Makary, Prasad lay out priorities for FDA: Reduced timelines, more AI, faster competition
The US Food and Drug Administration (FDA) will focus on reducing review timelines for drug development, leveraging technology powered by generative artificial intelligence (AI), and lowering costs using big data and expedited approval of generic medications and biosimilars, agency officials wrote in a recent JAMA Viewpoint article.The agency will also create new pathways for therapeutic and device developers and “rapidly usher to market new products with transformational potential” as part of priorities for a “new FDA,” FDA Commissioner Marty Makary; and Vinay Prasad, director of the Center for Biologics Evaluation and Research, wrote in the article.
“We will transition from a purely reactionary health care system to one that is proactive, intellectually curious about underlying causes, and financially aligned to promote health—not just treat sickness,” Makary and Prasad said.
Revamping review process
Citing the accelerated reviews that occurred during first few years of the COVID-19 pandemic, Makary and Prasad said FDA will develop pilot programs to “deliver sponsors a decision in weeks” rather than in a year. These pilot programs would ask sponsors to submit materials such as manufacturing plans, draft labels, and packaging in advance, giving the agency more time to review these materials before a clinical trial’s completion.
Makary and Prasad recently signaled their interest in bringing products more quickly to market through more streamlined pathways in a roundtable meeting with stakeholders discussing cell and gene therapy (CGT) products. (RELATED: Stakeholders urge FDA to update CGT regulations to ease path to market for promising therapies, Regulatory Focus 9 June 2025)
“The time from when pivotal trial results are known to when decisions are made must be shortened. This has implications for public welfare and will improve the risk-reward calculation of drug development,” they wrote in the article.
FDA intends to become more “user-friendly” and partner with industry when appropriate, such as providing “regulatory navigation” for a small company, while also guarding against a “cozy relationship” with industry, avoiding conflicts of interest, and maintaining independent evaluation of products, Makary and Prasad said.
Generative AI is a “top priority” at FDA, the authors wrote, and the agency will use the technology to do a first-pass review of documentation received by FDA. The agency’s new AI assistant, launched in June, is a part of that initiative, they said. (RELATED: FDA launches agency-wide AI tool ‘Elsa’ ahead of schedule, Regulatory Focus 2 June 2025; RELATED: FDA’s Elsa AI tool gets mixed response from some staff, Regulatory Focus 5 June 2025)
Makary and Prasad also pointed to the FDA’s recent roadmap for reducing the use of animal testing with the aid of AI-based toxicity and cell line computational models and organ-on-a-chip systems as another example of prioritizing AI in modernizing the agency. (RELATED: FDA seeks to reduce animal testing requirements for mAbs, other drugs, Regulatory Focus 11 April 2025)
The FDA officials said there will be a change in how the agency reviews AI-based technologies moving forward. “A diversity of use cases and rapid changes in technology make the legacy review mechanism appear byzantine. Rethinking our approach to AI, balancing safety and accuracy while fueling innovation, is a leading FDA priority,” Makary and Prasad wrote.
Leveraging big data, lowering costs
FDA will leverage big data for post-approval monitoring of products, which the authors said will let the agency “see safety signals in real time and evaluate effectiveness in the real world.” The use of big data analysis through health data and cloud computing has allowed for post-approval monitoring and identification of the “root causes of chronic diseases,” they noted.
“Advances in causal inference in nonrandomized data, including the use of target trials, which attempt to balance confounding and time zero, have potential to yield actionable causal conclusions, in many cases at lower cost,” Makary and Prasad said.
While FDA is not allowed to consider price when evaluating benefit and risk of drugs, “the FDA will use its power to address costs” through streamlining the development of biosimilars and expediting the approval of generic medications, the FDA officials said. They also stated that FDA supports most favored-nation pricing of pharmaceuticals, and criticized pharmaceutical company spending on direct-to-consumer advertising and other marketing relative to development costs of the drugs being advertised.
“The US is the largest purchaser of pharmaceutical products and should have price equity with other wealthy nations,” Makary and Prasad said.
JAMA Makary et al.
