The U.S. Food and Drug Administration has announced plans to shorten drug approval timelines and address increasing chronic disease rates among children, according to a viewpoint published by Commissioner Martin A. Makary, MD, MPH, and Deputy Commissioner Vinay Prasad, MD, MPH.
Accelerated Drug Review Pilots
The Food and Drug Administration (FDA) plans to launch pilot programs that are intended to "deliver sponsors a decision in weeks," Drs. Makary and Prasad noted. The programs build on procedures used during the COVID-19 pandemic when review processes that previously took a year were completed in weeks.
We believe this is clear demonstration that rapid or instant reviews are possible.
—Makary & Prasad, JAMA, 2025
Under the pilot, sponsors would submit materials such as manufacturing plans, draft labels, and packaging before trial completion to facilitate faster final decision-making following pivotal trial results.
Generative AI in Regulatory Review
On May 8, 2025, the agency began its first generative artificial intelligence (AI)–assisted scientific review pilot. The AI system performs a first-pass review of application documentation—reportedly often exceeding 500 000 pages—and generates standardized analytical tables.
The FDA is also exploring AI-based toxicity prediction using computational modeling and chip technology to reduce development time and reliance on animal testing.
Advisory Committee and Industry Changes
To address potential conflicts of interest, the FDA has removed industry representatives from all advisory committees where statutorily permitted. The authors cited the agency’s past approval of oxycodone for chronic pain as a precedent for regulatory reform, describing it as "the FDA’s illegal approval of oxycontin for chronic pain based on a 14-day study, the immediate hiring of the former FDA regulator by Purdue Pharma, and a subsequent epidemic that killed approximately 1 million people in the U.S.”
Food Additive Policy and Ingredient Reviews
The FDA has initiated a review of food additives and ingredients not permitted in other developed nations. The authors reported the removal of all nine petroleum-based food dyes from the U.S. food supply and the approval of food dyes derived from natural ingredients.
The agency has also conducted an expert panel review of talc, which remains present in medications and candy, despite its removal from topical baby powder due to carcinogenicity concerns. The authors wrote, “Like so many additives, its addition is strictly for convenience—to prevent pills from getting stuck in machines or wrappers from sticking to candy.”
Postapproval Monitoring With Big Data
The FDA intends to leverage the availability of health data and cloud computing for real-time product monitoring. Causal inference methods using nonrandomized data—including target trial emulation—may provide “actionable causal conclusions” at lower cost than randomized controlled trials, according to the authors.
The use of Big Data may allow the agency to reduce the traditional requirement of two pivotal trials to one for some products, particularly those that target rare diseases.
Generic and Biosimilar Drug Development
While the FDA is legally prohibited from considering price in benefit-risk assessments, the agency plans to address cost indirectly by expediting generic drug approvals and “massively streamlining the burden to develop biosimilar compounds,” the authors stated.
They added that “most large pharmaceutical companies spend more on marketing, including direct-to-consumer advertisements that can be misleading, than they spend on research and development.”
Public Health Context and Regulatory Focus
The reforms are being developed in response to current epidemiologic trends. The authors reported that 40% of U.S. children have a chronic medical condition, one in six has a neurodevelopmental disorder, and early-onset Alzheimer disease has increased by at least 300%. They noted that “historical and comparative global data do not support a genetic etiology for this rise, nor a deficit in willpower.”
Strategic and Philosophical Shifts
The authors described plans to move “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.” This includes reevaluating legacy FDA processes such as monographs for infant formula, which have remained largely unchanged since 1998, aside from the addition of selenium.
Disclosures can be found in the published viewpoint.
Source: JAMA