David Shaywitz is chief medical officer at DNAnexus, a visiting scientist in the Department of Biomedical Informatics at Harvard and an adjunct scholar at the American Enterprise Institute, where Scott Gottlieb also serves as a resident scholar.
Senators are convening Wednesday to evaluate Scott Gottlieb, President Trump’s nominee for FDA commissioner, conservatives will undoubtedly lament the state of the agency while liberals fret about Gottlieb’s business background. But both stances may be incorrect: The FDA is showing a real appetite for change, and the agency is likely to benefit enormously from Gottlieb’s experience in the industry.
According to many on the right, the FDA is a “government bottleneck that imposes costs, delays, and uncertainties”; an agency that “delays approvals for therapies to search for remote risks”; and a place where “a culture of control strangles innovation.”
There’s more than an element of truth to this, as I painfully experienced in a previous role leading a drug development team. I often tangled with the FDA’s ultra-cautious regulators, who worried a lot about remote risks — and seemingly less about the patients who were suffering without an approved effective therapy.
But over the past five years, I’ve also seen what feels like the beginnings of a new FDA — an agency that seems authentically committed to accelerating innovation, embracing creativity and bluntly acknowledging the need to improve.
The first indication that the FDA might be on the right track came in 2013 in the form of an impassioned speech by Norman Stockbridge, director of the agency’s prestigious Division of Cardiovascular and Renal Products. Speaking to a standing-room-only crowd at Stanford University, Stockbridge eschewed the anticipated bureaucratic banalities and dove right in, candidly highlighting concerns about the FDA’s “risk-aversion culture.” He emphasized the need to deal with false-positive safety signals more effectively to avoid negative consequences, such as delayed approvals and the reluctance of clinicians to use effective drugs that might have been unfairly impugned.
The new attitude seems to be taking hold: Many innovators working in digital health, for instance, now rave about their experience working with the agency, highlighting engagement with FDA leaders such as Courtney Lias, who directs the Division of Chemistry and Toxicology Devices, and Stacey Beck, who focuses on diabetes diagnostics. The FDA has also prioritized patient engagement through programs such as the Patient-Focused Drug Development Initiative, the Patient Representative Program and openFDA.
I’ve also witnessed this evolution. My current company (DNAnexus) partnered with the FDA in 2015 to develop a software platform called precisionFDA, which allows researchers to upload and share genome data with other scientists. The project — the brainchild of data scientist Taha Kass-Hout, who left the FDA last year — aims to build a research community that could improve the quality and accuracy of genome-based medicine.
The left, however, remains characteristically concerned that regulators will cozy up to the industry they are supposed to critically evaluate. They argue that Gottlieb’s lucrative relationship with the biopharma industry — as well as his lack of a university science background — makes him unsuitable for the position.
This critique reflects a surprisingly pervasive view within academia that both expertise and integrity are concentrated in universities and dissipate as you move further out.
For many in the academy, the true work of drug discovery is the journey from scientific inspiration to academic publication — a perspective that fails to appreciate the extraordinary effort required to go from the tenuous conclusion of a scientific journal article to a robust commercial product. This seems a bit like relying on a well-read virgin for advice on sex. There are some things you learn only by doing, and developing drugs is one of them.
As an investor in early-stage life-science companies, Gottlieb has presumably developed a hard-won, highly valuable understanding of the nuance of product development. This will help him identify opportunities for accelerating the approval process, as well as to head off any nefarious attempts to game the system at the expense of patients.
If Gottlieb can leverage his experience as a company builder (along with his experience as a doctor, policy wonk and cancer patient), he might be able to unleash the latent passion within the FDA and inspire its innate creativity. With the right leadership, the FDA — however improbably — might lead us out of our present health-care miasma.