A Conversation with FDA Commissioner Scott Gottlieb

• Historical Trivia Context: In October 1961, the FDA and the American Medical Association hosted a National Congress aimed at eliminating "quackery" as a major U.S. health problem.
• Case Study on Quackery: The discussion highlighted a specific historical example of quackery involving a micro-diameter device that falsely claimed to diagnose conditions such as cancer, diabetes, and leukemia by measuring electrical currents.

Scott Gottlieb's Background and Regulatory Philosophy

• Gottlieb cites his prior experience at the FDA, investment banking at Alex Brown, and role as a venture investor as critical to his current decision-making.
• He argues that the private sector experience allows him to prioritize "time cost of capital" and risk reduction as primary drivers in product development, not just direct development costs.
• His policy focus includes reducing review cycles and issuing clearer guidance to help innovators de-risk early-stage development programs.

Regulatory Scope and Anecdotal Examples

• The FDA regulates a vast portfolio including foods, drugs, biologics, medical devices, radiation-emitting electronics, cosmetics, veterinary products, and tobacco.
• Gottlieb noted the unexpected burden of regulating "baby turtles," specifically a rule requiring shell diameters greater than 1.5 centimeters to be bleachable to prevent Salmonella transmission, which impacted the Louisiana baby turtle export industry.

Impact of Hurricane María on Puerto Rico's Medical Manufacturing

• A significant portion of U.S. medical product manufacturing is located in Puerto Rico, representing 30% of the island's GDP and employing approximately 100,000 people.
• The region produces over 10% of all drugs consumed in the U.S., including biologics and sophisticated devices like insulin pumps, pacemakers, and cardiac defibrillators.
• Following the hurricane, manufacturing facilities faced critical power outages, with many operating on generators and producing at 20% to 70% capacity.
• Gottlieb warned of a potential for multiple product shortages if facilities do not return to the electrical grid by the first quarter of 2018.
• He praised the "corporate citizenship" of medical companies, which shipped thousands of generators and tons of aid to employees and local communities.

FDA Strategy on the Opioid Epidemic

• The agency is focusing on reducing new addiction cases by limiting the duration of prescriptions (e.g., eliminating 30-day prescriptions for tooth extractions) and packaging opioids in smaller, 2-to-6-day doses.
• FDA expanded risk management and educational requirements from extended-release formulations (10% of the market) to immediate-release formulations (90% of the market).
• In a precedent-setting action, the FDA recommended the withdrawal of Opana ER solely due to its high risk of illicit use and diversion via injection.
• To combat the shift toward heroin and fentanyl, the FDA tripled the number of inspectors in International Mail Facilities (IMFs), quadrupled package inspections, and expanded cyber crimes units targeting the dark web.
• The agency is developing specifications for generic abuse-deterrent formulations and creating incentives for Medication-Assisted Treatment (MAT) products.

Comprehensive Tobacco and Nicotine Regulation

• The FDA announced a new plan to render combustible cigarettes minimally non-addictive by regulating and reducing nicotine content in tobacco leaves.
• The strategy includes extending application deadlines for non-combustible nicotine delivery systems (specifically e-cigarettes) to allow for product innovation.
• While extending deadlines, the FDA committed to enforcing existing regulations on e-cigarettes regarding access for minors and labeling requirements.
• New regulations are being developed to address e-cigarette features, including battery specifications and marketing strategies for appealing flavors.
• A third pillar of the strategy involves creating a regulatory pathway for medicinal forms of nicotine (patches, gums) to encourage product innovation and provide alternatives for adults.

Balancing Safety, Efficacy, and Post-Market Surveillance

• Gottlieb affirmed the necessity of an assurance of both safety and efficacy, noting that patients may miss effective treatment windows if ineffective therapies are not vetted.
• The agency is increasingly relying on post-market data to assess effectiveness, particularly for low-risk technologies like digital health tools and in areas of unmet medical need.
• Recent enforcement actions have targeted cell-based regenerative medicine providers for marketing ineffective products that caused direct patient harm.

Emerging Technologies and Regulatory Reimagining

• The FDA is addressing the rise of digital health tools, artificial intelligence (AI), and cell-based therapies, noting that traditional regulatory paradigms often do not fit these novel technologies.
• A specific example cited was a Stanford-developed AI algorithm capable of distinguishing benign from malignant melanoma, matching the performance of 21 board-certified dermatologists while scanning 200 times faster.
• Gottlieb introduced a "firm-based approach" (pre-certification) for low-risk digital health tools, where the FDA certifies a company's validation processes rather than approving each software update individually.
• The agency is seeking Congress to clarify authority regarding laboratory-developed tests and other areas requiring new regulatory frameworks for novel technologies.