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Supercharging a New FDA: Marty Makary on Science, Power & Patients

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Watch on YouTube fda reform drug approval acceleration regulatory modernization vaccine policy nutrition policy pharmaceutical pricing ai in healthcare

FDA Commissioner Marty Makary discusses his ambitious agenda to accelerate drug approvals, reduce regulatory burden, and reform nutrition guidance in the U.S. health system. Makary outlines 42 major FDA reforms implemented in his first ten months, arguing that outdated medical dogma—from vaccine schedules to dietary guidance—has harmed public health and that modernization through AI, alternative trial designs, and reduced animal testing can make American biotech competitive with China while lowering drug costs. The episode covers his philosophy of questioning entrenched institutional practices and restoring public trust in health institutions through transparency and evidence-based policy.

Key takeaways
  • The FDA is reducing drug development timelines by cutting the default requirement from two pivotal trials to one and eliminating unnecessary animal testing, potentially saving companies $100-300 million per drug.
  • Bayesian statistics and continuous trial monitoring with AI tools can enable real-time safety and efficacy signals, allowing FDA decisions in weeks rather than years.
  • The U.S. vaccine schedule at 72 doses is an international outlier; Makary has introduced a list of 38 "core essential vaccines" to rebuild public trust without eliminating any vaccines, as vaccination rates have declined due to pandemic-era mandates.
  • Nutrition science has been corrupted by industry influence and flawed dogma demonizing fats; the new food guidance emphasizes protein and whole foods while removing nine artificial dyes, addressing the fact that 38% of U.S. children have pre-diabetes or diabetes.
  • Most-favored-nation pricing agreements with pharmaceutical companies will reduce drug costs dramatically—GLP-1 drugs dropping from $1,300 to $149, with biosimilar approval timelines cut from 5-8 years to 2.5 years.
  • Moving more medications over-the-counter increases price transparency and competition while bypassing pharmacy benefit manager markups, enabling patients to shop on price without insurance intermediaries.
  • The Right to Try law has been fully implemented; Makary has signed 100% of right-to-try requests and reformed cell and gene therapy manufacturing standards to reduce costs and accelerate access.

Recommendations (5)

"We have organ on a chip technology where you grow say the liver cells or heart cells in a lab administer the drug and then look for any enzyme leaks or disturbances. These are modern techniques."

Marty Makary · ▶ 15:48

"We have computational modeling now. Computer can look at a drug and actually make better predictions"

Marty Makary · ▶ 15:40

Bayesian statistics recommends

"We are now going to allow Bayesian statistics to be used. So now you can if something works instead of having a committee meet twice a year to do a cut of the data"

Marty Makary · ▶ 20:25

Right to try law recommends

"I believe in both the letter and the spirit of right to try. It's an amazing achievement of President Trump in his first term and I have signed 100% of right to try requests that come across my desk."

Marty Makary · ▶ 25:48

CAR-T therapy recommends

"CAR-T is amazing. I'm not here to promote any one class of drugs but I mean the stuff that we have seen is mind-boggling."

Marty Makary · ▶ 29:40

Mentioned (4)

GLP-1 drugs "GLP-1s are mimicking a natural hormone in the body. And so when you get a supplementation of that..." ▶ 43:07
Leucovorin "They have observed that if you give leucovorin as an example which bypasses the blocked receptor ..." ▶ 1:20:15
JAMA
JAMA "Sometimes the studies are not reproducible in JAMA, our most widely circulated medical journal, a..." ▶ 1:23:04
New England Journal of Medicine
New England Journal of Medicine "A treatment came out for a certain type of congenital deafness. It's a gene therapy device combin..." ▶ 1:28:15