A few days ago I highlighted a new paper by Vinay Prasad on "surrogate endpoints" - here.
Today, June 10, Markary and Prasad publish a new paper, in JAMA, called, "PRIORITIES FOR A NEW FDA."
Find it at JAMA here, open access:
https://jamanetwork.com/journals/jama/fullarticle/2835314
The use of AI in FDA reviews was rapidly picked up by NYT:
https://www.nytimes.com/2025/06/10/health/fda-drug-approvals-artificial-intelligence.html
At Genomeweb, Turna Ray discusses whether the FDA priorities leave space for precision medicine:
### OPENING OF FDA ESSAY
Why does it take more than 10 years for a new drug to come to market? Why are childhood chronic diseases so prevalent? And how can regulators adapt to meet the challenges facing clinicians today? These questions are at the forefront for the US Food and Drug Administration (FDA).
The US leads the world in sophisticated cell and gene therapies and other innovative treatments, but in terms of the health of the population, our medical system has been a 50-year failure....Obesity, diabetes, colon cancer, depression, and autoimmune diseases have increased in young people, and early-onset Alzheimer disease has increased by at least 300%. ...At the FDA, we will examine the role of ultraprocessed foods, food additives, and environmental toxins, the introduction of which has paralleled the epidemic of chronic diseases.
###
Here's a Chat GPT summary -
For Health Policy Experts — Summary of FDA Vision (Makary, JAMA, June 2025)
The FDA under Martin Makary presents a reformist vision aimed at addressing systemic failures in U.S. population health despite biomedical innovation. The strategy prioritizes faster therapeutic access, root-cause prevention, and regulatory modernization, emphasizing science-based pragmatism.
1. U.S. Health Outcomes: Innovation–Prevention Gap
-
U.S. leads in cell/gene therapies but lags in public health: 40% of children have chronic illness, 1 in 6 neurodevelopmental disorder, life expectancy plateaued or falling.
-
Rise in obesity, autoimmune diseases, early-onset Alzheimer’s not explained by genetics—points to environmental and dietary causes.
2. FDA Priorities: A Reoriented Mission
A. Accelerating Cures
-
COVID showed fast-track reviews are possible; pilot pathways will aim for weeks, not years.
-
Pre-submission paperwork review to reduce post-trial approval lag.
-
Target: Reduce delays in areas like infant formula innovation, outdated monographs, and redundant animal testing.
B. Regulatory Culture Shift
-
FDA will partner with industry for efficiency—especially for small firms—while avoiding regulatory capture.
-
Recent moves: Removed industry members from non-statutory advisory committees; ended COI waivers in VRBPAC.
3. AI Integration
-
Launched first generative AI pilot in May 2025 to support pre-review of massive application documents (>500,000 pages).
-
AI to modernize device/software reviews, especially in dynamic AI/ML domains.
-
Computational modeling (e.g., organs-on-chip) will aid in toxicity prediction and reduce animal testing.
4. Food as a Public Health Priority
-
FDA will now emphasize the “F”: food regulation.
-
Actions already underway:
-
Removal of 9 petroleum-based dyes.
-
Inventory of ingredients banned abroad but common in U.S. (e.g., talc in meds and candy).
-
Early definition and labeling standards for ultraprocessed foods.
-
5. Big Data as Regulatory Infrastructure
-
Shift from sole reliance on RCTs to causal inference from real-world data (e.g., target trials).
-
Use of postmarket surveillance to assess safety and real-world effectiveness, especially in rare diseases.
-
Potential to move from “2 pivotal trials” standard to 1 trial + real-world data.
6. Financial Toxicity
-
U.S. drug pricing is a global outlier. Though FDA cannot regulate price, it will:
-
Expedite generics and biosimilars.
-
Support most-favored-nation pricing.
-
Highlight excessive marketing vs. R&D spend in pharma.
-
Bottom Line:
The FDA plans to rebalance from reactive drug policing toward proactive health stewardship, including regulatory agility, food safety, and equitable access. A hybrid model of fast-tracked innovation, real-world validation, and conflict-free science is at the core of Makary’s FDA transformation.
###
###
Here's what Chat GPT makes of the NYT article:
The New York Times presents the FDA’s proposal to expand AI use—especially to speed drug and device approvals—as a bold but controversial initiative.
While Commissioner Dr. Marty Makary claims AI will “radically increase efficiency,” the article underscores widespread skepticism. Experts caution that current AI tools like the FDA’s new model “Elsa” still hallucinate, save little time, and must be checked closely. Critics also raise concerns that the AI push, combined with staff cuts and cozy industry ties, reflects Trump-era deregulatory ambitions more than true innovation. Overall, the Times casts the plan as aspirational but unproven, and potentially vulnerable to political and scientific backlash.
