Speeding Up Approvals, Getting More Drugs OTC Among FDA’s Top Priorities

The FDA is continuing its quest to speed up drug approvals and make more drugs available over the counter (OTC), FDA Commissioner Marty Makary, MD, MPH, said Wednesday.

“We want to challenge the assumption that it takes 10 to 12 years for a drug to come to market,” Makary said during a press conference with reporters. “We believe it can be done faster without cutting any corners on safety. We’d like to modernize the agency with technology, while maintaining our gold-standard thresholds for approving drugs, devices, food, cosmetics, and tobacco.”

He noted that the FDA approved 67 medications last year, more than the 5-year average (65), the 10-year average (64), and the 15-year average (60). “One of our big priorities at the agency is to reduce the idle time, use technology, and prioritize drug applications,” he said, touting the pilot National Priority Voucher Program, which addresses those issues.

“We are giving vouchers to drugs that can meet an unmet public health need, companies that are making steps towards affordability — to summarize, I would say drugs that are game-changing drugs,” Makary said.

One drug being evaluated for possible approval under the program is a gene therapy known as DB-OTO for a congenital form of deafness. “When you see an amazing set of results, as was reported in the New England Journal of Medicine, that is the type of game-changing finding that we want to see delivered to patients rapidly, without cutting any corners on safety,” he noted.

In line with the FDA’s goal of speeding up approvals, Makary said the agency was “100% compliant” with its Prescription Drug User Fee Act (PDUFA) deadlines. However, when asked by MedPage Today about a couple of PDUFA deadlines the agency had missed last year — a late April deadline for elamipretide (Forzinity) for Barth syndrome and a December deadline for mitapivat (Aqvesme) for thalassemia-related anemia, Makary said that those drug approval processes started before he arrived at the agency.

“You are correct; PDUFA dates were missed last year,” he added. “We achieved our goal of hitting 100% compliance with PDUFA in late fall of last year or so, and we’re still there … It is not an absolute that we have to be at 100%, but in the last several months, we’ve been at 100%.” (Editor’s note: Makary is the former medical editor-in-chief of MedPage Today.)

Regarding OTC drugs, “when I look at the reasons those drugs were not approved in the past, I think we can do better,” he said. For instance, “every single pill that is approved to be over the counter by the agency is required to have a study showing that people can read the instructions and take the pill … It’s just not common sense. So we want to reduce the requirements that are onerous that don’t make sense, and focus instead on safety and labeling.”

Putting more drugs over the counter “enables better access to medication, can reduce unnecessary healthcare utilization like waiting in an urgent care facility or emergency department to get [a refill],” and it encourages price competition, Makary explained. “It also enables people to buy drugs without going through the shell games of a PBM [pharmacy benefit manager].”

Vaginal estrogen is one drug Makary said he’d like to see go OTC. “There has been tremendous demand for [different types of] hormone replacement therapy for postmenopausal women; we’ve been tracking it closely.”

Institutional review boards (IRBs) that approve clinical trials at universities are another FDA focus, according to Makary. Sometimes the IRBs — which are usually staffed by volunteers — only meet monthly, “and if you think about it, our entire U.S. research infrastructure is dependent on this sometimes rate-limiting step of this committee meeting,” he said. “We can do better. We are seeing IRBs approved in shorter periods of time in Australia and other places.”

The IRB process needs to be re-evaluated, he added. “When I was at Johns Hopkins, it took a year and a half for one IRB proposal to get approved, and in the end, it was not even approved — and it was a survey. So we have to be more competitive and get decisions out.”

One idea for improving IRB response times is to pay the members who are reviewing potential studies, instead of relying on a voluntary committee, Makary suggested.

Competing with China for drug trials is another concern of Makary’s. “China, in the last several years, surpassed the United States and the number of phase I trials being conducted,” he said. “And when we look at initiation of phase I trials, [in 2024] they were at least four times more than the United States. … We have to be smart, and we have to think about how we can maintain our high standards for safety and yet be more competitive. We are looking at re-engineering the entire drug approval process from A to Z.”

Makary was also asked about the changes initiated by the Department of Government Efficiency, a non-governmental body headed up by tech entrepreneur Elon Musk. “The cuts that made the most sense were cuts that involved consolidation, because each of six major agencies or centers within the agency had duplicative services,” he said. For instance, they would each have their own teams to contract software licenses, “and they’d be buying the same software multiple times … So we have had success with shared services, with procurement, with centralizing a lot of it.”

On the other hand, he added, “I would not have offered early retirement to the scientists at the FDA. We have scientists who left with that package, but we are 1775084304 hiring scientists.”