FDA leaders outline vision for advancing US healthcare through big data, AI and drug pricing reform

FDA Commissioner Marty Makary and CBER Director Vinay Prasad outline strategic priorities for strengthening the US healthcare system, emphasizing the role of real-world data (RWD), AI, and pricing reform to improve outcomes and support innovation.

In a recent JAMA Viewpoint, Marty Makary, the new Commissioner of the US Food and Drug Administration (FDA), and Vinay Prasad, Director of the FDA’s Center for Biologics Evaluation and Research (CBER), outline strategic priorities for strengthening the US healthcare system. Their proposals emphasize the importance of leveraging RWD, adopting AI-driven tools, and implementing drug pricing reform to improve health outcomes, support innovation, and enhance system efficiency.

Makary and Prasad describe the US system as under increasing pressure from rising costs and persistently poor health indicators. They call for “fresh new ideas” to address underlying drivers of disease and inefficiency, advocating for a shift away from reactive care toward more anticipatory and prevention-oriented models. “We will transition from a purely reactionary healthcare system to one that is proactive, intellectually curious about underlying causes, and financially aligned to promote health—not just treat sickness,” they write.

One of their core priorities is the need for a “new FDA” equipped to adapt to evolving scientific, technological, and economic realities. The authors emphasize the expanding role of big data in overcoming well-recognized limitations of traditional randomized controlled trials, including inadequate control arms, poor generalizability, and underpowered study designs. They note that advances in causal inference and the integration of nonrandomized data are enabling more reliable conclusions, while RWD can improve real-time pharmacovigilance and help assess effectiveness in real-world clinical settings. In some cases, they suggest that robust real-world evidence may be sufficient to support regulatory decisions previously dependent on two pivotal trials.

The Viewpoint also advocates for strategic deployment of generative AI across the regulatory lifecycle. Proposed applications from the authors include expediting the review of submissions, enhancing the assessment of AI-based technologies, and using AI-driven computational modeling to reduce reliance on animal testing and accelerate drug development.

On pricing, Makary and Prasad highlight the comparatively high cost of pharmaceuticals in the US and reaffirm support for reforms that link pricing to clinical value. They express commitment to advancing the national priority of most favored-nation pricing, arguing that the US, despite being the largest purchaser of pharmaceutical products globally, continues to pay disproportionately high prices relative to other high-income countries.