A Conversation with FDA Commissioner Robert Califf | Future of Health Summit 2023

Current Health Metrics and Inequities

• U.S. life expectancy has declined to 76.4 years, marking the lowest level in over two decades.
• The United States spends the most on healthcare among high-income nations yet records the worst health outcomes.
• A study of three counties along Lake Erie revealed significant life expectancy disparities despite similar demographics, driven entirely by differing state health policies.
• Tobacco and hypertension remain the most significant remediable predictors of death in the U.S.
• Approximately 460,000 Americans are projected to die from tobacco-related illness this year, with vaping driving new nicotine addiction among teenagers.

FDA Mission and Post-Market Evidence Generation

• FDA Commissioner Robert Califf argues the agency excels at pre-market innovation (Phase 1-3 trials) but lacks a system to generate post-market evidence.
• Critical questions regarding optimal dosage, subpopulation benefits, therapy combinations, and treatment duration often remain unanswered after FDA approval.
• The current fragmented U.S. approach contrasts with real-time electronic health record systems in Israel and rapid clinical trial integration in the UK.
• Califf calls for a unified ecosystem involving CMS, NIH, and FDA to create a coordinated post-market evidence generation framework.
• Proposed CMS actions include incentivizing clinicians to participate in research on a grand scale.
• NIH portfolio adjustments are suggested to direct academic medical centers toward clinical studies that improve health outcomes.

Strategies for Misinformation and Public Trust

• Trust in public health institutions and science has not improved since the COVID-19 pandemic.
• The FDA plans to prioritize communicating in plain English and Spanish to bridge gaps between agency jargon and public understanding.
• Commissioner Califf advises against viewing misinformation fighting as a solo government effort due to limited budgets and the 24/7 nature of disinformation campaigns.
• A proposed solution involves creating a coalition of universities, medical centers, industry, and voluntary foundations to promote accurate health information.
• Effective counter-messaging requires a high "share of voice" that matches the frequency of misinformation and relies on personal, community-based interactions.

Real-World Data (RWD) and Real-World Evidence (RWE)

• RWD is not inherently "sloppy"; it has been used for decades in FDA-regulated trials, such as early heart attack studies involving tens of thousands of patients.
• RWE is defined by the rigorous methodology applied to data, not the data source itself.
• Califf emphasizes that randomization remains the gold standard for establishing causality, even when utilizing real-world data.
• A major systemic failure identified is the inability to track patient data over a lifetime without paying premium fees to data aggregators.
• AI algorithms for healthcare prediction require constant monitoring and tuning to remain valid as health systems evolve.
• The FDA will not trust AI models developed in isolation without a transparent system for continuous performance monitoring.

The Role of AI in Healthcare Operations

• Generative AI is viewed as a tool to reduce administrative burden, potentially freeing clinicians to focus on patient interaction.
• Current clinical workflows are described as disastrous, with physicians spending excessive time on billing forms and Electronic Health Record (EHR) corrections.
• Future AI applications aim to automate non-clinical tasks, restoring the physician-patient relationship.
• Califf notes that while AI is promising, the regulatory landscape for generative AI is complex and requires specific discussion separate from predictive AI.