In an interview with former Fox News journalist Megyn Kelly, FDA Commissioner Marty Makary introduced a new mechanism-driven pathway that could be leveraged by rare disease therapies while saying that autism could potentially be driven by certain environmental factors.
The FDA is eyeing a “new pathway” for rare disease drugs, potentially allowing for their approval even without data from a randomized, controlled clinical trial, recently confirmed FDA Commissioner Marty Makary said in an interview on Friday.
Speaking on The Megyn Kelly Show in his first major interview since taking the reins at the FDA, Makary said the agency will open a new regulatory pathway based on what he called a “plausible mechanism,” focusing mainly on rare or incurable diseases that affect “a small number of people.”
If there is an investigational drug, Makary explained, “that makes sense physiologically. The mechanism is scientifically plausible that this treatment would help these individuals.” The FDA could approve that therapy on “a conditional basis . . . even though we don’t have a randomized controlled trial because it’s not feasible.”
Makary, however, was quick to caution that “no one is forcing these medications [on patients].”
This new mechanism-driven pathway would also include a patient surveillance system, which would “monitor everybody who gets [the treatment], so that we can make inferences as soon as the data speaks,” Makary added.
The interview comes after Peter Marks, former head of the Center for Biologics Evaluation and Research, left the agency late last month. Marks was a supporter of regulatory flexibility for rare diseases.
In an interview with BioSpace in July 2024, Marks emphasized the importance of accelerated approvals for rare disease therapies, noting that timely treatment for these patients could lead to dramatic improvements in quality of life.
In the interview with Kelly, Makary also addressed what he called the “epidemic of distrust” toward the FDA—and health authorities in the U.S. more broadly—fostered primarily by the agency’s alleged downplaying of side effects of the COVID-19 vaccines.
Makary also touched on vaccines, seeming to partly back up claims made by Health and Human Services Secretary Robert F. Kennedy Jr. connecting vaccines to autism despite overwhelming evidence to the contrary.
While noting the possibility that certain environmental factors may indeed play a role in the developmental disorder, Makary stopped short of naming vaccines.
Of the supposed distrust against health authorities, Makary said that “part of it is warranted.”
“When you don’t want to look at complications, the complication rate looks lower than it really is. It makes products look safer than they really are.” At the center of this, according to Makary, is the FDA’s Vaccine Adverse Event Reporting System (VAERS), which is intended to monitor side effects associated with immunization.
VAERS, crucially, is self-reported, meaning it relies on patients and physicians to file reports of side effects. “You can make no inferences about rates” using self-reported data, according to Makary. “That’s just a basic scientific methodological principle.”
Instead, the FDA will now employ a more comprehensive method, tapping into what Makary called the “Health Information Exchange,” a “massive” repository of electronic health record data, which will allow the FDA to look at real-world complication data.
Last week, Kennedy also took aim at VAERS, saying, “it’s outrageous that we don’t have a surveillance system that functions.” Kennedy is eyeing certain improvements to the VAERS, including an automated adverse event monitoring system, which he said could help find the “contribution” of vaccines to a yet unclear outcome.
