cott Gottlieb may have landed the toughest job in Washington: President Trump has selected the physician and policy expert to run the Food and Drug Administration, where a culture of control strangles innovation. An iron triangle of interest groups, the bureaucracy and the press will resist change, but Dr. Gottlieb could save lives by renovating FDA’s drug-approval processes.
Mr. Trump deserves credit for picking a pragmatist who understands the agency: Dr. Gottlieb served as a deputy commissioner at FDA during the George W. Bush Administration, and he has also worked at the Centers for Medicare and Medicaid Services. He is a resident fellow at the American Enterprise Institute and his many contributions to The Wall Street Journal include insights on doctor autonomy, drug prices, antibiotic development and more.
One of Dr. Gottlieb’s priorities will be moving generic medicines to market, and competition is the best way to reduce the price of treatments like the now infamous EpiPen. About 10% of 1,300 branded drugs “have seen patents expire but still face zero generic competition,” Dr. Gottlieb wrote in the Journal last year. “New regulations have, in many cases, made it no longer economically viable for more than one generic firm to enter the market.” Now he can roll back such arbitrary directives.
Dr. Gottlieb has also suggested that the FDA should explain its reasoning when declining to approve a drug. FDA does not release a rejection notice known as a complete response letter. The rule ostensibly exists to protect manufacturers, but the silence allows the agency and a company to peddle divergent tales about what happened. The public is left with minimal information and FDA can operate without fear of accountability.
The press is overcome with relief that President Trump didn’t pick Jim O’Neill, a Peter Thiel pal who supports making drugs available to patients after testing for safety, though not for efficacy. But that idea is far from crazy, especially for drugs that treat rare diseases when no approved options exist. Why should desperate patients have to take a sugar pill so the FDA can satisfy its demand for 100% certainty that a drug works?
Laws in 2012 and 2016 directed FDA to include patient data in reviewing such orphan drugs, but the agency has refused to modernize or rely on anything but exhaustive placebo trials. Dr. Gottlieb should make the most of this legal flexibility, though he’ll be unfairly accused of lowering standards.
Most important is that Dr. Gottlieb understands that the fundamental problem at FDA is cultural: Staff reviewers think they are the “lone bulwark standing between truth and chaos when it comes to prescribing drugs,” as he put it in a 2012 piece for National Affairs. The agency delays approvals for therapies to search for remote risks, and the cost of this method is human lives lost from excessive delays in approving new medicines.
This culture has meant “that trials continue to get longer, larger, and harder to enroll,” he wrote. The average length of a clinical trial stretched to 780 days in 2005 from 460 in 1999, and median number of procedures (such as X-rays or blood draws) on patients in trials grew to 158 from 96. To speed approvals, Dr. Gottlieb has proposed that such decisions be made by a central committee of the agency’s most senior scientists, not the same reviewers who collect and analyze the data.
Dr. Gottlieb must win Senate confirmation, and some on the left are flogging that he has consulted for pharmaceutical companies and invested in health-care ventures, which they call a fatal conflict of interest. In other words, he’s disqualified because he’s qualified.
Dr. Gottlieb will have to adhere to government ethics rules, and the irony is that if he had never worked with the industry he’d be accused of inexperience. His understanding of entrepreneurship would be a valuable addition to an FDA that has for too long pretended its decisions have no influence on research or private investment.
One last credential: Dr. Gottlieb is a cancer survivor, which means he understands the urgency of helping treatments and cures reach the patients who need them. Dr. Gottlieb can expect relentless political and bureaucratic resistance, but he also has a tremendous opportunity to unleash medical progress in this era of rapid biological discovery.
