Makary vows FDA will uphold 'gold standard of trusted science' during Senate hearing

Key takeaways:

• Martin Makary, MD, MPH, has been nominated to lead the FDA.
• Discussion about the recently canceled meeting to select the components for next year’s flu vaccine was a major theme in the hearing.

Lawmakers grilled Martin Makary, MD, MPH, on transparency, his plans for mifepristone and his stance on the recently cancelled Vaccine Advisory Committee meeting during Thursday’s Senate hearing to be the next FDA commissioner.

Makary, a surgeon and public policy researcher at Johns Hopkins University, has been nominated to be the agency’s next leader under the administration of President Donald J. Trump. During the hearing, he told members of the U.S. Senate Committee on Health, Education, Labor and Pensions that he is a scientist who values good data and common sense.

“The FDA has a long history of using gold standard science to uphold public safety and help us as clinicians take care of patients when they come to us for help,” he said. “I hope to ensure the FDA holds to the gold standard of trusted science, transparency and common sense to rebuild public trust and make America healthy again.”

Key moments

A major focus of the hearing centered on the cancelled Vaccines and Related Biological Products Advisory Committee meeting. Senators from both sides of the aisle expressed concern that the FDA recently canceled a meeting to decide the composition of next season’s influenza vaccines. Committee Chairman Bill Cassidy, MD, a Republican from Louisiana, said that transparency was lost with the cancellation, as “the open meeting allows a postmortem, if you will” — an evaluation of “what went right, what went wrong and how to make it better.” He said the move seems to reverse HHS Secretary Robert F. Kennedy Jr.’s promise of radical transparency in health care. Makary stressed that he was not involved in the decision to cancel the meeting. Ultimately, he said that, if confirmed, he would convene the vaccine advisory committee, but evaded committing to rescheduling the previously canceled meeting.

Another major talking point concerned mifepristone’s safety and dispensing requirements. Tammy Baldwin, a Democrat from Wisconsin, asked if he would maintain current access to mifepristone, and Makary responded that his “position is that of a scientist,” and that he couldn’t judge data without seeing it.

“If that's your approach to something that has been approved for, now, decades, are you going to do the same with Tylenol? There are a lot of side effects for daily use, including liver damage,” Baldwin asked. “Are you going to do ongoing surveillance of the possible side effects for that drug that was approved many years ago? How about asthma inhalers?” Makary replied that he would follow the law, and an ongoing review of data is required for mifepristone as part of the Risk Evaluation and Mitigation Strategy.

Cassidy asked if Makary would reinstate the in-person dispensing requirement that was eliminated during the previous administration.

“I have no preconceived plans on mifepristone policy except to take a solid, hard look at the data and to meet with the professional career scientists who have reviewed the data at the FDA and to build an expert coalition to review the ongoing data,” Makary replied.

Maggie Hassan, a Democrat from New Hampshire, was one of several committee members who asked about prescription drug prices “once again skyrocketing.” She has been working with Rand Paul, a Republican senator from Kentucky, on a bill that would allow the FDA to communicate more transparently with manufacturers of generic drugs “in order to make more low-cost prescription drugs available for patients.”

When asked if he would work with her to speed up generic drug approval at the FDA in an effort to lower drug costs, Makary gave “an emphatic yes.”

Susan Collins, a Republican from Maine, suggested opening up the market to allow more competition from biosimilar and generic drugs as a way to lower prescription costs. When asked if he supported the concept of an expedited pathway for biosimilars to compete, Makary said he is “a big believer that we need to figure out a way to get biosimilars approved quicker without cutting corners on the scientific review,” and that is “one of several strategies” the FDA can explore in its effort to lower costs, along with moving some medications to over the counter rather than prescription only.

Lisa Blunt Rochester, a Democrat from Delaware, also asked about combatting misinformation. “How would you use your position as FDA commissioner to compete with uninformed voices and to share evidence-based, consensus-driven information that is free from political influence?” she said.

“I believe very much in empowering people with information,” Makary replied. “I do think you can explain to people with humility what we know and what we don't know, and then they're going to trust us more. Trust in doctors and hospitals is at a crisis right now.”

What’s next?

If confirmed, Makary would report to Kennedy.

Nominees need only a simple majority to be approved by the committee or confirmed by the Senate. Notably, republicans have a one-seat majority on the committee and control the Senate.

“If confirmed, my goals are very clear at the FDA: more cures and meaningful treatments for Americans, including diagnostics; healthy food for children and rebuilding the public’s trust,” Makary said.

Cassidy gave senators until 5 p.m. ET Friday to submit additional questions for the record.

Diagnosis

Makary appeared “well-qualified and insisted on being guided by the science and in listening to experienced FDA staff,” according to Arthur L. Caplan, PhD, a professor and founding head of the Division of Medical Ethics at NYU Grossman School of Medicine.

“I think his hearing gave me some hope,” he told Healio.

“Revisiting mifepristone is not necessary, but politics is in play on that matter. Similarly, I hope he will not waste too much time on finding dangers in food — we need to act on those we already know about, like microplastics,” Caplan added. “I think the big challenge will be working with a boss — RFK Jr. — who, so far, has shown little appreciation for science or established public health knowledge.”