A Defining Period for Future of FDA

FDA is at a “pivotal moment” for the nation’s public health and for global well-being due to the potential for realizing important gains in medical treatment based on science, Commissioner Robert Califf, MD, told the Washington legal and regulatory community last week at the annual meeting of the Food and Drug Law Institute (FDLI). He aims to address these challenges by improving FDA supply chain management in all areas, with digitally integrated systems that support preemptive actions and permit swift reaction to product shortages. Another priority is to counter widespread misinformation about vaccines and medical products through an emphasis on science-based decision-making at the agency and to advance medical product development based on appropriate communication between agency experts and those in regulated industry.¹

Califf emphasized the importance of improved information and data management systems in all FDA operations to support agency oversight and decisions about medical therapies. A priority is to FDA’s foods program, which will require sufficient funds from Congress to support something like a “21st century cures” initiative for food similar to that enacted to modernize and enhance the agency’s oversight of medical products in 2016. Under the current system, Califf noted that food producers don’t even have to tell FDA if they find contaminants in products awaiting shipment, limiting the agency’s ability to anticipate serious supply problems, such as the ongoing infant formula crisis.

Need for trust

At the same time, Califf defended the agency’s “appropriate” engagement with regulated companies as valuable for evaluating “the promise and risk” of new products and technologies. The broad knowledge of FDA staff about the full range of products in development—including many failures—can help guide developers to efficient pathways and prevent wasted and duplicative efforts, including clinical trials that could harm human subjects.

Similarly, Principal Deputy Commissioner Janet Woodcock urged a better understanding of the challenges and difficulties surrounding the many regulatory and scientific decisions made by FDA staff and leaders in another address at FDLI, which acknowledged the agency’s serious error in approving long-release opioids for market. Woodcock noted that a science-based regulatory process, as at FDA, has to issue regulations and policies that implement laws, a process that involves interpretation and inevitably generates dissent. Although science aims to assess the facts, many decisions are “surrounded by a cloud of uncertainty,” she said. Consequently, FDA’s critical approval of long-release opioids in 1995 was based on projections of harm and prescribing behavior presented at the time that were way off, Woodcock conceded. However, she acknowledged being pessimistic about this situation improving in the near future unless scientists and policymakers are able to communicate more broadly about the “big picture” of the uncertainty that inevitably surrounds agency decisions.

Califf raised similar concerns about “the growing dissemination of misinformation and disinformation about science, medicine, and the FDA, which is putting patients and consumers at risk” at the FDLI conference and also at the BIO International Convention in San Diego. Misinformation, he told biotech leaders, is the leading cause of death in the US, noting that almost all COVID-19 deaths now are preventable if patients become fully vaccinated and boosted. Misinformation, moreover, hampers the appropriate use of effective treatments for chronic diseases. It is paradoxical, Califf noted at BIO, that even though America is “innovating for the world,” US life expectancy is low, especially compared to other wealthy nations.

Califf observed at FDLI that he agreed to return to FDA last year after a previous stint as the agency’s leader in hopes of helping to modernize agency operations to prevent future critical delays and product shortages. This is a “pivotal moment” for the agency’s future effectiveness, he said, emphasizing that changes are needed to “stay ahead of the industries we regulate and the technology, science, and communications those industries employ.”

Reference

1. https://www.fda.gov/news-events/speeches-fda-officials/remarks-commissioner-califf-2022-fdli-annual-conference-06142022