FDA crunched by criticism at huge healthcare conference

J.P. Morgan Healthcare Conference in San Francisco

The Marty Makary self-marketing machine was on the road this week, as the FDA Commissioner flew to San Francisco for the annual J.P. Morgan Healthcare Conference. It’s billed as “the largest and most informative healthcare investment symposium in the industry, which connects global industry leaders, emerging fast-growth companies, innovative technology creators and members of the investment community.”

Makary was so excited to be there that he barely got off the plane before he recorded and posted this video, unable to contain his giddiness over “all the conversations, all the meetings and talks and panels” - almost like a young marketing intern going to his first conference.

In this less-than-60-seconds clip, Makary emphasized for the global industry leaders the flexibilities his FDA is now offering the industry on drug approvals. Adam Feuerstein of STAT News wrote on the X social media platform:

FDA tells cell and gene therapy developers not to worry about manufacturing. We got you, it's all good. We're here to help you. Marty Makary is walking through SFO shooting a video of himself to assure that THIS IS THE NEW FDA!!!!

Here is Makary’s money quote:

“While I was on the plane, we at the FDA just put out a press release announcing new flexibilities with cell and gene therapy….We are going to keep listening. We heard this was a pain point for many companies.”

That was on Sunday.

But on Monday, a flip-flop was revealed.

Atara Biotherapeutics announced that “in a complete reversal of position by the FDA,” the company was notified by the FDA that the company’s trial on a new immunotherapy drug…

“…is no longer considered to be adequate to provide evidence of effectiveness for accelerated approval. Furthermore, the FDA stated that the trial’s interpretability is confounded due to trial study design, conduct, and analysis.

The FDA’s new position is contrary to the FDA’s prior guidance to Atara, the FDA’s alignment with Atara on the clinical trial data set, and the acceptance of the trial design as a single arm study as relevant for this patient population. … We are surprised and disappointed by this FDA decision.”

The company’s stock plummeted on Monday.

Meantime, STAT also reported:

At an exclusive yacht club packed with hundreds of top hospital and health insurance leaders, Mehmet Oz and other Trump administration health officials took the stage Monday to explain and at times defend the health care policies that are reshaping the audience’s businesses.

There was wine, hors d’oeuvres, and a panoramic view of the San Francisco Bay. But the mood wasn’t entirely jovial. …

Hospitals and health insurers are staring down roughly $1 trillion in cuts to Medicaid over the coming decade, a historic change that will require them to rethink how they operate.

While Makary and Oz were trying to rub salve on industry leaders’ raw nerves, former FDA official Richard Pazdur, who suddenly quit in December as an FDA center director after just a month in that job, was at the same conference talking about politics and chaos damaging the FDA.

STAT reported on what Pazdur said:

The firewall between political appointees and drug reviewers at the agency “has been breached,” and that there is not enough transparency around a new voucher program that grants accelerated review to certain drugs selected by Trump administration officials. …

He said he was also deeply troubled by agency staff “being trampled on.” He referred to one individual who was “writing inflammatory emails using the F bomb,” telling center directors and deputy center directors that “they will go after them, that they were going to lose their jobs if they did not play ball.” He would not name that person. …

Pazdur, looking distraught, spoke of an agency crumbling, with leaders making up rules as they went and longtime, experienced staff members leaving after layoffs and perpetual uncertainty left them unable to do their jobs. At different points, he described “chaos” at the agency with staff having gone through “a lot of trauma,” and said “you do not have stability.”

After today there’s still another day left at the JPM Healthcare Conference. Who knows what tomorrow will bring? Count on STAT to keep us informed.

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