Does science require strict adherence to established drug-approval processes?
DOI: 10.1063/PT.5.8215
As debates about the March for Science proceed and the climate wars rage, another drama at the intersection of science and politics is playing out. The Food and Drug Administration, “the nation’s most ubiquitous regulatory agency ,” uses science in regulating drugs and other products accounting for roughly a quarter of consumer spending. Pending in the Senate is President Trump’s nomination of Scott Gottlieb to head the FDA. Media discussion about the nomination doesn’t address physics, but does merit attention from anyone interested in the intersection of science, government, and commerce.
Five years ago in a long National Affairs essay headlined “Changing the FDA’s culture,” Gottlieb telegraphed his vision. The article’s byline identified him as a practicing physician and resident fellow at the American Enterprise Institute who served from 2005 to 2007 as FDA deputy commissioner for medical and scientific affairs. The article’s opening identified him as focused on the human consequences of FDA actions.
Researchers at FDA’s Life Sciences Laboratory in Maryland evaluate drugs and medical devices.
FDA
“Hunter syndrome,” the essay began, “is a terrible disease that cripples, and often kills, children.” Parents had to “watch as the disease destroyed their children.” Yet when research began showing promise, what these families encountered was, Gottlieb charged, “a broken and dysfunctional approach to drug trials, driven by an FDA culture poorly suited to serving the needs of the sickest patients.”
Gottlieb perceived an indefensible excess of rigidity concerning clinical-trial methods in this case and others. He declared it the “product of a poorly understood, but now well-established, attitude within the agency: an excessive desire for certainty.” He saw this desire as primarily driven by “a deepening mistrust of the doctors who eventually prescribe such medicines and the companies that market them”—a mistrust “impeding the availability of safe, effective drugs that could today be helping real patients.”
But wait. What about the example of Frances Kelsey, the FDA stickler and stalwart who, a half century ago, famously saved American babies from the unforeseen birth-defect horrors of insufficiently examined thalidomide? In going by the book, she had made sure their mothers couldn’t be prescribed it. Gottlieb cited Kelsey’s example himself, but proposed that such “devotion comes with some downsides.” By “so heavily prioritizing one of its obligations—the protection of consumers—the FDA has sometimes subordinated and neglected its other key obligation, which is to guide new medical innovations to market.” “FDA reviewers,” he charged, “believe it is appropriate to prioritize safety over speed.”
Gottlieb’s 2012 essay went on to discuss “a few key structural reforms” for changing FDA culture. This year the essay has also contributed to opposition to his nomination.
An article at STAT, which calls itself “a national publication” for “compelling stories about health, medicine, and scientific discovery,” joined many others in naming some of Gottlieb’s pharmaceutical industry ties, which are a main source of objection to his candidacy:
Financial disclosure reports show that Gottlieb has helped launch at least 12 biomedical companies through his work as a managing director in the investment bank T.R. Winston & Co. and his role as a venture partner at New Enterprise Associates.
He also has been a board member, adviser, or speaker for a number of big pharma companies, among them: GlaxoSmithKline, Bristol-Myers Squibb, Daiichi Sankyo, Molecular Insight, and Vertex Pharmaceuticals.
And records show that pharmaceutical companies paid Gottlieb more than $400,000 between 2013 and 2015.
In a press release , Public Citizen called for Gottlieb’s rejection because of “an unprecedented web”of such ties and because of “dangerous deregulatory ideas.” After a New England Journal of Medicine article by Harvard’s Daniel Carpenter offered widely cited objections to the nomination, CNN quoted Carpenter: “I’m concerned that the scientific basis of drug testing and review is going to be undermined, whether by Gottlieb or by other things the Trump administration does.” That article also quoted the view of Michael Carome, director of Public Citizen, that Gottlieb has been a “shill” for corporations and that it is “really impossible to disentangle himself from these conflicts.” It also reported a January prediction by Trump that 9000 pages of FDA regulations would be reduced to 100.
