Gottlieb signals support for both ‘gold standard’ and expedited review for drug approvals
By Meghana Keshavan @megkesh
November 30, 2017
Regulatory standards for some clinical trials may soon slacken, per a new statement from Food and Drug Administration Commissioner Scott Gottlieb. And though his words are vague, they’ve been enough to raise red flags in some corners.
Testifying today before a Congressional committee on the 21st Century Cures Act, Gottlieb sent a mixed message: He wants the agency to “remain steadfast to our gold standard for safety and efficacy,” while making the development of breakthrough products “more scientifically modern and efficient, to meet the urgent needs of patients.”
Gottlieb said that in some cases, “when there’s a clear and outsized treatment effect,” a cancer drug might get expedited approval — and its efficacy will be evaluated only in postmarket studies.
The agency said it intends to release a document soon that will outline how it plans to expand on the Cures Act.
The term “gold standard” has historically referred to rigorous randomized controlled clinical trials. But this form of drug evaluation is onerous and costly — and many argue that it prevents promising experimental medicines from reaching patients quickly enough. The 21st Century Cures Act was devised, in part, to speed up that process — granting the agency $500 million over the course of a decade to work out the intricacies. And President Trump has spoken of hastening drug approvals.
Still, this gold standard is what keeps ineffective, or even dangerous, drugs away from patients. And there isn’t much evidence that speeding along approvals will bring better results to patients. A recent BMJ study, for instance, found that more than half of all new cancer drugs “show no benefits” for either survival or quality of life.
A big sticking point with Gottlieb’s apparent approach is that small clinical trials simply don’t offer enough meaningful data.
“Empirical evidence suggests that the findings from individual trials may be spurious,” said Joshua Wallach, a research fellow at Yale’s Collaboration for Research Integrity and Transparency. He added, as additional studies are performed, the effects observed in small, early stage trials often attenuate — and can even be invalidated.
“With cancer drugs, the patients in these small trials have been so carefully selected, they will do well whether you give them a good new drug or sugar water,” said Dr. Vinay Prasad, an oncologist at Oregon Health & Sciences University.
Meanwhile, the current system for monitoring postmarket drug efficacy remains highly flawed, according to a 2016 report from the Office of Inspector General. Since it’s harder to track a drug’s efficacy in the real world, which functions rather differently than a controlled trial, FDA already has issues with data management and workflow, the report concluded. As a result, many of these studies aren’t completed on time — or even at all, according to an August study in the New England Journal of Medicine.
The concern is that the agency is not equipped to handle a new influx of postmarket data — and much could slip through the cracks. It’ll take substantial investment to help plug those holes and create a smart system for postmarket analysis, Wallach said.
“FDA understands that different products need different regulatory pathways, from both a science and a patient-need perspective,” said Peter Pitts, president of the nonprofit Center for Medicine in the Public Interest.