April 29, 2021
I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Our center is a nonprofit think tank that scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products. Today I’m speaking from my perspective as a scientist who left Harvard more than 30 years ago to come to Washington D.C. to work in the House of Representatives. I worked as a Congressional investigator for the Subcommittee that conducted oversight over all of HHS, and that’s when I first learned about the laws and regulations governing the FDA. I was responsible for several oversight hearings that attracted enormous media attention, because we found that patients had been harmed when the FDA was not following the law pertaining to FDA approval.
The law is very clear: Drugs and biologics must be proven safe and effective, and that’s defined as having benefits that outweigh the risks for most patients. FDA’s memoranda that were provided to this Committee for this meeting and for each of these indications over these last 3 days have made it clear that the data do not support that. This Advisory Committee has looked at the data, seen reasons for optimism when looking at nonsignificant trends, and recommended that the FDA keep drugs on the market that don’t meet the standard specified by law. That’s your right to do that, since you are advising the FDA based on your perspectives, experiences, and interpretations of the data.
I want to thank the FDA scientists who carefully analyzed the data and presented their findings. You did a great job. I’m here to urge the FDA to follow in your footsteps and follow the law and rescind approval for these indications until the companies complete randomized clinical trials that prove that the benefits outweigh the risks. I especially want to thank Dr. Pazdur for explaining how the FDA’s Expanded Access program can fill in the gaps for patients who need access to these drugs. The companies agreed to complete confirmatory trials as part of the accelerated approval of their drugs, and I strongly urge the FDA to hold them to it.
All of these companies are leaders in their field and absolutely capable of conducting the research needed to prove whether or not their drugs have benefits that outweigh the risks for the exact specific indications they were previously approved for. The companies also have the ability to make expanded access quick and easy. Let’s face it, if they don’t have the expertise and resources to do the studies and help with expanded access, who does? If the data don’t confirm the initial accelerated approval, the companies should work with the FDA to design trials to narrow the indication to figure out which are the patients most likely to be helped and which are the ones most likely to be harmed.