Makary says he wants nimbler FDA, calls for new drug pathways and pilots
WASHINGTON – Marty Makary, commissioner of the US Food and Drug Administration (FDA), said he wants the agency to become nimbler in the next five years to adapt to the fast pace of medical innovation. As part of that modernization effort, he also said the agency is exploring new pathways and pilot programs.“I think in five years we'll be measured by how nimble we are, how we have adapted to the modern time, how fast we have changed the agency to adapt to the fast pace of medicine,” Makary said during a panel at the DIA Global Annual Meeting on Monday.
More specifically, Makary said he wants to see the development of a universal influenza vaccine that would include strains of bird flu and would not require annual administration.
Earlier this year, the Department of Health and Human Services (HHS) and the National Institutes of Health (NIH) said it was launching Generation Gold Standard aimed at developing a next-generation universal vaccine platform using a beta-propiolactone (BPL)-inactivated, whole-virus platform, though reactions to the announcement among scientists has been mixed.
Makary also said he wants to see a meaningful treatment for type 1 diabetes and more cell and gene therapy advancements, especially for rare diseases.
“There is essentially a spirit of a plausible mechanism pathway for some of these therapies, and I think we have to continue that flexibility,” said Makary.
Makary stressed that FDA needs question why it takes 10 years for new drugs to come to market. He said the barriers to getting new treatments to patients are often the processes, and the agency needs to be willing to challenge deeply held assumptions about legacy systems used to bring products to market.
“We’re exploring newer pathways for drugs and pilot programs,” said Makary. “You’ll see us put out something on that in the not-too-distant future.”
He also noted that competition is fierce, and everyone wants to see clinical trials and product evaluations in their home countries, and the agency needs to do better when communicating with sponsors.
“We have to be willing to learn from data other than randomized controlled trial data,” said Makary. “We can learn from real-world data. We can learn from synthetic controls now in national giant electronic health records. We never had access to do impeccable patient matching before.”
Makary also mentioned FDA’s recently launched Elsa artificial intelligence (AI) tool that can help reviewers evaluate premarket applications, which can sometimes contain more than 100,000 pages. He said at some point the agency needs to introduce a page limit.
Makary said that companies tend to include everything in their applications, so reviewers don’t come back to ask for additional information.
“At some point, we have to disarm, he added. “We need to recognize that we need core information to establish safety and efficacy, and we should be able to be clear about what that is.”
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