659. Can Marty Makary Fix the F.D.A.?

Key Takeaways

The FDA under Commissioner Marty Makary is shifting to a proactive, deregulatory approach.
Significant policy overhauls include reforming the food pyramid and eliminating animal testing requirements.
The agency aims for faster drug approvals and ambitious goals for disease cures.
Efforts are targeting industry influence, deceptive pharmaceutical advertising, and medical 'patterns of dogma'.
New AI tools are being implemented to modernize FDA review processes and enhance efficiency.
The FDA contends with challenges from the politicization of science and media misreporting on health funding.

The second Trump administration initiated public health policy changes, including overhauls to childhood vaccine recommendations and the food pyramid.
The FDA approved new treatments for rare diseases, cancers, rheumatoid arthritis, HIV, and a non-opioid pain medicine.
FDA Commissioner Marty Makary announced the agency will proactively identify and advance promising treatments, moving away from a passive role.
Makary's approach involves challenging assumptions and diagnosing issues before treatment, referencing 'patterns of dogma'.

Commissioner Makary identifies pricing failures and inappropriate care as high priorities for the FDA.
A new FDA initiative offers priority review for companies that commit to drug prices comparable to other developed nations.
This priority review voucher is valued at approximately $500 million as an incentive.
The FDA advocates for deregulatory policies to reduce red tape and aims to shorten the 10-12 year drug approval process.

The FDA's modernization priorities include integrating AI, removing petroleum-based food dyes, and reforming the food pyramid.
Animal testing requirements are being eliminated, with big data used for post-approval drug monitoring.
Application review times are targeted for reduction from a year to weeks by consolidating reviewing offices.
The FDA launched ELSA, a generative AI tool for scientific reviewers, agency-wide ahead of schedule and under budget, with thousands of reviewers using it voluntarily.

The FDA commissioner outlines ambitious goals for the next year, including cures for type 1 diabetes, certain cancers, and neurodegenerative disorders.
Goals also encompass a universal, long-lasting flu shot and expedited treatments for PTSD.
The FDA proactively reaches out to companies with promising early-stage treatments, such as lab-grown pancreas cells for type 1 diabetes.
This approach aims to facilitate development without compromising safety standards, as demonstrated by early access to gene therapy for a baby with a rare genetic defect.

In September, the FDA announced progress on autism research, including a proposed label change for leucovorin as a treatment.
Marty Makary states that leucovorin has sufficient evidence for potential use in treating autism, particularly for children with an antibody blocking the folate receptor.
Leucovorin is a century-old generic drug with a strong safety record, previously used in cancer care.
The NIH will fund studies to track its use; current observations suggest 20% of children show significant improvement, with another 30-40% showing some benefit.

Evidence is mixed regarding a link between prenatal acetaminophen (Tylenol) use and autism, with 27 studies suggesting an association versus 13-14 finding no association.
Despite an FDA 'Dear Doctor' letter stating no causal link is established, media headlines and President Trump's statements have created confusion.
President Trump directed the FDA to target deceptive direct-to-consumer (DTC) pharmaceutical ads, noting that the U.S. and New Zealand are among the few countries allowing extensive DTC advertising.
The FDA ensures that advertising claims are supported by clinical data and are not misleading, citing examples of ads with no spoken words or side effects.

The FDA is recommending the use of organoids, or 'organs on a chip,' for drug testing, which is more predictive for humans than traditional animal testing.
Approximately 90% of drugs passing animal tests fail human trials, highlighting the limitations of current methods.
The FDA plans to eliminate animal testing requirements in stages, beginning with monoclonal antibodies, for which an average test uses 144 chimpanzees.
The agency emphasizes its role as an independent scientific organization that follows the law, as demonstrated by the recent approval of a generic version of mifepristone.

Media reports on government spending cuts for health programs are often described as dishonest by the guest.
The Medicaid program budget has reportedly increased, and the NIH budget is projected to increase, contrary to some media reports.
However, a counterpoint notes that the Trump administration's proposed budget cuts to Medicaid (though rejected by Congress) and implemented cuts to NIH grants did disrupt clinical trials for cancer and heart disease.
Commissioner Makary aligns with HHS Secretary Bobby Kennedy Jr. on the importance of allowing questions and studying hypotheses for scientific progress.

The guest criticizes the politicization of science, particularly during the COVID-19 era, where the Democratic Party labeled itself as the 'party of science'.
This led to the marginalization of dissenting views and a concept of 'scientific apartheid'.
The guest advocates for scientific objectivity, highlighting how individuals from diverse political backgrounds united to question policies like prolonged school closures and the dismissal of natural immunity.
The FDA commissioner expresses enthusiasm for accelerating new cures and treatments, prioritizing applications from companies manufacturing in the U.S. that address unmet public health needs and offer lower prices.