’re treating patients in part due to a desire to study them.”

Any activity that requires an IDE must be reviewed and approved by an institutional review board before initiating the project.

New Factor in IDE Analysis?

Carol Pratt, an attorney with Lee & Hayes PLLC based in Portland, Ore., who specializes in FDA regulatory issues, said the FDA’s requirements for deciding when an IDE would apply for real-world data was particularly interesting. The agency’s interpretation appears to introduce a new factor in traditional IDE analysis, she said.

“The legal authority for potentially requiring an IDE is curious considering the definition of a clinical investigation is limited to research involving human subjects, which does not include research involving only existing data, and does not include the variable of whether treatment of patients will be affected by analysis of the data,” Pratt, a Bloomberg BNA health-care advisory board member, said.

The FDA’s rationale for imposing this new variable apparently related to whether the specific activity influenced treatment decisions of specific patients, Pratt said.

“In my view, this rationale seems tenuous and not rooted in legal authority,” Pratt said, adding the agency didn’t cite any legal authority in the guidance. “It will be interesting to see if this aspect of the guidance attracts comments from clinicians and medical societies.”

FDA Not Playing Data Favorites

The guidance doesn’t include a list of which data sources the agency would accept and specifically says the FDA doesn’t endorse one type of real-world data over another. Rather, the guidance goes through the factors the FDA will consider when deciding whether information collected from electronic health records, billing claims, and other areas of health data are reliable and relevant enough to be submitted as part of a device application.

“This is a little bit of a philosophical document, helping folks understand how we think about real-world data,” Faris said.

While the guidance covers a burgeoning field, Faris said, the guidance is consistent with the way the FDA has already conducted reviews of new medical products.

“We’ve always been an agency that wants to rely on high-quality, relevant, reliable information,” Faris said. “This isn’t a lowering or changing of the bar in any way. This is just: How we can get that relevant, reliable information in way that’s less burdensome, that’s more informative and fits in where the health system is moving?”

The FDA released the guidance on its website Aug. 30, but the Federal Register notice will go out Aug. 31.

To contact the reporter on this story: Jeannie Baumann in Washington at

To contact the editor responsible for this story: Randy Kubetin at

For More Information

The FDA guidance is available at The pre-publication notice in the Federal Register is available at

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