All the figurative FDA trains are running on time, according to the agency’s leader Marty Makary, M.D.
“I'm proud to report that we're on track to meet all the PDUFA targets and that morale is good and improving at the agency,” Makary said June 17 at this year’s BIO, an international convention that has attracted 23,000 attendees.
During a fireside chat with the FDA commissioner, BIO CEO John Crowley didn’t ask Makary about the recent agency delay to KalVista Pharmaceuticals’ investigational drug approval decision. The biotech claimed it was informed last Friday that the FDA would miss the June 17 PDUFA date due to “heavy workload and limited resources.”
But Makary told a different story about the state of the organization.
“The agency is strong, and my job is to make sure that every scientist and inspector and law enforcement official at the FDA has all the resources they need to do their job well,” Makary said.
Nominated for the position by President Donald Trump, Makary has been in office for about 10 weeks, taking the helm of the federal drug agency amid a massive overhaul that included laying off more than 3,000 FDA workers.
The terminations were said to align with Trump's executive order titled “Implementing the President’s ‘Department of Government Efficiency’ Workforce Optimization Initiative.”
When asked about DOGE-related cuts, Makary said he has “reinstated people at the FDA” and that the agency is currently “hiring scientific reviewers.”
“I do think, however, that some of the consolidation—in fact, most of it—has been good,” he continued. “We did not need nearly 2,000 HR procurement and budget staff at the agency.”
“When there are tremendous redundancies in IT or communications or legislative affairs or in the 12 travel offices or in the HR staff, those are opportunities for us to create more efficiencies and ultimately improve morale,” he explained, describing the previous culture at the FDA as a “fiefdom” of many autonomous centers within one agency.
The layoffs didn’t impact scientific reviewers or inspectors, according to the FDA leader, who added that he is making sure “core staff” have the required resources to keep “all the trains running on time.”
In fact, Makary wants the trains to arrive even earlier. The leader just announced a pilot program designed to speed up drug reviews for biopharmas that align with “national priorities.”
The nontransferable priority review vouchers can be tied to a specific investigational drug or can carry an “undesignated” status and be used for the biopharma’s novel drug of choice.
During the BIO fireside chat, Makary said the point of the program is to utilize “a lot of dead time.”
“Why does it take over 10 years for a new drug to come to market? What systems at the FDA should be reevaluated? Where is the wasted time in the system?” he asked. “I'm not talking about cutting corners on a scientific review. We're talking about unnecessary steps—avoidable delays.”
To qualify, sponsors must submit chemistry, manufacturing and controls and draft labeling information at least 60 days before submitting a final application.
The process means final clinical trial results will be submitted “essentially as an addendum” or a second part of the final application, Makary explained at BIO. In total, the new program aims to shorten the review process from 10 to 12 months down to one or two months.
“We're going to have to work some things out, but we have to try new things,” he said. “We are seeing an increasingly competitive landscape internationally. We are seeing the prolonged time and wasted steps result in a loss of investment.”
Makary, a pancreatic surgeon, highlighted the urgency of drug development in the context of patients waiting for treatment.
“So we're going to continue to look at everything we can do to try to challenge the deeply held assumption that it does take 10 years for a new drug to come to market,” Makary said.
“We have to be able to ask big questions we've never asked before ... because I want to see American drug developers thrive,” the agency head said. “I want to see companies that do business in the United States thrive, and our job at the FDA is to be able to provide an expeditious review of data submitted to make determinations about what's safe and effective when it comes to medical products.”