Scott Gottlieb is leaving the FDA. Now what?

Scott Gottlieb FDA
Scott Gottlieb, M.D. (FDA)

Scott Gottlieb’s resignation from the FDA has exposed the biopharma industry to the thing it perhaps likes least of all: uncertainty. The question now is whether the FDA will stick to the relatively steady, industry-friendly course it charted under Gottlieb or veer into choppier waters. 

To an extent, Gottlieb inherited an agenda for his time at the FDA in the form of the 21st Century Cures Act. That act has been the catalyst for some of the changes made by the FDA during Gottlieb’s tenure and will continue to provide a framework for his successor. Yet, Gottlieb’s agenda went well beyond the act and drove the FDA to embrace new technologies and smooth their paths to market, leading to guidance on Alzheimer’s disease, gene therapies, NASH and other fields.

Data collated by analysts at Jefferies shows Gottlieb’s FDA published more than 200 guidance documents last year, easily surpassing the annual outputs of his predecessors. Gottlieb’s FDA approved more drugs, too. That may be partly because Gottlieb’s reign coincided with an innovative period for biotech, but it is also true that the FDA has relaxed the rules on occasion over the past two years, for example by approving Johnson & Johnson’s Spravato on the strength of mixed clinical data.

After two years in which the FDA has gently relaxed the rules, the industry is worried about what may come next. The trepidation is underpinned by the views of some of the names that were reportedly in the hat for commissioner before Gottlieb landed the job and President Trump’s track record of picking people to lead other parts of the government.

Mick Mulvaney arrived at the Consumer Financial Protection Bureau (CFPB) having called the body a "sick, sad" joke and pushed for its elimination. Mulvaney’s actions as acting director of the CFPB, such as a freeze on rulemaking and $0 budget request, were in line with his prior comments on the body. Equally, Trump nominated Rick Perry as secretary of energy. As a presidential candidate, Perry called for the elimination of the Department of Energy. Scott Pruitt’s nomination to lead the Environmental Protection Agency was similarly controversial. 

None of those nominees have had the sort of largely-well-received, scandal-free reign that Gottlieb has enjoyed at the FDA. Of the three nominees, Perry is the only one still in the job, and his time at the Department of Energy has been dotted with controversies about his positions on climate change and other topics.

Had Trump nominated one of Gottlieb’s reported rivals for the FDA job, the agency could have faced a similarly turbulent period. The original field of contenders for the commissioner job, which was mooted, included one person who said the efficacy of drugs should be proved “after they’ve been legalized” and another who argued that a “Yelp for Drugs” would be “vastly better” than the FDA. 

In the near term, principal deputy commissioner Amy Abernethy, M.D., Ph.D., could step up on an interim basis. Beyond that, National Cancer Institute Director Ned Sharpless, M.D., HHS Assistant Secretary Brett Giroir, M.D., and others have been floated as potential replacements. Someone of that ilk is arguably more likely to land the role than the sort of wild cards that were mooted last time.

That is unlikely to stop people from worrying, though. After falling 4% on Wednesday, the XBI biotech index was down again in premarket trading on Thursday.