resident Donald Trump wants to remake the Food and Drug Administration. And he happens to have a “fantastic person” in mind to do it, he said Tuesday — someone who will turn the agency into an industry-friendly shop that cranks out new cures on the double.
But does anyone really want a deregulated FDA? STAT canvassed biopharma insiders, physicians, Wall Street analysts, and FDA veterans. Their verdict: Trump’s plan sounds like a solution in search of a problem.
Trump is weighing several candidates for FDA commissioner who would seek to radically change how the agency vets new drugs. One candidate has proposed scaling back the requirements for approval and letting doctors and patients sort out what’s worth trying. Another once claimed a “Yelp for drugs,” posting consumer reviews of medications, would better serve patients than the existing FDA. A third favors approving treatments once they’ve passed an initial safety test, regardless of whether they work.
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Such sweeping changes to the FDA’s standards could rattle the biopharma industry’s very foundation. Investors rely on the agency as a watchdog to keep companies honest. Doctors trust it to safeguard their patients from pharmaceutical snake oil. And, much as they grumble about regulation, even drug manufacturers count on the FDA to maintain a high standard, protecting their profits by blocking rivals whose products aren’t up to par.
Strip all that away and the business of making, prescribing, and profiting from new drugs could be plunged into uncertainty.
“It would be chaotic,” said Dr. David Knopman, a Mayo Clinic neurologist. Speeding up the development of powerful new treatments is a laudable goal, he said, but exposing patients to costly, ineffective, and potentially dangerous drugs could be disastrous.
“It would be a terrible thing for our patients and their families,” Knopman said. “There is no intermediate position.”
“It would be a terrible thing for our patients and their families. There is no intermediate position.”
Dr. David Knopman, Mayo Clinic
In his meeting with pharma executives Tuesday, Trump pledged not just to clear away regulations but to support fledgling startups seeking to get drugs approved.
Yet it’s not clear his reforms would actually benefit small companies.
In a world where the FDA approved medications on scant data, pharma’s wealthiest giants could litter the market with dubious new drugs — and flood the airwaves with ads touting them. That would crowd out upstart competitors and kill a whole industry’s incentive to try new things.
Dr. Leonard Schleifer, the billionaire CEO of biotech success story Regeneron Pharmaceuticals, said his company would never be able to compete with the Pfizers and Mercks of the world without a strong FDA to reward its endeavors.
“I actually am against making it really easy to get your drug approved,” Schleifer said at the Forbes Healthcare Summit in December. “… Having a high bar is a good thing in my opinion, because it allows innovators to compete.”
A rush of new, questionably effective treatments could also burden taxpayers. Under current law, Medicare and Medicaid are required to cover most FDA-approved drugs. If those drugs turned out to be duds, the entitlement programs would be shelling out for placebos.
The same dynamic could play out on Wall Street, where investors place their bets on biotech companies knowing that an FDA moment of truth awaits every promising drug. Eroding that standard could open cracks for less-than-honest CEOs to shade their data and exaggerate their product’s benefit, all to the detriment of the market.
“There are just too many companies I’ve seen that have tried to pull fast ones on the FDA and on investors,” said Brian Skorney, a biotech equities analyst at Baird. “With lower standards, you just know they’re going to try to sucker patients and physicians.”
“I actually am against making it really easy to get your drug approved.”
Dr. Leonard Schleifer, CEO of Regeneron
Dr. Michael Carome of the watchdog group Public Citizen believes the FDA has already conceded too much to the industry it regulates. The bipartisan 21st Century Cures Act, signed by former President Barack Obama last year, requires the FDA to shorten the review process for many new medicines. But the agency has already sped up its reviews; it’s approving new therapies at a record pace.
The standard review process now takes a median of 10 months, down from nearly 13 months in 2005, according to the FDA’s latest progress report. Companies can shave off another two months if they get a priority review. In fiscal year 2014, the most recent data available, 92 percent of drugs were approved on first pass, up from 60 percent in 2005.
“My general perspective is that industry is overall been happy with the pace” of FDA approvals, said Darshan Kulkarni, an attorney who focuses on FDA law. “I think there’d be more, for lack of a better term, quacks coming onto the market if there were less regulation.”
Calls to make things move even faster reflect a fundamental misunderstanding of what the agency actually does, according to former FDA medical officer Dr. David Gortler.
“Maybe 2 percent of the people on the whole planet get what we do,” said Gortler, who is now a consultant at Former FDA. “As a conservative, I’m all for less regulation, but I’m not prepared to let Jane and John Q Public make decisions about pharmacogenomics and pharmacokinetics on their own.”
The FDA itself has refrained from commenting on any speculation about its future. But last month, amid escalating talk of rolling back agency regulations, the agency released a report detailing 22 cases in which an experimental drug looked promising or even lifesaving in early clinical trials, only to prove useless at best — and deadly at worst — upon further study.
Under more lax regulations, each of those treatments could have been approved, wasting money and potentially costing patient lives.
Despite such cautionary tales, Trump is steadfast that his administration can accelerate the FDA without putting patients at risk.
“We’re going to be cutting regulations at a level that nobody’s ever seen before. … And we’re going to have tremendous protection for the people — maybe more protection for the people,” Trump said Tuesday.
“You people are going to do great,” he told the biopharma executives gathered at the White House. “You’re going to do great.”