Interview, Fireside Chat, Conference Presentation
Supercharging a New FDA: Marty Makary on Science, Power & Patients
- The FDA aims to restore public trust by challenging historical scientific paternalism and implementing transparency measures, including making rejection letters public after decades of discussion.
- To combat the projected rise of China's global biotechnology licensing share from 5% in 2022 to 42% in 2025, the agency plans a three-year strategic window to accelerate US competitiveness.
- Drug development timelines are targeted for reduction from the current 10 to 12-year status quo through priority reviews, the elimination of the baseline two-pivotal-trial requirement, and a shift toward one pivotal trial for equivalent statistical power.
- Early-stage clinical processes face aggressive streamlining, with goals to match China's four-week and Australia's six-week Phase I trial timelines via centralized Institutional Review Boards and hospital contracting.
- Application completeness confirmation is projected to drop from 60 days to one day using artificial intelligence, while the biosimilar approval timeline is targeted to shrink from five to eight years down to 2.5 years.
- The agency intends to hire 1,050 new scientists and consolidate HR, procurement, and IT functions to return staffing levels to 2019 numbers without laying off scientific reviewers.
- Operational efficiency is set to improve through internal incentive restructuring to eliminate end-of-cycle reporting bottlenecks and the centralization of services across seven siloed centers.
- User fee targets project 100% achievement by 2025, ensuring all accept or reject decisions align with primary review team recommendations, alongside a tiered fee structure favoring US-conducted Phase I trials over overseas ones.
- Drug pricing strategies include a "most favored nation" approach to lower costs and a specific projection for GLP-1 drug prices to reach $149 for the first three months.
- Biosimilar approval timelines and over-the-counter transitions are expected to lower consumer prices through increased competition and price transparency, with more prescription drugs moving to non-prescription status based on safety and lack of abuse potential.
- Regulatory frameworks for novel therapies will utilize the "plausible mechanism pathway" to bypass traditional randomized trials for rare infant diseases and transition toward continuous Bayesian trials for real-time safety evaluations.
- Manufacturing requirements for cell and gene therapies will be customized to remove mandatory multiple batch runs, aiming to reduce costs and accelerate patient access.
- Testing protocols will evolve to eliminate chimpanzee studies for monoclonal antibodies and replace animal testing with computational modeling and "organ on a chip" technology to improve prediction accuracy.
- Post-market surveillance will leverage big data to identify safety signals immediately after approval to prevent adverse events similar to Vioxx or the opioid epidemic.
- Enforcement actions against misleading pharmaceutical advertising will continue, with a projection of high issuance rates for enforcement letters and cease and desist orders similar to the 1,500 issued previously.
- The GRAS loophole for new food chemicals will be closed, shifting the US regulatory standard to "guilty until proven innocent," while wearables will be deregulated unless they make clinical medical claims requiring medical-grade validation.
- Dietary guidance has been updated to prioritize protein over saturated fats and refined carbohydrates, with plans to launch realfood.gov and reallocate NIH funding from genetic research to root causes like food as medicine and the microbiome.
- NIH funding allocation is planned to shift away from the 14% previously designated for DEI research toward studies on disease causes, including insulin resistance and inflammation.
- A core list of 38 essential vaccines will be established from the 72 currently recommended to rebuild trust, while maintaining all current recommendations to ensure critical protections.
- Vaccine discourse is intended to be restored to allow open debate on safety and efficacy without fear of professional censure, alongside a goal to eliminate state-level bans on cellular meat despite the FDA lacking jurisdiction over such bans.
- Research into the link between the microbiome and autism will focus on factors such as antibiotic overuse, noting that the average two-year-old has received over 2.5 courses of antibiotics.
- Specific treatment goals for the administration term include a cure or meaningful treatment for Type 1 diabetes, powerful ALS treatments, and treatments for certain cancers eliminating surgery, chemotherapy, or radiation.
- Other therapeutic targets include a universal flu shot for lifelong protection, a powerful PTSD treatment to address veteran suicides, and priority vouchers for treatments like those for congenital deafness and multiple myeloma.
- The agency aims to remove all nine artificial petroleum-based dyes from the food supply and plans to issue priority vouchers for 18 products meeting criteria for unmet public health needs, US manufacturing, or affordable pricing.
- The FDA has signed 100% of Right to Try requests while committing to prevent patients from spending money on ineffective products, and plans to increase funding for hormone replacement therapy research to reverse previous cancer risk narratives.