FDA Modernization: Accelerating Cures and Correcting Dogma

The FDA under Marty Makary is not merely a gatekeeper but a proactive architect of public health, aiming to accelerate cures and redefine regulatory dogma. This conversation reveals the hidden consequences of entrenched bureaucratic inertia and the profound advantages of challenging conventional wisdom, particularly in drug approvals and food policy. Those who understand this shift will gain an edge by anticipating faster access to novel treatments and a more scientifically grounded approach to public health, moving beyond reactive librarianship to a strategic engagement with innovation.

The Dogma Trap: How Conventional Wisdom Hinders Progress

The FDA, under Commissioner Marty Makary, is embarking on a mission to modernize its approach, moving away from a passive, "receive-only" mode to a proactive stance. This shift is a direct challenge to ingrained patterns of dogma that have historically slowed innovation and, in some cases, led to detrimental public health outcomes. Makary highlights how deeply held, but often scientifically unfounded, beliefs can create significant downstream negative consequences.

A prime example is the modern-day peanut allergy epidemic. The prevailing dogma, championed by organizations like the American Academy of Pediatrics, advised avoiding peanut butter until age three to prevent allergies. This advice, however, ran counter to emerging understandings of immune tolerance. By ignoring laboratory scientists and locking into a rigid dogma, the medical establishment inadvertently fostered a generation of children with increased allergy risks. Makary frames this as a "perpetual vicious cycle," where an initial, well-intentioned but flawed directive created a cascade of negative outcomes.

"The modern day peanut allergy epidemic which didn't exist two generations ago was in part because of a medical dogma magnified by the American Academy of Pediatrics that young kids should avoid peanut butter until they're age three. They thought they were doing the right thing by saying that to prevent a peanut allergy just stay away from peanut products early in life. Turns out they got it backwards."

-- Marty Makary

This pattern of dogma-driven decision-making has also plagued food policy. The long-standing belief that natural saturated fat caused heart disease led to the widespread demonization of fat, resulting in the proliferation of low-fat, high-sugar products. Scientists who pointed to refined carbohydrates and added sugar as the true culprits were largely ignored. Makary emphasizes that this was another instance where "the medical establishment locked arms and walked off a cliff together." The FDA's current effort to rewrite the food pyramid and end the "50-year war on natural saturated fat" is a direct attempt to correct these historical missteps, illustrating how past adherence to flawed dogma created a public health deficit that Makary's FDA aims to rectify.

The OxyContin Catastrophe: When Regulators Become Captives

The approval of OxyContin stands as a stark illustration of regulatory capture, a phenomenon where an agency meant to oversee an industry becomes beholden to it. Makary points to the approval of OxyContin for chronic pain, based on a mere 14-day study, as a "dark point in our history." The subsequent employment of the approving regulator by the company, highlighting the "revolving door" problem, underscores the systemic vulnerability to industry influence.

This capture not only leads to the approval of potentially harmful drugs but also stifles innovation by prioritizing industry interests over public safety. The historical lag in drug approvals, often taking 10-12 years, is a direct consequence of this inertia and the complex, multi-layered bureaucracy that has grown over time. Applications balloon to over 100,000 pages, with companies submitting every conceivable data point to avoid "gotchas," and the FDA responding with more reviewers and more layers of approval. This creates a system where speed and innovation are sacrificed for thoroughness, but not necessarily for better outcomes, as Makary notes: "it's okay we've got a year everyone take your time and get your answers back to us within a year."

"The approval of OxyContin by the F.D.A. represents one of the moments in F.D.A.'s history where it was captured by the industry it was supposed to regulate."

-- Marty Makary

Makary's vision for the FDA is to dismantle this inertia. By making decision letters public, he aims to demystify the review process, holding companies more accountable and providing innovators with clearer guidance. This transparency, coupled with incentives for domestic manufacturing, addressing unmet public health needs, and lowering drug prices, represents a strategic attempt to realign the FDA's incentives with public well-being, moving away from a system susceptible to capture toward one that actively fosters beneficial innovation.

The AI Revolution: Augmenting Human Ingenuity, Not Replacing It

Makary is embracing Artificial Intelligence (AI) not as a futuristic novelty, but as a tool to fundamentally modernize the FDA's operations and regulatory oversight. He acknowledges that the FDA's current regulation of AI technology has been "entirely broken," operating on outdated principles. The challenge, he notes, is not to halt progress out of fear of the unknown, but to adapt regulatory frameworks to the rapid pace of AI development.

The FDA has launched "ELSA," a generative AI tool for its employees, which has already demonstrated significant value in organizing complex applications and ensuring adherence to standards. This initiative is not about replacing human reviewers but about augmenting their capabilities. By providing reviewers with powerful AI tools, the FDA can process information more efficiently, identify potential issues faster, and ultimately accelerate the review process without compromising safety or efficacy. This represents a critical downstream benefit of embracing AI: a more agile and responsive regulatory system.

"We have to move with the times and so we have created a powerful AI tool that our scientific reviewers can use to review applications to organize the applications to provide supporting studies that they can review in depth."

-- Marty Makary

Furthermore, Makary sees AI as crucial for regulating AI itself. The agency must develop frameworks that can adapt to evolving AI technologies, ensuring safety and efficacy without stifling innovation. This proactive approach to AI regulation, coupled with the internal adoption of AI tools, signals a commitment to leveraging cutting-edge technology to achieve the FDA's core mission more effectively. The success of ELSA, developed ahead of schedule and under budget, serves as a tangible example of how embracing new technologies can lead to immediate operational improvements and lay the groundwork for future advancements in drug development and public health.

Actionable Insights for a Modernized FDA

• Immediate Action: Advocate for transparency by demanding public access to FDA decision letters for drug and device approvals. This empowers innovators and holds companies accountable.
• Immediate Action: Support initiatives that incentivize domestic manufacturing of medications, recognizing this as a national security issue that can accelerate review timelines.
• Short-Term Investment (Next 6-12 months): Encourage the adoption of AI tools within research and development teams to streamline data analysis and identify promising early-stage treatments.
• Short-Term Investment (Next 6-12 months): Prioritize research and development of treatments for significant unmet public health needs, such as Type 1 diabetes, neurodegenerative disorders, and PTSD, recognizing these as areas where faster approvals yield substantial societal benefit.
• Medium-Term Investment (12-18 months): Support the development and implementation of more predictive drug testing methods, such as organ-on-a-chip technology, to reduce reliance on animal testing and accelerate the identification of effective human treatments.
• Long-Term Investment (18+ months): Foster a culture of challenging established medical dogma by encouraging rigorous scientific inquiry and open dialogue, even when it conflicts with long-held beliefs.
• Strategic Investment: Recognize that embracing new technologies like AI and advanced testing methods, while potentially disruptive, creates a significant competitive advantage by enabling faster innovation and more effective public health interventions.