President Trump has named Scott Gottlieb as the next FDA commissioner, resolving an issue that has huge implications for the biopharma industry and drug development.
Gottlieb’s nomination will be widely applauded by the biopharma industry, which will see the move as a commitment for continued reform without the kind of wholesale deregulation that would scuttle the agency’s gold standard for drug reviews.
“Thank God it’s Gottlieb,” messaged Baird’s Brian Skorney in his weekly wrap-up.
As I reported two weeks ago, Gottlieb has mapped out an aggressive reform agenda in anticipation of this appointment.
According to his statements as well as comments he’s made to people familiar with his thinking on the FDA, Gottlieb intends to shoot for the rapid approval of complex generics, ushering in a wave of less expensive rivals to some of the biggest blockbusters on the market. He’s also likely to spur the FDA to follow the course laid out by agency cancer czar Richard Pazdur in speeding new approvals, possibly setting up a special unit aimed at orphan drugs to hasten OKs with smaller, better designed clinical trials.
Gottlieb is likely to roll back rules related to pre-market development, most noticeably the cardiovascular outcomes studies for some new meds. A new emphasis on biomarkers as a surrogate endpoint along with an open embrace of simulation and modeling could be next.
Other potential reforms include the possible quick adoption of new devices that could be used to improve the kind of medtech Apple, Verily and others have been working on.
Gottlieb has also backed the publication of the FDA’s complete response letters, detailing the reasons why the agency rejects a drug. Over the years regulators have routinely complained that many companies have been less than honest in recounting the FDA’s position on a drug. The FDA is currently restricted by law in its public discussion about a new drug approval.
The move could spell relief for Amicus Therapeutics $FOLD and CEO John Crowley, who personally lobbied the president on the FDA’s decision to delay any final decision on their drug for Fabry disease so they could see the results of a safety study in 2019.
Left out in the move: Jim O’Neill. The close associate of Peter Thiel, O’Neill famously suggested that drugs should be approved based on safety alone, letting consumers sort out what works. That left many fearing that Trump intended to toss out the regulatory framework for new drug approvals, raising fears that his idea of competition would allow de facto placebos to compete for market share.
Trump has repeatedly criticized biopharma for outrageously high drug prices, vowing to simplify the drug development process as he also sought to dramatically lower the price of drugs. Gottlieb will be expected to deliver.
“Among the FDA commissioner candidates we’ve read about, Dr. Gottlieb is well known to industry and has the requisite experience and talent to reform and streamline the FDA approval process,” Holland & Knight’s Michael Gaba told me recently.
One of the reasons that Gottlieb is well known in the industry is that the physician and former FDA official under George W. Bush has plenty of direct ties to it. As a venture partner at New Enterprise Associates he’s a board member at MedAvante, which markets software for analyzing clinical trial data. And he’s been an advisor to GlaxoSmithKline, which has shown no appetite for radical change in the way developers prove a drug works.
Those ties, along with Trump’s freewheeling comments on deregulation, will now become targets for Democrats in Washington DC. But unless there’s a nasty surprise hidden from view, none of it presents a serious challenge to his acceptance as head of the FDA.
— Donna Young (@DonnaYoungDC) March 10, 2017