Despite his relatively short tenure, outgoing Food and Drug Administration Commissioner Scott Gottlieb left a big imprint on the agency. In particular, he leaves behind what may be one of the most forward-thinking legacies of any FDA commissioner in recent memory. As such, he may be a hard act to follow, but experts chimed in about some attributes they would like to see.
On Tuesday, Gottlieb resigned from the agency, having served as commissioner since May 2017. He reportedly wanted to spend more time with his family given a weekly commute to Washington from his home in Westport, Connecticut. Gottlieb is expected to step down in about one month. The Wall Street Journal reported that National Cancer Institute Director Norman Sharpless and Department of Health and Human Services Assistant Secretary Brett Giroir are possible successors, with the latter favored by HHS Secretary Alex Azar and Gottlieb favoring the former. Amy Abernethy, who was named deputy commissioner in December, could serve as interim commissioner.
One of the most notable attributes of Gottlieb’s leadership of the FDA has been his embrace of new technologies, in drugs and elsewhere. With him at the helm, the agency approved the first CAR-T cell therapies – Novartis’s Kymriah (tisagenlecleucel) and Gilead Sciences’ Yescarta (axicabtagene ciloleucel) – in 2017. That same year, it approved the first gene therapy, Spark Therapeutics’ Luxturna (voretigine neparvovec-rzyl) and also the first drug to include a digital ingestion tracking system, namely Otsuka Pharmaceutical’s Abilify MyCite. 2018 saw the first approval for a cannabis-based drug, GW Pharmaceutical’s Epidiolex (cannabidiol). And on Tuesday, the agency approved the first antidepressant with a novel mechanism of action in decades, Janssen’s Spravato (esketamine).
Gottlieb has been very “gung ho” about cell and gene therapy, and it is hoped his successor will be too, said Pratap Kedkhar, managing principal for consulting firm ZS, pointing to Gottlieb’s expectation of 10-20 new cell and gene therapy approvals by 2025. “The new commissioner could very well say, depending on priorities, those things are very expensive and niche, so maybe they’re not such a priority,” Kedkhar said in a phone interview. “If you do that, then the whole cell and gene therapy scene gets a bit of a setback.”
Three attributes the next commissioner should have, Kedkhar said, are a clinical background, an ability to see what is possible technologically and an awareness of where various technologies can take the world so that the US can gain an edge over China and Europe.
Dovetailing with the embrace of new drug technologies has been the embrace of digital health technology. In June 2017, Gottlieb unveiled a plan for the agency to focus on digital health products. Indeed, Abernethy was recruited from Flatiron Health, where she had served as chief medical officer. She could also be a contender to be the next commissioner, given that she was brought in with the same vision that Gottlieb had for the agency, said Chad Landmon, chair of the FDA practice at law firm Axinn, Veltrop & Harkrider.
“[Gottlieb] was certainly attuned to those issues and focused on them,” Landmon said in a phone interview. “I do think we need a commissioner focused on those.”
The tools the FDA has to regulate new technologies have often lagged their development, and digital health is an example, Landmon said. Thus, the next commissioner will have to figure out how to fit their regulation into the current confines of the FDA or encouraging Congress to make changes.
Generics and biosimilars are another one of Gottlieb’s success stories and will be important for the next commissioner. The aggressive action generics was a part of Gottlieb’s outspokenness on the issue of drug costs, even though it is not part of the agency’s mission. “He, in a refreshing way, has brought free-market competition thoughts to the drug cost debate and said that while FDA does not have a direct role in setting costs, in can play a role in reducing costs,” Landmon said.
Although Gottlieb was successful in reducing the backlog of generic drug applications, Kedkhar wasn’t so sure that the commissioner had made much progress in getting biosimilars to market, even though he made that a priority. The big question is whether the next commissioner will similarly prioritize biosimilars – whose uptake in the US is still two or three years behind Europe’s – or place them on the back burner.
But the legacy of Gottlieb’s FDA on medical devices is a bit spottier, said Jonathan Havens, an attorney at Saul Ewing Arnstein & Lehr. Havens pointed to the plan to the modernization plan that would impose a controversial 10-year limit on older predicate devices. “I get why the agency did that, but attacking the 510k system is not the panacea [or] the silver bullet to medical device regulation,” he said in a phone interview. While he said it was understandable that the FDA would desire a modernized approach, Havens pointed out that devices have not changed much anyway, and making it more difficult for devices to get to market would not be a good idea.
One potential risk as the administration looks for Gottlieb’s successor is that it could choose someone who does not push as aggressively on issues like tobacco and electronic cigarettes, biosimilars and generics or new technologies, but only wishes to act as a custodian of safety, Kedkhar said. The result could be someone who does not see it as the FDA commissioner’s role to spur innovation. On the other hand, the risk of the next commissioner being a career bureaucrat who goes back to the old-fashioned way of doing this is comparatively low, he added.
One refreshing way that Gottlieb stood apart from other commissioners in the past was his very public nature, particularly on the opioid issue, Landmon said, adding he hoped the next commissioner would embrace a public role as well. It’s likely that the next person to lead the agency will be someone in the mold of Gottlieb. “But he’s a pretty individual, so he’s hard to replace,” Landmon said.
Photo: Alaric DeArment, MedCity News