The online version of a New York Times headline warned that Gottlieb could “undo decades of drug safeguards.” The article reported that the president’s statements about the FDA have led critics to say that “the market for drugs could be destabilized and the door opened to unproven products based on junk science.” The Times quoted an industry chief executive’s worry that an FDA standards rollback would muddy public abilities to detect sham drugs. It also noted that drug executives want the FDA to fill “an estimated 1,000 staff vacancies” as a way to speed decisions without lowering or changing standards.
Media reports also show that objections to the nomination have been tempered by a realization that STAT explained this way:
When President Trump nominated Scott Gottlieb … you could almost hear the sigh of relief that rippled through the health care community.
That’s because Gottlieb isn’t Jim O’Neill, whose name had also been floated…. O’Neill has no background in health care except as a venture capitalist, and thinks that the way to speed new drugs to the market is for the FDA to drop any requirements that drug makers demonstrate that their products actually work. Gottlieb, in contrast, is an internal medicine physician and a drug company insider, who presumably knows you need at least some scientific evidence for efficacy.
The now apparently mooted O’Neill possibility, whatever it may still say about the Trump-era FDA, has been widely linked to billionaire, “libertarian iconoclast” and informal Trump adviser Peter Thiel. Vox reported his vision for “the same speedy and disruptive approach to medical regulation that Silicon Valley brought to the taxi and hotel industries” to be brought to the FDA to “unlock cures—fast.”
Among media supporters of the Gottlieb nomination, the editors of the Wall Street Journal have been energetic, sometimes sarcastic, and occasionally downright bitter.
In an editorial musing that Gottlieb might be Trump’s “most important nominee,” the WSJ editors also mused that thinking like Thiel’s might well be “far from crazy, especially for drugs that treat rare diseases when no approved options exist.” The editors nevertheless also noted the widespread relief that the president didn’t pick a candidate “who supports making drugs available to patients after testing for safety, though not for efficacy.” But the editorial emphasized skepticism about what might be excessive rigidity concerning clinical trials, asking, “Why should desperate patients have to take a sugar pill so the FDA can satisfy its demand for 100% certainty that a drug works?” It asserted that at the FDA, “a culture of control strangles innovation,” that an “iron triangle of interest groups, the bureaucracy and the press will resist change,” but that “Dr. Gottlieb could save lives by renovating FDA’s drug-approval processes.”
In a sarcastic 6 April piece , the WSJ editors used disdain-conveying quotation marks to disparage the main criticism against the nominee. Democrats criticize him, they wrote, “for the ‘conflict of interest’ of knowing too much about the industries he’d regulate.” (In an earlier editorial, they had quipped, “In other words, he’s disqualified because he’s qualified.”) These are the same Democrats, the editors observed, who “pummeled” Education secretary nominee Betsy DeVos “for not having enough experience in public education.”
A week later, the WSJ editors selected, and printed alone, a particularly bitter letter from Richard E. Ralston, executive director of Americans for Free Choice in Medicine. He asserted that Gottlieb threatens both power-hungry bureaucrats who seek “to make sure that no patient obtains effective treatment or survives illness without government permission” and ideologues who would rather see “patients suffer and die than to allow a profit for those who might save them.” Not much room for the inspiring example of Frances Kelsey in those accusations.
More than once the WSJ editors have revealed their own bitterness about the FDA. In a February editorial endorsing right-to-try legislation to allow terminal patients to seek treatments that haven’t received FDA approval, they aggressively mocked the worry that right-to-try might hamper recruitment for double-blind placebo trials. The editors wrote, “For the uninitiated, that is the human experiment during which the FDA intentionally denies patients treatment for the profound insight that terminal patients die. The FDA continues to rely on this method even though Congress has directed the agency to incorporate patient-reported outcomes and other evidence.” The editors condemned the FDA as a “bureaucracy that for too long has enjoyed power over how quickly a cancer patient dies.”
Meanwhile the candidate declares steadfastly that he will be guided by science. And a Nature editorial , though emphatic about the conflict-of-interest worry, has acknowledged the value of his experience, praised his tendency “to back up his arguments with data,” and judged that he “may well prove to be an effective FDA commissioner.”
Steven T. Corneliussen is Physics Today‘s media analyst. He has published op-eds in the Washington Post and other newspapers, has written for NASA’s history program, and was a science writer at a particle-accelerator laboratory.