Time for Changes at the FDA, Expert Group Says

The FDA is in need of structural and cultural changes to bring the agency in line with its original mission of acting as an independent government regulator of the food and drug industries, according to a panel of experts.

Their call for change rested on the “eroded” reputation of the FDA as the gold standard in evaluating drugs, which has been weakened over recent decades by legislation, regulations, external pressures, and internal practices, argued Sharon Batt, PhD, of Dalhousie University in Halifax, Nova Scotia, Canada, and Adriane Fugh-Berman, MD, of Georgetown University Medical Center in Washington, D.C., in a report released by PharmedOut, a project from Georgetown University’s department of pharmacology and physiology.

“We’re cognizant that FDA has to meet a huge demand now dealing with two pandemics, multiple public health crises,” said Reshma Ramachandran, MD, of the Yale School of Medicine in New Haven, Connecticut, who is part of PharmedOut’s working group, during a panel discussion. And that’s atop the agency’s burden of regulatory work across a large group of industries, she added. “However, at the end of the day, it’s clear that there needs to be a truly independent FDA.”

Batt and Fugh-Berman outlined four areas in which the agency should consider changes to bring it back in line with its mission, including transparency and accountability, commitment to innovation, standards of evidence for drug marketing, and value in the healthcare landscape overall.

They said that to improve transparency and accountability, the agency should clarify every advisory board member’s conflicts of interest, increase the diversity of opinions from those members when grappling with politically charged issues, and minimize use of invited speakers with ties to a drug sponsor.

Furthermore, they highlighted the need for more clear internal and public FDA communications on the complexity of drug approval decisions, including sharing any concerns that reviewers may have had about an approved drug after the process.

The authors also recommended accurately using the term “innovation” when it is applied to new drug approvals, noting that the term is typically misused to describe all drugs that are approved by the agency, especially when granted on an expedited approval track.

Panelist John Powers, MD, of George Washington University School of Medicine, who is also part of the working group, emphasized the need for more transparency in these accelerated approvals.

“You want to call it what it is. It’s conditional approval,” Powers said. “We don’t know that the evidence is there yet, and some of these things, like breakthrough therapies, almost seem erroneous on their face. You can’t possibly know something is a breakthrough at the point in which they give that designation.”

Fellow working group member Susan Molchan, MD, of Walter Reed National Military Medical Center in Bethesda, Maryland, noted that the way the FDA describes its processes and it designations can have far-reaching implications.

“Language is important,” she said. “The FDA, who are they working for? More and more, it looks like corporations versus valuing public health.”

To further improve standards of evidence for approvals, the authors recommended that the agency rely more on clinical trials that compare different drugs directly as opposed to the standard use of placebo comparisons.

This reliance on placebo comparison and fast-tracking of new drug approvals is problematic, Powers suggested.

“The focus now in the current era seems to be more on speed of drug approvals to market and the quantity of drugs approved, with an assumption that more is better and access is better for patients, even if it’s not clear whether those interventions improve patient outcomes,” he said.

Overall, Batt and Fugh-Berman suggested that the agency needs to focus on approving new treatments that are “truly innovative” based on evidence that sufficiently assesses how useful the therapy will be for patients.

The wide range of recommended changes will have to be enacted by an equally broad-based coalition, they added. They expect that these changes will require legislative efforts, as well as internal policy updates and cultural shifts in the way that the agency interacts with industry representatives.

Still, they wrote that they were “optimistic” that the agency will be able to make these changes and reaffirm its mission to improve the lives of all Americans.