With just one week left to go in his time as FDA commissioner, Scott Gottlieb, M.D., described the serendipity of being able to lead the agency while it’s “writing the modern rules.”
When he was sworn in to the post just under two years ago in May 2017, Gottlieb was aware that it would be an exciting time to come back and lead the agency: Among other reasons, the FDA had recently been handed a new set of directives in form of the 21st Century Cures Act, and was also looking to renew its user fee power by the end of that summer—big tasks, even by themselves.
But when faced with sprinting advancements in cell therapies and regenerative medicine, as well as cheaper genomic lab tests and an explosion in digital health, the FDA found that its existing legal and regulatory frameworks didn’t really apply, and couldn’t keep up.
“I felt I had a good window into the agency, having worked for three different commissioners—but it’s very different when you're the one in the chair,” Gottlieb said to one of those three, Mark McClellan, during something of a fireside chat before he joins the club of ex-agency chiefs, hosted by the Alliance for a Stronger FDA and the Pew Charitable Trusts in Washington, D.C.
“I think what's changed most notably about the agency from the time I was there was that the scope has expanded so dramatically, in not a long period of time,” he said, with the FDA approving its first cell-therapy based cancer treatments, true genetic therapies and new avenues for the industry to reach patients in the form of in vitro diagnostics and smartphone apps.
“These are dramatic expansions in the scope of the work that we do. It's really changed the contours of the agency's mission, and has made it far more diverse and vast,” Gottlieb said. “We actually had to conceive of different ways of regulating.”
Many of those initiatives focused on making the regulatory process more efficient: as one of his accomplishments, Gottlieb pointed to the new records the agency has set for new drug approvals (and apologized to McClellan for breaking one of his.)
But while scientific progress has produced transformative therapies, the costs of treatment and the prices of new drugs have become their own public health issue, McClellan said. “Can you comment on that at all?”
“Well I’ll have a lot more to say on that in about seven days,” Gottlieb said with a laugh.
But in that vein—though certain topics remain taboo for a commissioner—Gottlieb has been noted for being an open and outspoken FDA leader, and showing that the job can be not just about protecting the public’s health, but promoting it as well. He’s brought the role further into the public eye, through orchestrated news events, frequent statements to journalists and heavy engagement on Twitter.
“I would ask you what keeps you up at night, but as one of two people in this administration fairly reliably tweeting at both midnight and 5:00 a.m., I know you don’t sleep very much,” McClellan said.
One of his main worries, Gottlieb said, was the idea of catastrophic risks being widely distributed before the FDA caught wind of them. While it’s gotten very good spotting early signals in drug therapies, those fears remain on the food and blood sides of the house, he said, pointing to recent steps toward bringing pathogen inactivation technology into the blood supply testing system.
“I mean, aside from waking up and seeing a tweet about me in the morning—that always worried me,” Gottlieb said.
Going forward, Gottlieb said he had “extreme confidence” in the agency’s leadership team to continue the initiatives he began, including in the incoming interim commissioner, Ned Sharpless, currently the director of the National Cancer Institute.
“He’s exceptionally public health-minded, and an outstanding clinician, as well as an inventor himself,” Gottlieb said.
He also named Amy Abernethy, formerly of the real-world data firm Flatiron Health, who joined as principal deputy commissioner last December, and said that the agency’s informatics experts would be “taking a leadership role” on information technology in the near future.
In addition, he pointed to the triumvirate of the agency’s long-time center directors: Janet Woodcock covering drugs, Peter Marks in biologics and Jeff Shuren overseeing devices
“So all the policy I worked on was formulated in concert with them and their staff,” Gottlieb said. “It all came out of the centers, and I think that made it successful. And it made sure that we had a broad consensus around what we were doing.”