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

Summary

David Friedberg interviews FDA Commissioner Marty Makary during the JPMorgan Healthcare Conference about the 42 reforms announced in his first 10 months. Makary describes moves to compress drug development (one pivotal trial instead of two, less animal testing, Bayesian and continuous trials, priority vouchers with decisions in weeks) and to make US phase 1 trials competitive with China and Australia. He lays out a drug-price agenda built on most-favored-nation pricing, a faster biosimilar pathway, moving more drugs over the counter and cracking down on misleading pharma advertising. The conversation also covers the revised food pyramid, GLP-1s, the reduced core vaccine schedule, AI and wearables guidance, alternative proteins and autism hypotheses.

• FDA cut the default pivotal-trial requirement from two to one, which Makary says saves developers $100-300 million per drug.
• Animal-testing requirements are being reduced in favor of computational models and organ-on-a-chip; Bayesian statistics and continuous trials are now allowed.
• A national priority voucher pilot produced a first approval in 55 days; about 18 products hold vouchers.
• Drug companies agreed to most-favored-nation pricing; the GLP-1 example falls from $1,300 to $149 for the first three months.
• Biosimilar approval path shortened from 5-8 years and about $300 million to roughly 2.5 years, with a flood of biosimilars expected.
• Push to move more drugs over the counter to create shelf-price competition and bypass PBMs; 1,500 ad-enforcement letters sent.
• Food pyramid flipped toward protein and away from refined carbohydrates; nine petroleum-based dyes removed and the GRAS loophole is being closed.
• New CES guidance creates a consumer lane for AI decision support and wearables; core childhood vaccine schedule reduced to about 38 doses.