FDA to Allow ‘Real-World Evidence’ for Approved Drug New Uses

Stay ahead of developments in federal and state health care law, regulation and transactions with timely, expert news and analysis.

By Jeannie Baumann

Drug companies could use existing data sources to support new uses for approved drugs under a policy under development at the Food and Drug Administration.

The forthcoming new policy would increase the agency’s use of real-world evidence to inform regulatory decisions, which FDA Commissioner Scott Gottlieb said can make the medical product development process more efficient and cheaper.

Widespread adoption of real-world evidence also can help doctors and patients make more effective and efficient medical choices, he added. “This will ultimately help us achieve better outcomes, and safer and more efficient use of expensive technology.”

Gottlieb announced the developing policy Sept. 19 during a two-day National Academies meeting on methods for collecting and evaluating real-world evidence. The FDA sponsored the meeting.

Smaller Initial Studies

Sonali P. Gunawardhana, a former FDA attorney who is now at Wiley Rein LLP, told Bloomberg BNA the idea behind this developing policy is that drug companies complete smaller, initial studies to demonstrate safety and effectiveness for the proposed new indications. They also then agree to conduct postmarket studies that further evaluate safety and effectiveness. While not every regulated product will be eligible for this approval process, she said, real-world evidence will shorten the clinical trial process, which may result in savings for the drug company.

“The ultimate savings may vary, but the reality is that the quicker you receive FDA approval and are able to market, the more money the company may be able to make, though there is a cost associated with postmarket studies as well,” Gunawardhana wrote in a Sept. 19 email.

Already Used in Devices

The two-day workshop occured less than three weeks after the FDA released final guidance on using real-world evidence to support regulatory decisions for devices. Gunawardhana, who is also a member of Bloomberg BNA’s health-care advisory board, said the use of real-world evidence on the device side has allowed several medical devices to be marketed more quickly. “I believe the Center for Drug Evaluation sees the merits of this process to further streamline the process for drug evaluations,” she said.

Gottlieb said the adoption of real-world evidence probably makes more sense on the device side. Doctors administering devices are also the ones using them, and therefore the ones who are collecting any data. But there is less opportunity to capture drug data when an individual patient takes the drug, sometimes without a physician’s supervision.

“There’s this more natural construct on the medical device side with these kinds of approaches,” Gottlieb said. “But that’s actually a good thing.” New concepts typically will gain traction in one FDA center, he said, and then migrate to other parts of the agency.


Robert M. Califf, who was Gottlieb’s predecessor at the FDA, told Bloomberg BNA he sees real-world evidence as a solution to many problems in the health system.

“The health system’s saying ‘We have to make decisions with terrible evidence for what really matters. We don’t really know what the value of a drug is because no one did the study to find out,’” Califf said.

Using existing data could reduce overall costs of product development, leaving money for longer-term follow up studies."If we’re spending all of our money on the artificial, old fashioned, regular randomized trial, then there’s nothing left to generate the evidence that you need,” Califf said.

Confusion Lingers

Eleanor Perfetto, senior vice president of strategic initiatives for the National Health Council, an umbrella group of patient advocacy organizations, said unclear definitions for real-world evidence have led to confusion in the patient community.

“We need some really clear definitions with really clear explanations for patients,” she said during a panel that focused on patients. “This information is overwhelming and very complicated.”

Gottlieb, in his keynote address, acknowledged the FDA isn’t always clear about its approach to real-world evidence in regulatory decisions.

The FDA will be publishing consensus definitions on real-world evidence to clarify how different parts of the agency use this information as one component of its regulatory considerations, Gottlieb said. These definitions will include a detailed description of how to use real-world evidence to satisfy aspects of FDA’s pre- and post-market requirements, he said, and will be part of a guidance document under development.

For medical devices, the agency currently defines real-world data as information relating to patient health status and the delivery of health care routinely collected from a variety of sources such as electronic health records or mobile apps. Real-world evidence is the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of real-world data.

Problem in Drafting Cures Law

Califf said there was a problem with the drafting of the 21st Century Cures law ( Pub. L. 114-255), the biomedical innovation law to spur new drugs and devices, that added to the confusion about what real-world evidence is.

“It said essentially that real-world evidence is everything but randomized trials, and that’s not what we intended,” Califf told Bloomberg BNA. “The key is that real-world evidence is getting the data from the real world.” Those data can still be randomized, he said. “It’s done in the real world in business every day. It can also be done in medicine.”

Congress updated the legal definition, but Califf said that definition, which he called a “total misfire,” keeps showing up.

“The Cures legislation was really great,” he said, “but I had certainly let people know how unhappy I was” about the description of real-world evidence.

The FDA has a legal requirement to pursue real-world evidence through Cures and the user fee law ( Pub. L. 115-52), which sets the rate for fees the agency can collect to review medical products.

Andrew Powaleny, a spokesman for the Pharmaceutical Research and Manufacturers of America, told Bloomberg BNA the brand-name drug industry group supports Gottlieb’s efforts. “We do look forward to working with the agency on this, and we are pleased with the agency moving forward on implementation of the real-world evidence provisions under PDUFA VI as well as 21st Century Cures,” he said, referring to the Prescription Drug User Fee Act that’s in the latest user fee law.

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bna.com

To contact the editor responsible for this story: Randy Kubetin at RKubetin@bna.com

For More Information

The National Academies workshop is available at http://www.nationalacademies.org/hmd/Activities/Research/DrugForum/2017-SEP-19.aspx. Gottlieb's keynote address is available at https://www.fda.gov/NewsEvents/Speeches/ucm576519.htm.

Copyright © 2017 The Bureau of National Affairs, Inc. All Rights Reserved.

Request Health Care on Bloomberg Law