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

Administrative Leadership & Mandate:

• FDA Commissioner Marty McCary, appointed days after President Trump's reelection, has served for 10 months with a primary mission to "deliver more cures and meaningful treatments faster" rather than focus on censorship or misinformation suppression.
• McCary cites a specific goal to rebuild public trust in health institutions, which he argues was eroded by "paternalism" and the suppression of scientific inquiry during the COVID-19 pandemic (e.g., mandates for toddlers, ignoring natural immunity).
• He asserts that the FDA has announced 42 major reforms in his first 10 months, challenging the "status quo" of the 10–12 year drug approval timeline.

FDA Restructuring & Workforce:

• The agency reduced staff from 20,000 to 2019 levels, eliminating 2,000 HR positions and 1,500 IT roles, but confirmed no scientific reviewers or scientists were laid off.
• The FDA achieved a 100% target date compliance rate for user fees in 2025, with a turnover rate maintaining a baseline of 5–7%.
• The agency is currently hiring 1,050 new scientists to support ambitious new accelerated pathways and has centralized previously siloed centers (e.g., ending independent press offices and legislative affairs for each center).

International Competitiveness vs. China:

• Citing a Congressional National Security Commission report, McCary states the U.S. has a three-year window to prevent losing biotech leadership to China, noting Chinese companies rose from 5% to 42% of licensing deals between 2022 and 2025.
• The administration prioritizes streamlining U.S. clinical trial infrastructure to match foreign efficiency: Chinese Phase 1 trials take four weeks, Australian trials six weeks, whereas U.S. trials currently take much longer due to slow Institutional Review Boards (IRBs) and complex hospital contracting.
• Proposed reforms include establishing centralized IRBs and standardized hospital contracts to reduce red tape and accelerate Phase 1 processes.
• McCary announced plans to reduce the FDA's initial application review decision time from 60 days to one day using AI.

Drug Approval & Clinical Trial Reform:

• Pivotal Trials: The FDA is moving from a default requirement of two pivotal trials to a single pivotal trial per drug to reduce costs by $100–$300 million and shorten timelines without sacrificing statistical power.
• Animal Testing: The agency is eliminating requirements for chimpanzee studies for monoclonal antibodies, citing misleading data (90% of drugs passing animal studies fail in humans) and ethical concerns; instead, it is adopting computational modeling and "organ-on-a-chip" technologies.
• New Pilots: A pilot program launched 55 days ago issues priority review vouchers for drugs meeting specific national priorities: unmet public health needs (e.g., tumor shrinkage without surgery), U.S. manufacturing, or affordability.
• Continuous Trials: The FDA is implementing Bayesian statistics and "continuous trials" to allow real-time data analysis and earlier approval or withdrawal, moving away from annual data "cuts" to improve safety signal detection.
• Post-Market Surveillance: The agency plans to utilize big data for real-time monitoring of adverse events post-approval to avoid issues like the Vioxx or opioid epidemic delays in detecting safety risks.

Right to Try & Manufacturing Flexibility:

• McCary has signed 100% of "Right to Try" requests and supports flexible regulatory pathways for patients with terminal conditions.
• Regulatory requirements for cell and gene therapies have been customized, removing strict "Process Performance Qualification" (PPQ) batch run mandates for bespoke therapies where the platform is already proven.
• The FDA shut down a Biden-era initiative to ship U.S. patients' cells to China for gene editing, citing national security and safety concerns.
• The "plausible mechanism pathway" (described by Dr. Vinay Prasad) is being utilized to combine gold-standard science with common sense for rare disease therapies.

Food, Nutrition, & Dietary Guidelines:

• The FDA and White House have officially "flipped the food pyramid upside down," shifting focus from demonizing saturated fats to prioritizing protein and whole foods, citing a 38% pre-diabetes/diabetes rate in children driven by refined carbohydrate diets.
• McCary attributes the rise of chronic disease and obesity to "ultra-processed foods," refined carbohydrates, and the lack of protein in the standard American diet, which leads to insulin resistance and systemic inflammation.
• The agency is moving to close the "GRAS" (Generally Recognized As Safe) loophole, which currently allows chemicals to be added to food without proving safety (innocent until proven guilty), aligning more with international standards.
• The administration has removed the recommendation to delay peanut butter introduction until age three, a policy McCary notes contributed to the modern peanut allergy epidemic, and is advocating for early exposure to build immune tolerance.

