How Scott Gottlieb changed the FDA
Scott Gottlieb took over as head of the Food and Drug Administration with a somewhat unconventional agenda, and amid some criticism from Democrats of his ties to drugmakers and other corporate interests.
He quickly won over skeptics, however, and news of his departure elicited praise across the political spectrum. And while the agency's core missions of drug and food safety remain unchanged, Gottlieb was able to elevate different, specific issues into the limelight during his time as commissioner.
The FDA is not radically different than it was two years ago, said Wayne Pines, a former senior FDA official and agency observer for 50 years. But, Gottlieb's focus on e-cigarettes, opioids and drug prices gave the agency new focus at the highest level, he said.
"None of those three issues were being discussed before Gottlieb got there," Pines said in an interview. "And now, they're being discussed."
More specifically for pharma, Gottlieb's willingness to call out drugmakers for what he memorably dubbed "shenanigans" and "Kabuki drug pricing" pushed the industry in a more public manner than past agency chiefs.
Under his leadership, the FDA flagged anti-competitive concerns including drugmaker abuse of the REMS system and citizen petitions, as well as publishing a list of off-patent drugs with no generic competition.
Still, he remained popular among the pharmaceutical and biotech industries. The agency clocked record-high numbers of both generic and novel medicines in 2017 and 2018. And the physician pushed an array of modernization efforts for the agency, including a recent outline for how the agency will prepare for an influx of cell and gene therapy development.
Gottlieb was "about as market friendly, patient focused and industry friendly as one could have hoped," wrote Tyler Van Buren, an industry analyst at Piper Jaffray, in a Mar. 5 note to investors. That friendliness, though, came with questions on how well the FDA under Gottlieb was upholding the agency's historical standards on safety and efficacy.
Those questions are particularly relevant as the FDA faces a coming wave of experimental treatments that will test the traditional frameworks for assessing drugs.
Michael Gladstone, a principal at the venture capital firm Atlas Ventures, applauded Gottlieb for approaching review of cutting-edge therapies in a transparent and balanced way.
"This is perhaps especially true when it comes to novel modalities, like gene therapy and cell therapy, and novel regulatory frameworks, like biomarker-guided histology agnostic approvals for Viktravi and Keytruda," Gladstone wrote in an email to BioPharma Dive.
Stephan Grupp, director of the cancer immunotherapy program at Children's Hospital of Philadelphia, said in a statement to BioPharma Dive the agency was a great partner in working alongside trials he helped run for Novartis' then-investigational CAR-T therapy, Kymriah.
Grupp added the commissioner pushed for nimble trial designs, and said his statements on cell and gene therapies have been "incredibly important for the FDA to hear."
"He is a thought leader and a real loss," Grupp said.
Pines, who has written a book on the agency's history, called Gottlieb "the most digitally savvy commissioner ever."
"He'll be a tough act to follow, especially in regards to his intimate understanding of communications and how to use the FDA bully pulpit to advance his issues," Pines said.
In addition to a steady stream of official statements put out by the agency coming from the commissioner's desk, Gottlieb's Twitter account, @SGottliebFDA, grew into a vital, direct source of information.
Through semi-regular Twitter threads, dubbed "tweetorials," he went in-depth on issues ranging from the opioid crisis to continuous trials, previewing guidances and other agency actions to the public.
And during the longest government shutdown in U.S. history this winter, Gottlieb leaned on the social media platform to spread updates on the agency's plans and actions while the communications department was impacted.
He tweeted timelines for how long user fees would last and how the FDA was prioritizing its funds during what he called "one of the most significant operational challenges in FDA's recent history."
"His legacy is going to be a more activist agency," said James Shehan, chair of Lowenstein Sandler's FDA regulatory practice. "One that his successors are going to follow his lead. They are going to be more actively communicative with the public than they've ever been and more willing to take a role in some of the great public health controversies of the FDA."
Further, in a Trump administration where the morale of many federally-employed scientists has fallen, Gottlieb was able to keep the FDA's regulatory mission humming along, observers said.
The end of his era
In a letter to agency employees, Gottlieb attributed his resignation to the time spent away from his family, made more challenging by a lengthy interstate commute he regularly made to and from Connecticut.
When he leaves the White Oak campus in a month, the vacancy will carry questions of how, and if, the successor will pick up on the multitude of ongoing plans, particularly some of the more aggressive agency regulatory ambitions.
Outside the pharma industry, his plans to tackle rising use of e-cigarettes and vapes by teenagers has reportedly stirred some discontent within GOP ranks.
But, by and large, experts expressed approval for the balance Gottlieb was able to strike in his nearly two years.
"It really stands out in a deregulatory environment, you've seen someone who's actually advanced regulation but in a way that has not been particularly onerous or generated a lot of controversy," Shehan said. "That's quite a trick."
Ned Pagliarulo contributed reporting to this article.
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