Scott Gottlieb has submitted his resignation as commissioner of the Food and Drug Administration, after nearly two years at the helm of the agency. Gottlieb plans to leave next month.
The unexpected exit has created a number of uncertainties for the pharmaceutical industry. More clarity should come in the lead up to the leadership change. In the meantime, here are five key takeaways from Gottlieb's departure and time at the FDA.
1. Who will be Gottlieb's successor?
It's still up in the air. Gottlieb is set to step down in one month, at which point Amy Abernethy, who was recently appointed principal deputy commissioner of the FDA, could presumably helm the agency on an interim basis until HHS finds a replacement. Abernethy came from Roche's Flatiron unit.
There may already be a short list forming in that search. The Wall Street Journal reports Norman Sharpless, director of the National Cancer Institute, and Brett Giroir, assistant secretary at the Department of Health and Human Services, are already viewed as possible successors.
A spokesperson for the National Cancer Institute said in a statement to BioPharma Dive that the institute is not involved in decisions regarding the FDA Commissioner.
Bethany Hills, chair of FDA practice at law firm Mintz Levin, said in an interview with BioPharma Dive that Abernethy will likely serve as interim commissioner, at least early on. Hills noted that FDA commissioners tend to have a clinical background and a well-known presence in their research field. Experience dealing with administrative agencies or leading large institutional structures are also advantages for any potential successor, she said.
Gottlieb's quick departure suggests that whoever takes on his role will need to hit the ground running. While the FDA is riding high on some victories, such as clearing a record number of generics for market, the agency has also spurred some concern about its oversight of drug quality. Questions also persist over whether the FDA's speed in clearing novel drugs has weakened its historical standards on safety and efficacy.
A replacement will need to navigate a split Congress where Democrats now control the House of Representatives. Gottlieb was able to gain support on both sides of the aisle, a "rare" feat for an official in Donald Trump's administration, according to Cowen & Co. analyst Rick Weissenstein.
"Gottlieb has built a team of senior staff with solid GOP bona fides ... to help him stay on top of politically sensitive topics and to manage FDA's public agenda," Weissenstein wrote in a March 5 note.
Should Abernethy get the top spot at the FDA, she "will need to build a similar support network — either with the same group or new faces — that can help in the new political climate with Democratic oversight," he added. "Her industry background will not be viewed as an asset in that context."
2. Why is he leaving the FDA?
Although it is a common refrain amid many departing government officials, according to Gottlieb, it's a family matter.
"There's perhaps nothing that could pull me away from this role other than the challenge of being apart from my family for these past two years and missing my wife and three children," he wrote in a letter to FDA staff.
Gottlieb, 46, is husband to Allyson Nemeroff and father to three daughters. Their home is in Connecticut. Gottlieb noted to The Washington Post that his commute was crimping the amount of time he spent with his family. "I get home late Friday, work on weekends and come back to Washington on Sunday," he said in an interview with the paper.
3. What work does Gottlieb leave unfinished?
The FDA had its hands full while under Gottlieb.
A national opioid epidemic pushed the agency to call for new and safer pain medications. A growing outcry about the cost of prescription medications — a problem over which the FDA does not have direct control — fueled the agency's focus on increasing market competition through generic and biosimilar approvals. And an accelerated use of e-cigarettes among teens caused regulators to take compliance actions targeting the way such products are marketed and sold.
These efforts were happening as the FDA dished out guidance on other areas of drug development, including cell and gene therapy, combination products and continuous manufacturing. Those topics look set to grow in importance, particularly as more DNA-based treatments arrive before the FDA.
"To the extent that he has recognized this trend early and convened workshops and panels for industry, Dr. Gottlieb has done a good job of showing FDA is forward-thinking," said Brad Loncar, a biotech investor and founder of two exchange-traded funds. "It will be important for the next FDA commissioner to also show that the agency will be a partner in the development of these new technologies as well."
According to Hills, the agency will surely continue to restructure how it regulates these areas to make the process more effective and transparent.
She suspects opioids will remain a priority, though the focus on e-cigarettes may die down somewhat — perhaps because of a "difference of opinion" between the FDA and that industry.
4. How will the industry react to Gottlieb's departure?
Gottlieb will leave the FDA with widespread backing from drugmakers.
"He's making it easier for companies to understand the transparency in the policy," Hills said.
Trade groups representing the pharmaceutical, biotechnology and generics industries commended the agency head for his leadership, which many argue helped streamline and invigorate the drug approval process. "[T]he generic and biosimilars industry will miss this true champion of patient access," said the Association for Accessible Medicines in a statement.
Novartis CEO Vas Narasimhan and Alnylam Pharmaceuticals head John Maraganore were among the biopharma executives to personally express gratitude.
Thank you @SGottliebFDA, we are grateful for your support to ensure the US continued to foster breakthrough medical innovation to extend and enhance people’s lives, reduce healthcare costs and improve public health. All the best for the future. https://t.co/GIbry4H2OI— Vas Narasimhan (@VasNarasimhan) March 6, 2019
Gottlieb also received plaudits from industry critics. David Mitchell of Patients for Affordable Drugs and Walid Gellad of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh separately noted to BioPharma Dive that Gottlieb's departure is a loss — particularly in the fight to lower drug prices.
"He was willing to take on tough issues head-on and try to make a difference, and not everybody does that," Mitchell said in an interview.
"This is a man who worked hard to ensure the FDA was doing everything in his power, within the scope of its jurisdiction, it could do to lower drug prices," he added. "Whether it was speeding more generics to market or listing the drug companies who have received complaint letters for not making samples available, or whether it was flagging the problem with sham citizen petitions."
Investors signaled disappointment as well. The Nasdaq biotech index was down more than 2% Tuesday morning.
5. What will Gottlieb's legacy be?
Gottlieb will likely be remembered for his balancing act, keeping the FDA's core mission at the forefront even as he worked closely with industry to streamline clinical and approval pathways. Outside FDA's official mandate, he also took an active role in speaking about drug pricing, at times blasting both pharmas and generic companies for gaming the system.
He'll be too known for his transparency efforts, having upended agency communications with his steady stream of releases and Twitter omnipresence.
"One thing that is really distinguishing factor of Scott was he seemed to have unbounded energy. I mean, we get a press release every day out of the FDA, which is crazy," Hills of Mintz Levin said. "I would expect we may not have another superhuman quite like him."
While generally considered positive, Gottlieb's impact on the FDA does raise some issues. The volume of new drug approvals, as well as the hastened speed at which those therapies are coming to market, has been met with concern about data requirements.
Ned Pagliarulo contributed reporting.