As the newly appointed commissioner of the FDA, Scott Gottlieb, MD, might as well be starring alongside Tom Cruise in the latest installment of Mission Impossible. Since being named commissioner in May, Gottlieb has said he wants the FDA to speed the approval process for generic drugs, introduce competition to drive down prices, and get the opioid crisis under control.
That’s a lot more than one Mission Impossible.
While few would argue with these goals, Gottlieb’s critics worry that any movement to speed the approval process could put unsafe or unproven medications on the market. Critics also charge that Gottlieb’s ties to pharmaceutical companies are concerning. And Gottlieb himself acknowledges there’s not much the FDA can do directly about drug prices and that instead the agency needs to foster the circumstances in which companies can compete on price.
A 44-year-old survivor of non-Hodgkin’s lymphoma who once worked as a hospitalist and a clinical professor of medicine, Gottlieb has previously held several different roles in the FDA. In 2003 and 2004, he was a senior advisor to the FDA commissioner and then the FDA’s director of medical policy development. From 2005 to 2007, he served as the agency’s deputy commissioner for medical and scientific affairs. In between those jobs, he left the FDA in the spring of 2004 to help implement the Medicare Part D drug benefit.
When Republicans were out of power during the Obama administration, Gottlieb established himself as a health policy wonk while working as a resident fellow for the conservative-leaning American Enterprise Institute and as a columnist for Forbes. Bearing witness to an abiding faith in markets and competition, he talks fasts and comes across as brainy and energetic, up to the job, with a good handle on the issues, even if you disagree with him.
“While the FDA doesn’t have a direct role in drug pricing, we can take steps to help address this problem by facilitating increased competition in the market for prescription drugs through the approval of lower-cost, generic medicines,” he wrote on the FDA’s blog in June.
Last year, he expressed skepticism about candidate Trump’s endorsement of allowing drug imports as a way to reduce drug prices. In a piece for Forbes, Gottlieb said Trump’s proposal was good politics but would do little for consumers who must comply with health plan rules on where to buy and what those plans cover.
But among all of these challenges, Gottlieb’s first priority is the use and abuse of opioids. During his confirmation hearings in April, he called opioids, “a public health emergency on the order of Ebola and Zika.” In July, he followed up by saying the FDA would require manufacturers of immediate-release opioids (which are 90% of those prescribed) to give physicians and other prescribers extensive education about these medications.
On the issue of how to improve access to generics, one lever the FDA could pull is streamlining the approval process. In June, the FDA said new generic drugs would get priority reviews until at least three are available to consumers. When three generic drugs are on the market, prices tend to drop by as much as 85% off the brand-name price, the AP reported. Also, the FDA published a list of 180 drugs that have lost patent protection but have no generic competitors, saying it would be a priority to review applications for generic versions of these medications.
Making it easier for companies to get new generic medications to market would presumably head off future Martin Shkrelis and Turing Pharmaceuticals—companies that buy low-cost generics and jack up prices knowing it could take two to four years for new generic drugs to enter that same market.
Another way the FDA can stimulate competition, Gottlieb suggested, is to speed approvals of new drugs under the 21st Century Cures Act, which Congress passed last year. “Congress gave us tools to incentivize the development of novel therapies for rare diseases, and we intend to use these resources to their fullest extent,” he said in June. That same month, the agency announced it would reorganize its drug review staff to eliminate a backlog of requests for rare-disease drug designation.
While Gottlieb has said FDA regulations slow the process to approve new drugs and that such delays keep effective treatments from reaching patients, his critics see a flip side: deregulation and off-label marketing of drugs and devices adding expense and increasing the risk of adverse events.
And some see the critique of the FDA as a slow-moving, nay-saying regulator as political posturing and plain wrong.
“In reality, the FDA has become the fastest drug regulatory agency in the world, going from an average of 30 months per drug review in the 1980s to 8.5 months today,” Judith Garber and Shannon Brownlee of the Lown Institute wrote in a recent Stat commentary.
Other critics have questioned Gottlieb’s ties to the pharmaceutical industry, although Gottlieb has promised to recuse himself from decisions affecting companies to which he had connections. During his confirmation hearing, Sen. Patty Murray, a Democrat from Washington, said he had “unprecedented financial entanglements” with pharma companies.
In April, Public Citizen reported that the federal Open Payments database showed Gottlieb received $414,000 from August 2013 through December 2015 from multiple drug and medical device companies, mostly for consulting and speaking fees.
That same month, the Washington Post reported that in 2006, when Gottlieb was at the FDA, he advocated on behalf of Cephalon to increase the amount of fentanyl the manufacturer could produce. At the time, Cephalon was under federal investigation for advocating that doctors prescribe the painkiller for headaches and back pain when it was meant for patients with late-stage cancer. Fentanyl, a highly potent opioid, has led to many fatal overdoses.
So now the question is how far will Gottlieb push the agency to approve drugs faster and will his critics question his work with pharmaceutical companies as a conflict of interest.
Other questions to answer about Gottlieb include what stand he would take in the debate over whether patients have a right to try a medication before it gets FDA approval. Stat reported that Gottlieb carved out what it called a middle ground. In a letter to Murray, he wrote that he would ensure that the agency has policies in place to balance individual patients’ needs for access to investigational therapies while maintaining “a rigorous clinical trial paradigm for testing investigational products and demonstrating safety and efficacy.”
Where does he stand on the Prescription Drug User Fee Act? In the past, he said he favored allowing drug companies to seek faster approvals under PDUFA’s rules.
What’s his position on adaptive trials? In his role as deputy FDA commissioner, Gottlieb favored allowing drug companies to adjust trials as they proceeded, making them larger or smaller as needed and changing how patients get assigned if necessary. Such approaches could shorten some trials, he said. During his confirmation hearings, he reconfirmed his support for such an approach, Science magazine reported.