Pandemic pushes FDA to 'accelerate' real-world evidence efforts
The Food and Drug Administration has worked to build up its use of real-world data for evaluating COVID-19 diagnostics, participating in a partnership exploring how to use such data to improve testing and help understand the virus' spread.
Using real-world data and evidence in regulatory decision-making has been on the FDA's agenda for years. But the pandemic sped up the adoption of newly generated data so that guidelines could be updated for coronavirus tests that were cleared for emergency use based on limited information, Commissioner Stephen Hahn said at an event hosted by the Medical Device Innovation Consortium in June.
That month, the FDA joined the COVID-19 Diagnostics Evidence Accelerator, an initiative organized by the Reagan-Udall Foundation, a nonprofit that supports FDA, and Friends of Cancer Research. Among other efforts, the diagnostics accelerator will tackle a key question in understanding of the virus: whether the presence of antibodies signals protection against future infection, and if so, which ones help.
Real-world evidence provides insights on the benefits or risks of a medical product taken from real-world data, which is information derived from registries, electronic health records, claims and billing data, as well as mobile devices and other sources that reflect patients' actual use of products.
For drugs, FDA leaders have previously signaled that, while companies shouldn't rely too heavily on real world evidence, the agency is open to evaluating this information and allowing it to be used for expanding a drug's label. The agency has also said it is advancing real-world data tools to help speed up development of COVID-19 treatments. The FDA said these tools will help analyze outcomes and illness patterns, and could help to supplement placebo-controlled trials.
COVID-19 has lent urgency to the FDA's efforts on RWE, both for therapeutics and for diagnostics.
In mid-May, the agency engaged Aetion, a New York-based healthcare technology company that specializes in analyzing real-world evidence for payers, providers and regulators, to address questions about tests and medications — as well as risk factors for COVID-19 complications in various patient populations. The agency also said it's leveraging Sentinel, its electronic system used to surveil medical product safety.
But the FDA's focus on real-world evidence will continue beyond the current COVID-19 pandemic, Hahn said.
"Now as we look forward, we need to think about how real-world evidence fits into the initial authorization or approval of a product," he said. "But on the [post-approval end], what data can we collect to look at really how these devices, how the tests operate, how they perform, and then incorporate that back in?"
With the agency leaning heavily on the emergency use pathway during the COVID-19 public health crisis, the need to revisit authorizations once new evidence emerges is arguably more apparent now than ever before.
The agency has shown that it's willing to reverse course on the products it allows during the public health emergency, as additional insights on efficacy come to light. In June, for example, the FDA revoked the emergency use authorization for a COVID-19 antibody test from Chembio, one of the earliest of such tests to have received an EUA, or emergency use authorization.
"You take the data you have, you take the information you have, you make decisions, and then you update it as you go based on new information," Hahn said during the June event. "And I think that's what we've seen here during this pandemic. It's what the agency has always strived to do, we've just done it in an accelerated timetable."
Hahn said the agency has tried to balance the need for speedy access to products that can help treat the new coronavirus while continuing to ensure safety and effectiveness, as well transparency about its processes.
Still, "in a fast-moving pandemic, that's been a challenge," he said.
The FDA has been criticized for getting that balance wrong in some cases. For example, it allowed scores of antibody tests onto the market without agency review under an umbrella-type policy aimed at increasing the volume of tests available. Many of those tests were found to have high rates of false results, prompting the agency to later order some off the market and require emergency use authorizations for all test makers wishing to continue selling a product.
The agency was also scrutinized for its handling of hydroxychloroquine, which it cleared for emergency use early on only to later revoke the authorization once more data showed the drug unlikely to benefit COVID-19 patients.
Article top image credit: Dufour, Tia. (2020). "White House Press Briefing" [Photograph]. Retrieved from Flickr.