FDA Commissioner Makary outlines agency guides: gold-standard science and common sense

Gold-standard science and common sense will be the guiding philosophy of the U.S. Food and Drug Administration (FDA), said Commissioner Martin A. Makary, MD, MPH.

“Priorities for a New FDA” is the policy plan published this week by Makary and co-author Vinayak Kashyap “Vinay” Prasad, MD, MPH, director of FDA’s Center for Biologics Evaluation and Research (CBER). The viewpoint article published in JAMA did not mention the Make America Health Again Report published in May. But it touched on some common themes and topics already publicized by President Donald J. Trump and Robert F. Kennedy Jr., his secretary of Health and Human Services.

“Why does it take more than 10 years for a new drug to come to market? Why are childhood chronic diseases so prevalent? And how can regulators adapt to meet the challenges facing clinicians today? These questions are at the forefront for the U.S. Food and Drug Administration,” they wrote.

Makary summarized some of the current woes in the U.S. health care system, and the ailments affecting millions of Americans. Collectively, “our medical system has been a 50-year failure,” the article said.

“Fresh new ideas are needed to address root causes and develop innovative approaches,” they said. That means examining the role of ultraprocessed foods, food additives, environmental toxins and how those have paralleled the epidemic of chronic disease in the United States. The American health care system needs to change from a reactionary responder to a proactive, pro-health force against “the current forest fire that is the worsening health of the U.S. population.”

The top priorities will be:

• Accelerating cures, taking lessons from the COVID-19 pandemic, when yearlong review processes were completed in weeks. Another example is infant formula, which has gotten one new element since 1998, showing that some of FDA’s legacy process “slow down decisions and do not increase safety.” The solution is to partner with industry while avoiding the “cozy relationship” to food and pharmaceutical makers FDA had in the past.
• Unleashing artificial intelligence (AI) to review FDA applications and to predict toxicity-leveraging chip technology. Those processes will save time in drug development. FDA also will modernize how it reviews AI-based technologies.
• Advocating for healthier and safer food for children, improving the nation’s “increasingly chemically manipulated diet.” “The FDA will restore focus on the ‘F’ in FDA,” they wrote.
• Harnessing big data to research root causes of chronic diseases and to conduct postapproval monitoring of new products.
• Lowering drug prices. U.S. patients pay more for drugs than people in other countries, representing “a great American rip-off.” By law, FDA will not consider price in benefit-risk calculations. But the agency will use its power to address costs, for example, by expediting generic medications and streamlining burdens to create biosimilar compounds. The United States is the largest purchaser of pharmaceutical products and should have price equity with other developed nations, Makary and Prasad wrote.