Makary Says FDA Exploring New Pathway to Accelerate Rare Disease Therapies

Rare Daily Staff

New U.S. Food and Drug Administration Commissioner Marty Makary said the agency is considering a new pathway for rare disease drugs that can allow for an approval based on a “plausible mechanism.”

Makary made the comments during an interview with Megyn Kelly on Friday on her SiriusXM show.

Makary said rare and incurable conditions that affect a small population could receive conditional approval on such a basis for treatments where the mechanism is scientifically plausible, with post-approval monitoring of how the drug is performing in real time immediately after approval.
“No one’s forcing these medications on if they want to try these new medications, even though we don’t have a randomized control trial because it’s not feasible,” he said. We will allow that and at the same time, monitor everybody who gets it so that we can make inferences as soon as the data speaks with a signal in the data,” he said.

Makary didn’t provide additional details or timing on such a pathway.

During the wide-ranging interview, Makary criticized the National Institutes of Health’s emphasis on genetics over nutrition. He said the culture of the NIH and academic medicine in the United States during the era of Francis Collins focused on the genetic causes of health problems, but no one has been talking about the food children eat.

“The genes are not the cause of our chronic disease epidemic. It’s what we are doing, or what is being done to children by adults today, unknowingly with good intentions,” said Makary. “Sometimes you go to the National Institute of Environmental Health at the NIH, go to the website, and you’ll see the director has on there that they were involved in identifying a gene that may be associated with obesity. What are you doing? I mean, there’s value in that research. How about the Ding-Dongs and cupcakes and donuts and French toast that the kids are eating with government tax dollars every morning at school.”

He said we have been focused on treating a rise in colon cancer among young people by advancing new chemotherapies, but that is not going to address the root causes. He said we need to talk about the two underlying causes of many chronic diseases, which he identified as insulin resistance and general body inflammation.

“We’re not just going to be looking at new chemo for colon cancer, we’re going to be focused on healthy food at the FDA,” he said.

Photo: FDA Commissioner Marty Makary