Supercharging a New FDA: Marty Makary on Science, Power & Patients
All-In PodcastKey Moments
FDA Commissioner Marty Makary discusses reforms, US vs. China biotech race, drug pricing, and food policy.
Key Insights
The FDA is undergoing significant reforms to accelerate drug approval processes and enhance transparency, aiming to regain global biotech leadership.
There's a critical race with China in biotech innovation, necessitating streamlined research and development timelines in the US.
Drug pricing is a major concern, with efforts focused on negotiating lower prices and introducing more biosimilars to increase competition.
The FDA is revising food guidance, emphasizing protein intake and challenging past dogmas that led to increased childhood diabetes and obesity.
Reforms in vaccine scheduling and a move towards evidence-based, less paternalistic medical advice aim to rebuild public trust.
The agency is embracing AI and modern technologies for drug development and post-market surveillance, while also tightening regulations on misleading pharmaceutical advertising and AI medical claims.
REFORMING THE FDA AND RESTORING PUBLIC TRUST
FDA Commissioner Marty Makary outlines a sweeping reform agenda aimed at modernizing the agency, fostering transparency, and rebuilding public trust. His tenure has been marked by 42 major reforms in 10 months, challenging long-standing dogmas and operational inefficiencies. A key focus is shifting from prioritizing censorship to delivering cures faster and ensuring healthier food options for children. This includes making rejection letters public for accountability and moving beyond outdated practices that stifle innovation and public confidence.
ACCELERATING BIOTECH INNOVATION AMIDST GLOBAL COMPETITION
Makary emphasizes the critical 'race' with China in biotechnology, noting the US was falling behind due to red tape and slow processes. The FDA is implementing changes to expedite drug development, particularly Phase 1 trials, by addressing lengthy IRB approvals and streamlining hospital contracting. Centralized IRBs and faster contracting are crucial to compete with countries that can complete Phase 1 trials in weeks. Modernizing the agency with AI and reducing regulatory burdens on Investigational New Drug (IND) applications are also priorities to ensure US competitiveness.
STREAMLINING DRUG APPROVAL AND REDUCING TIMELINES
The FDA is actively working to shorten drug approval timelines, a process that historically took 10-12 years. Reforms include reducing the default requirement for two pivotal trials to one, which significantly cuts costs and time for companies. The agency is also minimizing animal testing, adopting modern techniques like computational modeling and 'organ on a chip' technology. New pilot programs aim to provide decisions in weeks, not months, prioritizing drugs for unmet public health needs, those manufactured in the US, or those made affordable, demonstrating a commitment to faster patient access.
RETHINKING FOOD GUIDANCE AND COMBATING CHRONIC DISEASE
Makary criticizes decades of 'corrupted' nutrition science that led to flawed food pyramids, blaming industry influence for promoting refined carbohydrates over healthy fats. This has contributed to a surge in childhood pre-diabetes and diabetes. The FDA's new guidance flips the food pyramid, emphasizing protein intake and the importance of 'real food'. The focus is shifting from the 'boogeyman of saturated fat' to addressing root causes like high sugar intake, promoting whole grains, and better farming practices. This represents a significant departure from past dogmas.
ADDRESSING DRUG PRICING AND INCREASING ACCESS
Drug pricing in the US is significantly higher than in other developed nations. The administration aims to secure 'most favored nation status' pricing, which would drastically lower costs for Americans. Efforts are also focused on cutting FDA red tape for biologics and their generic versions, biosimilars, to encourage competition and reduce prices. This includes shortening approval times for biosimilars and moving more drugs to over-the-counter status to increase price transparency and bypass Pharmacy Benefit Managers (PBMs) and their 'money games'.
MODERNIZING VACCINATION SCHEDULES AND MEDICAL PATERNALISM
The FDA has reviewed international vaccine schedules, identifying the US as an outlier with a high number of recommended doses for children. A new approach proposes a 'core essential vaccines' list to rebuild trust and potentially increase vaccination rates. Makary criticizes medical paternalism, using examples like a newborn Hepatitis B vaccine when not indicated, or doctors resisting ultrasounds instead of mammograms. The goal is to engage with patients, acknowledge their concerns, and move towards more evidence-based, less absolutist medical advice.
HARNESSING AI AND MODERN TECHNOLOGIES FOR HEALTHCARE
The FDA is actively developing guidances for AI in healthcare, distinguishing between AI decision support tools and those making medical claims. Wearable devices will see deregulation to improve accessibility and lower prices, provided they meet validated standards. The agency is also exploring advanced statistical methods like Bayesian statistics for continuous trials and real-time monitoring to improve safety and efficacy assessments, moving away from outdated, lengthy submission processes. This embrace of technology aims to accelerate discovery and improve patient outcomes.
REFORMING RESEARCH FUNDING AND SHIFTING PRIORITIES
Contrary to perceptions of being 'anti-science,' the administration has not cut NIH funding but is reallocating it to focus on root causes of disease, such as food, sleep, and environmental exposures, rather than solely on genetics or chemotherapy. Significant funding shifts away from DEI research are noted. The NIH's historical focus, including funding related to the Wuhan lab, is questioned. The goal is to direct resources toward areas like the microbiome and gut health, vital for understanding chronic disease and conditions like autism, in line with objective scientific inquiry.
DIALOGUE ON AUTISM AND THE MICROBIOME FRONTIER
The rise in autism diagnoses is acknowledged, with hypotheses pointing to environmental triggers and immune responses. Makary discusses the potential role of the microbiome in neural development and mood regulation, highlighting how modern lifestyles and medical practices can disrupt it. The focus is shifting towards studying these complex interactions and exploring treatments like leucovorin that bypass blocked receptors, emphasizing the need for research into causes rather than just treatments, and moving away from a purely genetic focus.
