Jan 6, 2020, 11:00 AM ETFireside Chat, Interview

Food, Drugs, and Tech: 100 years of Public Health

The FDA, established in 1906 during an earlier Industrial Revolution, remains the largest consumer protection agency, formed from 100 laws to address hygienic conditions in Chicago stockyards.
The agency's core mission to protect public health through safe, effective medical products and the promotion of innovation is argued to have increased rather than decreased relevance in the era of rapidly emerging biology.
Current regulatory responsibilities cover approximately 20% of international GDP, including food, drugs, biologics, devices, animal products, cosmetics, and nicotine-based products.
Unlike past eras where data arrived via paper ("truckloads"), the FDA is transitioning from legacy physical archives to structured digital data to enable large-scale review and continuous surveillance.

Despite modernization efforts, many applications still arrive as PDFs or "large digital representations of paper," including recent approvals for orphan diseases received on physical paper.
The agency is implementing "Smarter Food Safety" initiatives, piloting machine learning algorithms to replace a 10-year-old rules engine (PREDICT) for predicting unsafe food imports at borders.
A new experiment is underway to utilize machine learning for inspecting trucks to improve safety efficiency without causing traffic bottlenecks.
The FDA has developed a dedicated staff to avert drug shortages, successfully preventing over 160 shortages in 2018 despite there being more than 50 active shortages that year.
Foodborne outbreak detection has shifted to whole genome sequencing, allowing the FDA and CDC to trace pathogens like Listeria from individual consumers back to specific manufacturing facilities.

Regulatory culture must balance extreme risk aversion (e.g., Thalidomide backlash) with the need to approve life-saving innovations through flexible, risk-based scientific decision-making.
During the opioid crisis, the FDA executed an action plan focusing on its specific authorities: reducing opioid tablet access post-surgery, increasing naloxone availability, and developing new treatments for pain and addiction.
The agency emphasizes "right-sizing" review expectations based on safety risks, population impact, public perception, and the ability to de-risk via manufacturing preconditions or toxicity data.
The FDA is moving away from traditional "N of thousands" trials toward "N of 1" frameworks for highly individualized therapies like gene editing and CAR T-cell therapies.
To manage individualized therapies, the FDA is establishing frameworks that assess biological plausibility, in vivo/in vitro safety, manufacturing consistency, and ethical considerations.

The FDA distinguishes between two algorithm categories: Software as a Medical Device (acting as treatment) and support software; for the former, they are developing preconditions to allow update cycles that do not require full regulatory review.
Pilot projects are testing "preconditions" for software updates to ensure quality controls and performance expectations are met without hindering rapid iterative development.
Regarding DIY CRISPR kits sold online, the FDA notes its authority is limited to products making commercial medical claims; individual private use falls into a "fuzzy area" of privacy and rights rather than direct regulatory approval.
FDA CIO leadership is advocating for a Chief Privacy role to address re-identification risks in "de-identified" data, acknowledging that longitudinal health patterns can uniquely identify individuals despite HIPAA standards.
The agency is exploring synthetic data and new tools to preserve privacy while enabling data sharing for drug surveillance and research.

Traditional clinical trial phases are blurring, with some drugs approved based on Phase 1 data containing thousands of patients (expansion cohorts) rather than the traditional small sample sizes.
The FDA is exploring "platform trials" to evaluate multiple investigational products simultaneously within a single infrastructure, though contractual and intellectual property hurdles remain.
The 21st Century Cures Act (signed December 2016) serves as a key legislative accelerant, enabling the use of surrogate endpoints, patient-reported outcomes, and real-world evidence (RWE).
Real-world evidence is increasingly utilized to create a "totality of evidence" for approved products, supplementing traditional prospective trials with retrospective and prospective data.
Historical context shows that major regulatory shifts often align with broader political or economic events, such as the 2008 Global Financial Crisis which catalyzed the HITECH Act for electronic health records.

The FDA and USDA finalized agreements delineating responsibilities for cell-cultured foods, with the FDA regulating the cell culture process and the USDA overseeing marketing and labeling.
New regulatory paradigms are being developed to address labeling consistency for alternative protein products (e.g., almond milk, lab-grown meat) to prevent consumer confusion.
Blockchain and distributed ledger technologies are being contemplated to monitor full supply chains, potentially allowing consumers to scan products (e.g., peaches) to verify safety and traceability.
The FDA envisions future longevity therapeutics requiring longitudinal biomarkers to measure preventative efficacy over time, rather than fixed endpoints used in traditional disease treatment.

The future FDA is projected to be fully digital and data-informed, with automated activities freeing personnel to focus on critical decision-making.
Regulatory frameworks will evolve to utilize longitudinal performance data to dynamically right-size product indications over time.
The agency anticipates a shift from treating biological aberrance to preventative approaches that expand healthy life spans, requiring new measurement standards and biomarkers.