FDA’s Makary talks up modernizing agency to get drugs to market faster

FDA Commissioner Marty Makary said that one of his key priorities is to modernize the agency by streamlining the drug review process, using AI tools more, and increasing transparency and clarity with the biotechnology and pharmaceutical industries to get more therapies to market faster.

“We are rethinking some of the legacy processes for how we do things at the FDA,” Makary said at the BIO International Convention in Boston Tuesday afternoon, adding that he wants to eliminate “guesswork and mind reading” out of the system.

“We are going to look at everything we can do to address the long time to get a new drug to market.”

One of the ways he sees this being done is to “custom tailor” the review process depending on the condition being treated. He cited instances where the patient population for a disease is low yet randomized controlled trials are still needed. Synthetic controls gathered from a large dataset could provide a control group in these situations.

“FDA would be in partnership with industry…and at the same time the scientific review is impeccably independent.”

He added that the agency is open to ideas from industry as well. “If you have a great idea and we adopt it, that’s not a cozy relationship. It’s a healthy partnership.”

Makary also highlighted the agency’s recent launch of Elsa, a generative AI tool to help employees, including scientific reviewers, work more efficiently. The language model–powered application “can summarize adverse events to support safety profile assessments, perform faster label comparisons, and generate code to help develop databases for nonclinical applications,” according to the agency.

He added that a “version 2.0” would be rolled out in the coming weeks.

Makary also addressed cuts made by DOGE at the agency, noting that the positions eliminated were not to scientific reviewers or inspectors. He noted that there were many redundancies across FDA offices, such as each having their own IT, communications, legislative, and travel staffs. He wants to see more teamwork among departments and sharing of resources.

When he started several months ago, he described the agency as a “fiefdom culture where centers were built independent of each other.”

He contended that the cuts have had no impact on the FDA meeting PDUFA deadlines, and he is ensuring that staff have the resources they need, adding that the agency is hiring due to turnover, and is looking to “replenish our staff from people on inside and outside.”

Earlier Tuesday, the FDA announced a new national priority voucher program that aims to reduce the time to review a drug to 1-2 months after a company’s final drug application submission.