hen President Trump nominated Scott Gottlieb for commissioner of the Food and Drug Administration, you could almost hear the sigh of relief that rippled through the health care community.
That’s because Gottlieb isn’t Jim O’Neill, whose name had also been floated for commissioner. O’Neill has no background in health care except as a venture capitalist, and thinks that the way to speed new drugs to the market is for the FDA to drop any requirements that drug makers demonstrate that their products actually work. Gottlieb, in contrast, is an internal medicine physician and a drug company insider, who presumably knows you need at least some scientific evidence for efficacy.
The trouble with feeling relieved by Gottlieb’s nomination? He may not be much better than O’Neill when it comes to protecting the public from the health and financial risks of harmful or useless drugs and devices.
Gottlieb has a long history of supporting deregulation of medical products. He supports off-label marketing of medical devices, including Class III devices, which are the most potentially dangerous. He’s stated that “FDA’s caution is hazardous to our health,” claiming that the agency is too slow in its approvals, and prevents patients from getting new drugs they desperately need because it is wedded to “statistical results.”
In reality, the FDA has become the fastest drug regulatory agency in the world, going from an average of 30 months per drug review in the 1980s to 8.5 months today.
But speedier approval doesn’t necessarily translate into better outcomes. Seventeen drugs approved by the FDA between 1993 and 2010 — six of them on expedited schedules — were prescribed 100 million times before subsequently being withdrawn from the market because of safety concerns.
Gottlieb has also recommended that the agency speed up product approval by accepting secondary measures of disease, or so-called surrogate markers. These are things like a drop in cholesterol standing in for the outcome we really care about — a decline in heart attacks or strokes.
Using surrogate markers to gauge the effectiveness of new products would allow companies to conduct shorter and smaller studies. That is great for the bottom line, but, again, past experience suggests it may not be good for patients.
In 2012, the FDA gave fast-track approval to Sirturo (bedaquiline), a tuberculosis drug, based on a small trial of just 47 patients. The trial looked at a surrogate marker — the drug’s effectiveness in getting rid of the tuberculosis bacterium in the sputum of patients. A follow-up study showed that patients who took the drug were five times more likely to die — an outcome that certainly worries people with tuberculosis a lot more than having bacteria in their spit.
Fast-tracking, small trials, and surrogate markers are all music to manufacturers’ ears, because of the high costs of testing products before they can go to market. But FDA commissioners are supposed to protect the public’s health, and it’s not clear whose interests Gottlieb will represent.
The fact that Gottlieb served as deputy commissioner of the FDA from 2005 to 2007 under President George W. Bush and is now a fellow at the American Enterprise Institute, a conservative think tank in Washington, D.C., might seem to suggest he’s had relevant experience for the job. In fact, he’s had scant scientific, academic, or government experience compared to previous commissioners.
His background also suggests his loyalty lies with industry. Gottlieb is a partner at New Enterprise Associates, a global venture capital firm that invests in technology and health care. If confirmed to the new post, Gottlieb will hold the dubious distinction of having more financial conflicts of interest with the industry he is supposed to regulate than any previous FDA commissioner. Between 2013 and 2015, Gottlieb received consulting fees and speaking honoraria amounting to more than $400,000 from drug and device companies according to the federal OpenPayments database.
Will Gottlieb’s decades-long relationships with and six-figure payouts from the drug and device industry affect his ability to regulate those companies? He doesn’t think so.
In his view, there is nothing wrong with physicians being paid by industry for “valuable scientific consulting.” Problems have arisen only from the “small number of doctors [who] took excessive payments from medical product companies.” (Might $400,000 over two years counts as “excessive”?)
Despite Gottlieb’s coziness with the industry he is slated to regulate, Democrats in Congress have largely let his nomination slide, with only a few members expressing concern. Even Senator Elizabeth Warren, who railed against the 21st Century Cures Act as a law that would “cover up bribery” by relaxing disclosure rules, has yet to put out a statement or tweet about Gottlieb’s nomination.
Part of this acceptance likely stems from the fact that Gottlieb is not O’Neill. It also reflects the normalization of conflicts of interest in medicine, which has been debated in the pages of the New England Journal of Medicine and in the Lown Institute blog.
Gottlieb has criticized government efforts to shed light on conflicts of interest, such as the Physician Sunshine Act, as “federal tinkering” leading to “the demise of American medicine.” We believe his confirmation will lead to the demise of some FDA rules that are already barely keeping a lid on useless or dangerous medical products.
Judith Garber is a health policy and communications fellow and Shannon Brownlee is senior vice president at the Lown Institute, a nonprofit, nonpartisan think tank based in Boston. One of its missions is to expose and eliminate the harm caused by financial conflicts of interest in health care.