The FDA said it plans to accept new forms of real-world evidence in product applications, starting with a subset of medical device submissions.
The agency said the policy change aims to enable the use of more types of massive, deidentified databases—including those collected through national registries, health systems, insurers and electronic health record networks, potentially spanning millions of people receiving care outside of clinical trials.
Previously, the FDA preferred that any real-world datasets offered up for review were capable of being traced back to the health outcomes of individual patients, with links to their private and confidential health information.
Now, the agency said it will start considering the strength of submitted real-world evidence for medical devices on a case-by-case basis—and that it will consider extending the new policy to drug and biologic reviews in the future.
“We're removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster,” Commissioner Marty Makary said in an agency statement. “This common-sense reform will unlock access to vast databases like cancer and cystic fibrosis registries that contain critical insights about how treatments work in the real world.”
According to the FDA, more than 250 medical device green lights have been issued since 2016 to applications incorporating real-world evidence—while 35 drugs, biologics and vaccines have included the data in their submissions during the same time period. However, the agency said that the rate of those device authorizations “has plateaued in recent years.”
In one example, Johnson & Johnson MedTech was able to secure a 2023 approval enabling a zero-fluoroscopy workflow for some of its radiofrequency ablation catheters used to treat heart arrhythmias. The company submitted an observational, prospective registry dating back to 2019 that tracked ablation outcomes in atrial fibrillation, alongside the procedure’s efficiency and safety, showing that it could be performed using ultrasound and without doses of X-ray radiation.
In its release, the FDA specifically pointed to the National Cancer Institute’s Surveillance, Epidemiology, and End Results database, known as SEER, as a potential source. Its cancer statistics, going back decades, are also used to compile the government’s annual report on incidence rates, deaths and trends among the most common tumors.