Shortly after taking office, President Trump told pharmaceutical CEOs visiting the White House that he would accelerate drug approvals by eliminating 75 to 80 percent of the regulations enforced by the US Food and Drug Administration. “Instead of it being 9,000 pages, it’ll be 100 pages,” he said. Slashing those rules could fall to Scott Gottlieb, the president's pick for FDA commissioner.
As with so many Trump nominees, expect Democrats to press the 44-year-old physician on his conflicts of interest when he appears before the Senate for confirmation. Gottlieb's deep ties to the industry he would regulate go back more than decade, and he currently serves on the board of three pharmaceutical companies. His venture capital firm, new Enterprise Associates, funds 150 others. Gottlieb received more than $400,000 from Big Pharma between 2013 and 2015, according to a database maintained by the Centers for Medicare & Medicaid Services.
Of course, Gottlieb must resign from those boards and sell any stock in companies regulated by the FDA if he wants the job. He could also recuse himself from anything involving companies he's worked with, as he did during his stint as the deputy commissioner of the FDA under President George W Bush. But Democrats worry Gottlieb might use his authority to push through products developed by his pharma pals. Cronyism aside, would that be such a bad thing? The companies he's worked with do vital work that few others can afford to do.
No one likes that a prospective head of the FDA took $410,000 from GlaxoSmithKline. But this isn't the same as putting the former head of ExxonMobil in charge of the State Department, a climate change enthusiast in charge of the Environmental Protection Agency, or a charter school advocate in charge of the education department. Gottlieb's friends in the industry have robust product pipelines and strong reputations for R&D and productivity---suggesting they've got some promising drugs in the works. Here’s a look at just a few of them:
Gottlieb’s relationship: Independent advisor from September, 2007, through August, 2016
Money exchanged: $156,000 in consulting fees between 2013 and 2015
Disease: Cystic fibrosis
Patients affected worldwide: 70,000
In 2012, Vertex received FDA approval for Kalydeco, the first drug to treat the underlying causes of cystic fibrosis---the misfolding and malfunction of various channel proteins in lung cells. That malfunction causes mucus and fluid to accumulate in the lungs, eventually causing death. Kalydeco binds to the misfolded proteins, increasing their ability to remain functional. But the drug works in just 10 percent of patients. Because cystic fibrosis is a genetic disorder, about a dozen mutations can affect the proteins. In the most common mutation---which causes two-thirds of cystic fibrosis cases, the protein is so misfolded up it never makes it to the cell surface.
The FDA approved a second Vertex drug, Orkambi, in 2015. It causes Kalydeco to combine with a molecule called an effector that binds to the protein and brings it to the cell surface. Together, the two drugs can treat half of all patients. The company is now testing the drug regiment with five other molecules to target other mutation-specific misfoldings. Getting the full suite could effectively cure cystic fibrosis.
Gottlieb’s relationship: Independent member of Product Investment Board from January 2010 until now
Money exchanged: $410,000 between 2010 and 2015
Disease: Adenosine deaminase deficiency
Patients affected: 1 in 100,000 births annually
Adenosine deaminase deficiency is a rare genetic immune disorder that leaves people susceptible to chronic infections (the “bubble boy syndrome”), but the impact of this drug could be immense. Strimvelis uses a “replace and repair” gene therapy strategy to swap a dysfunctional gene in bone marrow cells with the correct copy. Regulators in Europe approved the drug in May, and GlaxoSmithKline plans to seek approval from the FDA later this year. It would be a landmark for the company’s gene therapy platform, and for gene therapy in general. Today, the field includes dozens of companies developing several hundred drugs that work in a similar fashion. An approval for GSK will pave the way for others, opening new avenues of treatments and perhaps even cures for the 5,000 rare diseases caused by errors in a single gene.
Gottlieb’s relationship: Has served on the company board since May, 2015
Money exchanged: $50,000 in 2015
Disease: Chronic pain
Patients affected in US: More than 75 million
The roots of the nation's opioid epidemic lie in a troubling statistic: one in four Americans live with chronic pain, and doctors have few non-narcotic treatments to choose from. Daiichi Sankyo, Japan's second-largest pharmaceutical firm, is developing novel small molecules to block pain signals before they reach the brain. The company has no small experience with opioids, most recently with extended release products Morphabond and Vantrela ER that can't be snorted, and it is eager to untangle and target other pain receptor pathways in the hope of finding a nonaddictive solution.
Many other drugs and therapies, along with medical devices, lab tests, and stem cell treatments, will cross Gottlieb's desk should the Senate confirm him. (No date has been set for the hearing). Democrats may lament his ties to industry, but David Gortler, a pharmacologist at George Washington University and a former FDA medical officer, says it is difficult finding candidates who doesn't have some ties. Robert Califf, the current commissioner who came to the FDA from Duke University, saw as much as half of his research funded by the industry. The nature of the work, where a single clinical trial can cost $100 million, means only the biggest companies can finance it. And the companies on Gottlieb's list have a strong track record in public health. “It’s not an exaggeration to say that literally hundreds of millions of people owe their lives to these companies,” Gortler says. As FDA commissioner, Gottlieb's experience could help save millions more.
