LAUREN GARDNER and DAVID LIM, Politico, November 14, 2025
PLAUSIBLE PATHWAY QUESTIONS — FDA Commissioner Marty Makary’s “plausible mechanism pathway — detailed in a New England Journal of Medicine article — is drawing questions from patient advocates who wonder how it will be used in the years ahead.
Robert Steinbrook, director of the Health Research Group at the consumer advocacy nonprofit Public Citizen, said he hopes the FDA issues more details about how the pathway will work. He said the pathway should be used for individual patients who have diseases with well-understood genetic and biological causes — a use he called an interesting and appropriate application.
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Diana Zuckerman, president of the nonprofit think tank National Center for Health Research, also questioned whether insurance companies would pay for products approved through the new pathway if questions remain about their evidence.
“It seems to me what it’s basically doing at best is offering desperate families or desperate individuals the opportunity to pay however much the company wants to charge for something that plausibly might work,” Zuckerman said of products that would be approved through the new pathway.
Often, companies providing experimental treatments through the FDA’s compassionate use program don’t charge patients for those products, even though they can charge for an investigational medical product. That’s because costs charged to a patient receiving them through the FDA’s expanded access program must be justified and limited to recovering manufacturing, research, development and handling costs.
“It’s the job of the FDA to make sure that new, effective products are on the market when possible,” Zuckerman said. “But I just think this should be more of an extension or an expansion of the expanded access program.”
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