REGULATING ALTERNATIVE PROTEINS AND FOOD SAFETY
The FDA is reforming the GRAS (Generally Recognized As Safe) system, which has been abused to allow numerous unvetted chemicals into the food supply. The new approach will be more in line with international standards, requiring a demonstration of safety before introduction. This aims to eliminate the use of petroleum-based dyes and reduce the number of ingredients in ultra-processed foods. The agency also supports the evolution of cellular agriculture and alternative proteins, recognizing their potential for lower cost, reduced cruelty, and improved nutritional profiles, while ensuring consumer safety.
TACKLING PHARMACEUTICAL ADVERTISING AND MISLEADING CLAIMS
Makary addresses the issue of pharmaceutical advertising, particularly for expensive biologics, which can create demand for unindicated or unnecessary medications. The FDA is cracking down on misleading impressions and ensuring 'fair balance' in ads, closing loopholes that allow side effects to be relegated to websites. Enforcement letters have dramatically increased. The aim is to promote 'fair speech' and encourage pharmaceutical companies to redirect some marketing funds towards lowering drug prices, ensuring patients receive accurate information to make informed decisions.
VISION FOR THE FUTURE OF SCIENTIFIC FRONTIERS
Looking ahead, Makary expresses optimism about the pipeline of innovative treatments. Key areas of interest include cures for type 1 diabetes, effective treatments for ALS and PTSD, and universal flu vaccines. The FDA's role as a 'referee' is to evaluate emerging technologies like gene therapy and monoclonal antibodies fairly. The agency is committed to streamlining processes for groundbreaking treatments, such as a gene therapy for congenital deafness and new multiple myeloma therapies, issuing priority vouchers to accelerate their path to patients and emphasizing that regulatory speed must match patient needs.
Mentioned in This Episode
●Supplements
●Tools & Products
●Books
●Studies Cited
●People Referenced
Common Questions
Marty Makary's involvement with the FDA began with his book on hospital price transparency, which led to invitations to the White House during the Trump administration. After contributing to policy changes, he was offered the role of FDA Commissioner.
Topics
Mentioned in this video
A major public health crisis discussed in relation to medical dogma and policy.
Mentioned as a tool to modernize the FDA and speed up application reviews.
A class of therapeutics used for oncology and potentially autoimmune conditions, with high costs.
Entities involved in drug pricing negotiations, criticized for 'money games'.
An example of a scientist whose research on ulcers was initially rejected but later proven correct.
Former director of NIH whose approach Makary contrasts with his own.
Mentioned in the context of NIH funding for coronavirus research, compared to funding for microbiome research.
A society holding a conference where an abstract about a new multiple myeloma treatment was presented.
The political administration during which Makary was invited to the White House and later offered his role.
Boards that review and approve research, with Makary highlighting delays in the process.
Described a novel pathway for bespoke therapies in the New England Journal of Medicine.
A well-known biologic drug where biosimilar competition has been slow to reduce prices.
Discussed as a critical frontier in health, involved in mood regulation and potentially autism.
Former director of NIAID whose approach Makary contrasts with his own.
An institution where Marty Makary has faculty tenure.
Makary's clinical practice specialty at Johns Hopkins.
A pharmaceutical company involved in a major licensing deal with 3S Bio.
Legislation allowing patients access to investigational drugs.
A policy to ensure the US gets the best drug prices in the developed world.
Generic versions of biologic drugs designed to be structurally similar and reduce costs.
Cereal example used to illustrate differences in food ingredient regulations between the US and Canada.
A national effort led by Makary to increase transparency in hospital pricing.
The registration process for new drugs before clinical trials.
Therapies for rare diseases where manufacturing requirements are being customized.
A treatment discussed for autism patients, with some debate over its data.
A disease for which Makary hopes to see a powerful treatment developed.
A university where cell and gene therapies can be developed.
A major global biotech and healthcare conference attended by the commissioner.
Mentioned as an ally in fighting against school closures during the COVID-19 pandemic.
A drug that caused significant harm, highlighting the need for post-market surveillance.
Bacteria historically linked to ulcers, initially met with skepticism.
An agency involved in healthcare pricing, working with the FDA on drug pricing policies.
Event where new guidances on AI decision support and wearables were outlined.
A condition Makary hopes to see powerful treatments for, especially among veterans.
A vaccine that was taken off the market in 1999 due to safety concerns.
A medical journal where Dr. Vani Prasad described a novel pathway for therapies and where a gene therapy for deafness was published.
A statistical method that the FDA is now allowing to be used for drug evaluation.
Mentioned in relation to an approval for shipping American cells to China for CARTT therapy.
Mentioned in relation to CMS and the effort to implement most favored nation status pricing.
Tools being used for diagnosis and providing health information, with regulatory considerations.
Cancer treatments that have shown success in shrinking tumors without surgery or chemotherapy.
The academic institution where Marty Makary previously served on the faculty.
A Chinese company that had a significant licensing deal with Pfizer.
Discussed as an example of a public health crisis that should have been identified sooner through big data.
A type of drug discussed in relation to high prices in the US and potential benefits beyond weight loss.
A class of medications with high costs, where biosimilars are being developed to increase competition.
A system for self-declaring food ingredients as safe, which has been abused.
A disease for which Makary hopes to see a cure or meaningful treatment during his term.
A desired development for lifelong protection against influenza strains.
Vice chair of surgery at Columbia University and a doctor on the core essential vaccine list committee.
Author who questioned the senior author of the Minnesota Heart Study about suppressed results.
Cancer treatments that have shown success in shrinking tumors without surgery or chemotherapy.
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