When chemist Harvey Washington Wiley took the helm of the agency that would become the U.S. Food and Drug Administration more than a century ago, his mandate was narrow: making sure the label on a bottle or can matched the food or drug inside. Concerns about the safety of the contents were not addressed until 1938, and companies hawking their wares did not have to prove they were actually effective until 1962.
Scott Gottlieb, President Donald Trump’s nominee for FDA commissioner, has strong conservative credentials and a close relationship with the pharmaceutical industry. But there is no sign that Gottlieb, a doctor, former deputy commissioner at the agency and a partner at a large venture capital fund, truly hopes to return the FDA to the norms of half a century ago—an approach floated by Jim O’Neill, who had earlier been seen as Trump’s other top candidate for the position.
But short of changes on the level of attempting to cut the product efficacy requirement, the FDA’s new chief will still be able to leave an indelible mark. The behemoth agency’s more than 14,000 employees are tasked with responsibilities as varied as approving drugs, ensuring food imports are safe, combating antibiotic resistance and regulating tobacco products. Its reach is domestic and global. And to tackle its agenda, the Obama administration has requested a budget of $5.1 billion for fiscal 2017. Donald Trump’s 100-day action plan to “make America great again” offers some early hints about his plans for the agency. The document, which the president says is a contract with American voters, states, “Reforms will also include cutting the red tape at the FDA: there are over 4,000 drugs awaiting approval, and we especially want to speed the approval of life-saving medications.” Pres. Trump named Gottlieb as his nominee for the post on Friday.
If the Senate approves Gottlieb as FDA commissioner, big-ticket items before him will include hastening those approvals, as well as shaping the future of clinical trial requirements. Other issues will include considering when terminal patients should have access to investigational drugs, the agency’s control over off-label drug uses, and the changing landscape of genetic tests, to name a few.
Gottlieb already has significant ties to the FDA and other federal agencies. He is currently a resident fellow at the conservative American Enterprise Institute, where he analyzes health policy, regulation and public health. He is on the Federal Health IT Policy Committee, a group that advises the Department of Health and Human Services on health care IT issues. His resume also boasts stints as the FDA’s deputy commissioner, and at the Centers for Medicare and Medicaid Services as a senior official focused on new medical technologies.
Gottlieb has called for a faster approvals process for new drugs. Yet he has taken a different tack than suggesting that the efficacy requirement be scrapped. Instead he has focused on ways to move away from waiting for large, clinical trial results. Writing in Massachusetts newspaper South Coast Today last year, Gottlieb praised the 21st Century Cures Act (which was signed into law in December 2016), and said that it would help smooth the way for quicker approvals by zeroing in on results from small trials and interim study results instead of waiting for more traditional clinical trial findings. "These are interim endpoints that can be used to more quickly gauge a medicine's benefit," he wrote.
Further insights about his views come from an article he penned in 2012 for National Affairs,in which he criticized the FDA for setting up too many regulatory hurdles. There, too, he argued that extensive clinical trial requirements may have a chilling effect on bringing new therapies to market. “These demands also further entrench established players, and the limited competition from new market entrants means that existing drugs remain hideously expensive,” he wrote. He blamed such requirements on an institutional culture that he said values “an excessive desire for certainty,” and does not trust the doctors who do the prescribing.
He called for a fundamental change to that culture, whereby FDA employees who review the science involved in drug evaluations would delegate final approval decisions to another part of the agency or an outside group—so that the same individuals are not acting as both judge and jury. Gottlieb also suggested that final approval decisions be made by a committee composed of the agency’s most senior scientists, who he believes might be more comfortable with uncertainty than junior scientists.
The head of the FDA is not involved in all the minutiae that come up—that’s what the agency’s division directors do, says David Gortler, who was a senior medical analyst at the FDA from 2007 to 2012 and is now a drug safety expert with FormerFDA.com, a website that connects past FDA employees with pharmaceutical companies or individuals seeking advice or services. The commissioner is intended to act as a check on the system, Gortler explains. “If a poor regulatory decision is made by some high-ranking person, the commissioner would have to deal with that directly,” Gortler explains. Michael Carome, who heads the Health Research Group at a liberal nonprofit organization called Public Citizen, says having a doctor or someone with a strong science foundational is critical because the FDA is a science-based organization. “The commissioner can help guide the policies and how the agency approaches its review and oversight activities,” he says. “The commissioner can work with Congress to help formulate legislation that can change the procedures and policies of the agency
Perhaps the first indications of the FDA’s future under a Trump administration will emerge from how Congress acts on a pair of bills already under the early stages of review. If passed they would renew the agency’s current ability to charge pharmaceutical companies and medical device makers a fee for approvals reviews—and the FDA would set those prices. These bills come up for renewal every five years and are largely supported by lawmakers and the pharmaceutical industry, so the renewals provide opportunities for other provisions to be slipped in—including ones that may loosen rules in other areas related to medical regulation, Carome says. That reality will make those bills an attractive vehicle for lobbyists hoping to loosen other regulatory requirements, he says. “I think there is little doubt that Trump administration will bring a deregulatory agenda,” he adds.
Other early tests will come from how the Trump administration and the Republican-led Congress act on drug pricing issues. The president has said that allowing more drug imports would be a key approach to lowering prices, and increasing those imports would require legislative action. That, too, may be something that could be tacked onto other legislation in the next session of Congress.
The prospects for the nominee’s quick confirmation remain unclear. Former FDA head Robert Califf, a cardiologist with close ties to the pharmaceutical industry, was only confirmed in February 2016 after Senators—mostly Democrats—blocked the appointment for weeks and used Califf’s nomination hearings as an opportunity to sound off about their disapproval with various FDA actions including its clearance of various painkillers, which lawmakers said helped to fuel the opioid epidemic. Califf stepped down when Trump was sworn in.
Editor’s Note (3/13/17): This story was updated to reflect that the White House announced Gottlieb as the nominee. When the article was first published his nomination was not yet confirmed.