WASHINGTON, D.C.—FDA Commissioner Martin Makary, M.D., offered more details into the agency’s recently announced plans to place artificial intelligence programs at the center of its medical product reviews as well as his goal to have those systems up and running by the end of June.
Speaking at the annual conference of the Food & Drug Law Institute in Washington, D.C., Makary said implementing AI is one of the agency’s top priorities.
“On day one, we pulled together some really talented AI minds, both within the agency and outside, and we asked them, ‘What can we do?’” Makary said.
“We got our scientific reviewers together and had a pilot to have some open and frank conversations about some of the work that they would love to see addressed with AI,” he said, pointing to submission packages that may contain thousands of pages with a lot of repetitive background information.
“How do I pick out the key points that are unique in one article, that are unique from another article? AI can do that really well. It can summarize,” Makary added. “So they've taken this massive amount of content and the AI is summarizing it for our scientific reviewers. The first reviewer that was engaged in this pilot … said that the AI did in six minutes what it would normally take him two to three days to do.”
“So by June 30, we're going to have that AI-assisted feature available to all scientific reviewers, and we're going to keep going,” he said. “The other piece, of course, is how we regulate new AI technologies. We have to think creatively about that.”
Makary also pitched potentially linking FDA reviewers into clinical study data as trials are being performed.
“Can we think really big and ask, can we run more continuous trials, with the endpoints in the cloud available to the reviewers to look into—rather than resubmitting large applications after each step?” he said.
“You wouldn't submit a 64-page application after your freshman year to start your sophomore year in college, and again for your junior year and again before graduation day. We can think creatively about ways to run more continuous trials and use cloud-based endpoints.”
When it comes to regulation, Makary also proposed shaping the FDA’s approval processes toward the disease in mind—while committing to the agency’s current center-by-center structure based on the type of product being reviewed.
“We want to rebuild the public trust by combining gold-standard science and common sense—and you’ve got to use common sense, especially when it comes to conditions where a randomized, controlled trial may not be feasible,” he said. “Maybe in rare diseases, or certain incurable conditions—you've got to tailor the approval process to the condition where the application is sought for.”
There will be “no major re-org” at the agency, he continued, addressing the rumors of a plan to reduce or consolidate the FDA’s drug, biologic and device centers. But, he added—and following the reductions in force seen over the past few months—the new leadership in the Trump administration wants “to challenge deeply held assumptions when it comes to medical science and the processes at the FDA.”
“There was a bit of territorialism with the centers. It was a bit of empire-building, and we want to see more teamwork,” he said. “We want to see less tribalism, less duplicative services, and not only at FDA.”
“In [the Department of Health and Human Services (HHS)], there were nine offices for women's health. Do we need all nine? Because it's an important cause, with an important title—and the office sounds important, because it is—that doesn't mean there cannot be benefits from consolidating.”
Late last month, the Women’s Health Initiative, a nationwide study launched in 1991 with National Institutes of Health grants, announced that the HHS planned to cut off its funding later this year. The HHS reversed course within days.
In terms of the FDA’s staffing, Makary said the layoffs have brought the agency back to its pre-COVID head count, circa 2018 and 2019.
“It was a little hard coming in after the change with the reduction in force. We're doing everything we can to do an assessment, restore some services and individuals, and also to try to rebuild the culture, because it is a great culture. The FDA is a great brand,” Makary said. “I think it is perhaps the greatest brand in the world, and so we have an obligation to uphold that ancient tradition in this country.”
When it comes to vaccines, Makary said the FDA wants to create a transparent framework for developers so they can understand the agency’s thinking. He said Vinay Prasad, M.D.—the recently named director of the Center for Biologics Evaluation and Research (CBER) after its previous chief, Peter Marks, M.D., Ph.D., was forced out of his position last month—plans to meet with industry and CBER staff in the coming days to help build those guidelines.
“He also believes in gold-standard science and common sense, especially when it comes to rare diseases,” Makary said about Prasad, who was recently a professor at the University of California, San Francisco. “He is bringing the perspective of a hematologist, where some of these drugs that have been approved, we don't know if it influences overall survival.”
Makary also described the agency’s goals for expanding post-approval surveillance along with plans to build a large database linked to electronic health records to help spot emerging safety signals as soon as a drug is approved.