Gottlieb’s experience with the White House when serving as a senior FDA and US Centers for Medicare and Medicaid Services official during the George W. Bush administration was quite different from the Trump presidency. Under Bush, everything had to go through the Office of Management and Budget and the White House “exerted a lot of control,” forcing the FDA to operate differently, he said.

Compromise At 90%

Gottlieb’s independence from the White House during his time as commissioner under Trump likely is a key reason why he was able to rack up a long list of policy accomplishments during his relatively short leadership stint, while also insulating the agency from some the more disruptive fates other government agencies experienced during Trump’s presidency. (Also see “Gottlieb’s Greatest Strength As US FDA Commissioner? Stability” – Pink Sheet, 27 Mar, 2019.)

Gottlieb credited many of his policy wins to his willingness to accept 90% of what he wanted when pushing for new regulatory reforms or paradigms at the FDA.

“If I can get 90% of the regime implemented, I’ll leave that last 10% on the table if I think it’s going to imperil the whole regime,” he said.

Stem cell therapy reform was an area cited as an example of where the strategy worked. Gottlieb said there was a recognition that the agency needed to focus on the most high-risk bad actors who were providing stem cell treatments without FDA approval because if the agency tried to take on every bad actor it would not be able to get the rest of the reform regime implemented.

Taking enforcement discretion against lower-risk actors to focus on people “who are doing things that are literally killing patients,” was a “hard thing to swallow at the time for the agency,” Gottlieb said. “But by doing that we were able to implement the regime successfully and get it sustained in court and get it implemented, get the guidance out. And I do think that if we had gone after everything and not been willing to exercise enforcement discretion in that one realm you would [have] had Capitol Hill stepping in to block what we did.” (Also see “Unapproved Cell/Tissue Product Enforcement Discretion Ends; How Hard Will US FDA Crack Down?” – Pink Sheet, 28 May, 2021.)

New Legislation Needed for N of 1

When it comes to the challenges facing the drug industry and FDA today, one area where Gottlieb thinks legislation likely is needed is to “enable a robust pathway,” in the world of “N of 1” products. (Also see “Antisense Oligonucleotide Clinical Guidance Emphasizes Individualized Nature Of Treatment” – Pink Sheet, 8 Dec, 2021.)

“I don’t know that the existing authorities give the agency sufficient flexibility,” he said. “And I don’t know if the agency is going to be aggressive enough in taking advantage of those at least on the drug side of the house. I think you might have more flexibility if this was sort of a device construct. I don’t know if you’ll get it on the drug side of the house absent legislation.”

How To Talk About Emerging Issues

Gottlieb also gave advice that might be helpful to current Commissioner Robert Califf, who continues to make tackling misinformation a key part of his leadership agenda.  (Also see “Califf’s Top Priorities For New Year: Misinformation And Evidence Generation” – Pink Sheet, 1 Feb, 2024.)

Gottlieb said the FDA’s experience talking about emerging drug safety issues could be used as a guide for public health officials trying to discuss evolving science with the public.

In the early 2000s, the FDA was initially very reluctant to talk about emerging drug safety issues, he said.

“There was a view that if you go out too early and talk about what you’re concerned about, you’re going to scare the public and people are going to come off therapy and not seek out therapy,” Gottlieb said. “And what we recognized was no, we needed to talk about these things early, tell people what we were looking at, but reassure them that we’re going to get to the bottom of it. And as long as we reassure them that we’re gonna get to the bottom of it and put it in a proper context, the public actually won’t do the wrong thing. They’ll get it. And there was a way to communicate risk information, where you could both inform the public and not drive bad decision making.”

Contrast this with COVID-19, where there has been a tendency for public health officials to “exude much more certainty,” than the science could support over concerns of how the public would handle the information if it wasn’t presented with such certainty, Gottlieb said.

“I think there was a way to promulgate some of the uncertainty around some of the things we were asking the public to do and still get a lot of people to agree to the behavior collectively,” he said.

This also means speedily updating the public when the information base changes, Gottlieb added.

Not In On Mifepristone Brief

Gottlieb has been a vocal public figure on the FDA and other pharma-related topics since leaving the agency, perhaps the most public of any former FDA commissioner. But one area where he has been a bit quieter than his peers is on legal challenges to the FDA’s approval of the abortion pill mifepristone. (Also see “Supreme Court Could Curtail Mifepristone Access, Lay Out ‘Road Map’ To Challenge FDA Approvals” – Pink Sheet, 13 Dec, 2023.)

Seven former confirmed or acting FDA commissioners filed an amicus brief in the pending Supreme Court case 1 February. The dispute, which is set to be heard by the Supreme Court on 26 March, has raised broad concerns from those in the biopharma space due to fears it could set a broader precedent around the ability of courts to overrule the FDA’s scientific judgement. (Also see “Mifepristone Ruling Casts ‘Shadow Of Uncertainty’ Over Every FDA Approval, Biopharma Industry Tells Supreme Court” – Pink Sheet, 12 Oct, 2023.)

The brief says that there could be “catastrophic consequences to public health,” if the US Court of Appeals for the Fifth Circuit’s decision, which would significantly curtail access to the abortion pill, is upheld.

“This unprecedented decision turns Congress’s desired regulatory scheme on its head and opens the door to constant legal challenges of drug approval decisions,” the brief says. “If permitted to stand, the Fifth Circuit’s approach would allow courts to substitute their lay analysis for FDA’s scientific expertise and to overturn the agency’s approval and conditions of use for drugs, even after they have been on the market for decades. The resulting uncertainty would threaten the incentives for drug companies to undertake the time consuming and costly investment required to develop new drugs and ultimately hinder patients’ access to critical remedies that prevent suffering and save lives.”

Trump’s other FDA commissioner, Stephen Hahn, is not a party to the amicus brief either.

Gottlieb was not asked about the case at the Friends of Cancer Research event.