Commentary – Scott Gottlieb’s Legacy

Scott Gottlieb, FDA Commissioner, in November 2017 (A. Kotok)

10 Mar. 2019. A standing rule in Washington, D.C. is when an office-holder resigns to “spend more time with my family,” he or she is about to be caught in some kind of scandal. In the case of Scott Gottlieb, Commissioner of the Food and Drug Administration, however, he really wants to spend more time with his 3 growing children in Westport, Connecticut, where he commutes each weekend. Gottlieb announced his resignation this past Tuesday, 5 March, and he’s shown you can believe what he says.

When Gottlieb was sworn in as commissioner in May 2017, he came well prepared for the job. He served as FDA’s deputy commissioner in the George W. Bush administration, and also as a partner at New Enterprise Associates, a venture capital company in Chevy Chase, Maryland. In addition, Gottlieb has an M.D. degree from Mount Sinai medical school in New York, and later practiced internal medicine at New York University medical center. And he brings a patient’s perspective to the job as a survivor of Hodgkin’s lymphoma, a form of blood cancer, according to Wikipedia.

When Gottlieb announced his resignation, much of the news coverage focused on his battle against e-cigarettes, commonly called vaping. But his legacy goes much deeper. Gottlieb is probably the most un-Trumpian of this administration’s appointees: serious, professional and evidence-driven. In an administration that prides itself on disregarding, if not battling, scientific findings, Gottlieb stood out for letting science govern FDA’s work.

Gottlieb’s legacy is aided by the 21st Century Cures Act, signed into law in December 2016, that gave FDA new tools to speed new kinds of medical products to the marketplace, such as regenerative medicines. During his tenure, FDA issued a number of guidance documents outlining new regulatory processes and practices that streamline reviews, yet are still dictated by scientific evidence. Here are 10 of these documents and advances reported by Science & Enterprise:

• A digital health innovation plan in June 2017 to review and certify the rapidly growing number of new medical apps for mobile devices
• Clinical trials for precision medicine in December 2017, and a companion document covering diagnostic tests that determine a patient’s status for taking part in clinical studies of therapies based on genomic factors
• Release of more clinical trial data in January 2018, and in a more user-friendly form
• New methods for collecting data from patients in June 2018 that the agency says will help improve evaluations of more precisely targeted drugs
• A series of regulatory guidance documents on gene therapies, in July 2018, covering therapies for hemophilia, retinal disorders, and rare diseases
• Pilot tests of new types of complex clinical trials in August 2018, and proposals for a procedure to evaluate machine learning algorithms used in diagnostics
• Regulatory guidance in October 2018 to cover advances in biotechnology, including genome editing, in animals and plants
• Streamlined policies in November 2018 for making drugs and medical devices in clinical trials or under review available for people in the U.S. with life-threatening conditions
• Identification of a public database of genomics and diseases in December 2018 as a resource to assess new diagnostics tests using genetic data for precision medicine
• Regulatory guidance in February 2019 for FDA review of implanted medical devices that connect human brains to computer systems

I heard Gottlieb speak at National Press Club in November 2017, and was struck by the serious, technocratic, and business-like tone of his talk, hardly the rantings of an ideologue. And the industry reacted negatively when he announced his leaving FDA; according to CNBC, a biotechnology stock index fell more than 2 percent the next day, attributed to the resignation announcement.

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