Vaccine Schedules & Public Trust:

• The CDC/FDA has released a "core essential vaccine" list comprising 38 doses (down from the U.S. outlier standard of 72 doses from age 0 to 18), aiming to restore trust by distinguishing essential vaccines from recommended ones.
• Hepatitis B administration for newborns is now being reviewed for necessity, particularly when maternal exposure risk is low, to address parental concerns about the aggressive vaccine schedule.
• McCary notes that childhood vaccination rates have dropped due to lost trust in "absolutist" pandemic policies and argues for "meeting people where they are at" rather than enforcing rigid mandates.
• The FDA acknowledges historical vaccine failures (e.g., the 1999 Rotavirus vaccine withdrawal) to counter the narrative that all vaccines are infallible.

Drug Pricing & Access:

• The administration is enforcing "Most Favored Nation" pricing, aiming to lower GLP-1 drug costs in the U.S. to $149 (from $1,300) and secure similar pricing for other biologics by requiring other nations to pay their "fair share" of R&D.
• The FDA has cut biosimilar approval timelines from 5–8 years to 2.5 years and reduced R&D costs by $100+ million per product by aligning biologics approval standards with those for small molecules.
• A major 2026 goal is to move drugs currently requiring prescriptions to Over-The-Counter (OTC) status to increase price transparency and reduce PBM (Pharmacy Benefit Manager) markups.
• The agency is targeting drugs with no abuse potential, low safety risks, and no need for laboratory monitoring for OTC conversion, citing that 60% of antibiotics and 60% of opioids are prescribed unnecessarily.

Advertising & AI Regulation:

• The FDA has issued 1,500 enforcement letters for pharmaceutical advertising, including 100+ cease-and-desist orders, to close the "adequate provision" loophole and ensure fair balance of risk information in ads.
• New guidance on AI separates consumer-grade wearables (deregulated for health metrics) from medical-grade devices (requiring validation), while creating a clear lane for AI decision support tools that do not trigger automated interventions.
• The administration intends to crack down on misleading AI-driven diagnostic claims and online pharmacies advertising drugs without proper side effect disclosures.

Research Funding & NIH Reallocation:

• McCary states the Trump administration has not cut NIH funding; rather, funds are being reallocated from a "myopic" focus on genetic research to root causes like the microbiome, food as medicine, and environmental exposures.
• The administration has removed 14% of NIH grants previously dedicated to Diversity, Equity, and Inclusion (DEI) studies, arguing that describing disparities does not solve them.
• Emphasis is shifting toward studying how environmental factors, sleep quality, and the gut microbiome contribute to diseases like Type 1 diabetes, autoimmune conditions, and autism.

Autism & Microbiome Science:

• Rising autism diagnoses (1 in 12 boys in California) are being investigated as potential autoimmune phenomena where antibodies block folate receptors; treatments like "Lucavorin" (methylated folate) are showing clinical improvement in some cases.
• The agency is prioritizing research into the gut-brain axis, noting that 90% of serotonin is produced in the gut and that the modern microbiome is compromised by C-sections, infant formula, and excessive antibiotic use (average of 2.5 courses by age two).
• McCary highlights a randomized trial using protease for autism as a potential area for further study, emphasizing the need to move beyond genetic cures to address environmental contributors.

Future Scientific Priorities:

• The administration aims for a cure or meaningful treatment for Type 1 Diabetes, ALS, and PTSD (targeting the 7,000+ veteran suicides annually).
• Goals include a universal flu shot providing lifelong protection and the expansion of gene therapies and monoclonal antibodies that eliminate the need for surgery or chemotherapy in cancer.
• The FDA is actively issuing priority review vouchers for novel mechanisms, such as gene therapies for congenital deafness and treatments for multiple myeloma showing 80% remission-free survival.