Via STAT News:
Gottlieb’s pledge to uphold ‘gold standard’ raises red flags
When Dr. Scott Gottlieb pledged at his confirmation hearing to uphold a “gold standard of safety and efficacy” at the Food and Drug Administration, he raised an important question:
What exactly does he mean by that?
Traditionally, “gold standard” refers to robust, randomized controlled clinical trials. But there’s enormous pressure on the FDA — from pharma companies and Congress, for starters — to accept other kinds of evidence, perhaps even patient anecdotes, to determine whether experimental drugs work. And President Trump is promising to speed drug approvals.
So when Gottlieb said: “I think there are ways to modernize clinical studies without sacrificing the gold standard,” he raised red flags.
“I don’t think he was using the phase ‘gold standard’ in the way that most of us who think about the agency for a living understand it,” said Rachel Sachs, an associate professor of law at Washington University in St. Louis.
“I think he views adaptive clinical trial design as in keeping with that gold standard,” Sachs said.
And that’s the rub, because modifying clinical trial standards has many people concerned. Reshma Ramachandran, co-chair of the National Physicians Alliance’s FDA task force, says she, too, doesn’t know what Gottlieb meant by “gold standard” — but isn’t optimistic about his interpretation.
She pointed out that Gottlieb has criticized the use of adequate and well-controlled Phase 3 trials in the past, saying they’ve needlessly delayed access to important drugs.
“That seems to indicate that his idea of ‘gold standard’ means sacrificing safety and efficacy you’d expect from FDA in favor of expediting an approval pathway,” Ramachandran said.
Joshua Wallach, a research fellow at Yale’s Collaboration for Research Integrity and Transparency, had similar worries.
“I’m concerned: I don’t want a complete erosion of the quality of clinical trial evidence that’s required from industry,” Wallach said. “There’s definitely a movement still toward deregulation — and lowering of evidentiary standards.”
Wallach is afraid, in particular, of changes to clinical trial endpoints. Modernizing the clinical trial process might force trials to lean on surrogate endpoints. That could mean assessing an experimental drug based on whether it has some interim effect (like improving a dementia patient’s performance on a memory test) rather than whether it meets more significant long-term goals (like prolonging that patient’s ability to complete daily tasks). It could also open the door to pharma companies scouring their clinical trial data to find metrics that look good — and presenting those to the FDA, even if the trial was originally designed to answer an entirely different question.
“I’m worried about seeing more small, crappy trials that don’t actually tell you anything about efficacy and safety — which are extremely important before drugs can be approved or marketed,” Wallach said.
Part of upholding the FDA’s gold standard could involve respecting and keeping on career staff, Sachs said. And for that, she gives Gottlieb high marks.
“I think he understands the importance of a strong, well-resourced FDA,” Sachs said. “I don’t think he will burn it all down. I don’t think he’s inclined to destroy the agency from within.”
Peter Pitts, president of the nonprofit Center for Medicine in the Public Interest, saw another glimmer of good news in the confirmation hearing: He said he thinks Gottlieb’s definition of “gold standard” includes keeping FDA funding strong.
“The president and his team understand that you can’t get more for less — and that’s especially true on staffing,” said Pitts, who was a former associate commissioner of FDA and worked with Gottlieb at the agency for two years. “And Scott came pretty close to saying he was against a reduction of budget.”
Pitts added that he was impressed by Gottlieb’s performance at the hearing.
“I think Scott is very much for good regulation, and very much against bad regulation,” Pitts said. “Importantly, he recognizes where FDA core mission stops, and where mission creep begins — and that’s an important finesse he’d bring to the commissioner’s chair.”