November 19, 2024
I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Our center is a nonprofit public health research center that scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products — so we have no conflicts of interest.
Thank you for the chance to share our views today and we thank these Committees and the FDA for your important work. Prior to my current position, I was a post-doc in epidemiology and public health at Yale Med School, and was a faculty member and PI at Yale and Harvard. I also conducted hearings on FDA safety standards while working in the US Congress and the White House. I’m a founding Board member of the nonprofit Alliance for a Stronger FDA, which educates Congress about the need to financially support the essential work of the FDA.
We work closely with many patients and patient advocates, many of whom were harmed or had family members harmed by psychotropic drugs. So although the barriers to Clozapine due to the REMS may be excessive, we need to focus on safety.
We’ve heard today that the Clozapine REMS aimed at reducing severe neutropenia is too burdensome, even though most of the requirements have not been enforced. I agree it makes sense to make the REMS less burdensome but we urge the FDA to keep the REMS strategies that are proven to be essential to keep patients safe.
While the Clozapine labeling includes vital information about neutropenia, it can’t take the place of the REMS, because we all know that not all physicians read the labeling and even those who have read it might not remember exactly what it includes. Monitoring required by the REMS could be reduced to make it less burdensome, but having careful monitoring in the early months is absolutely essential to save lives. Even though there has been enforcement discretion, we heard this morning that some universities stick to the REMS requirements despite the discretion, as if there was no flexibility. Since many follow the REMS even when there is enforcement discretion, we can’t assume that they would do so if there were no REMS requirements at all. On the contrary, having the REMS is sending a clear message that these safeguards to prevent neutropenia are important, and many physicians will follow them. Many of those same physicians might not do so if there was no REMS, and the outcome for patients would likely be very different. As one of the Committee members noted this morning, when you relax some elements of the REMS, you increase the risks to patients. And has been stated, even if there were no REMS at all, there would still be substantial barriers to Clozapine because of its numerous adverse effects.
In conclusion, while considering how to reduce the barriers to access, please remember that safety is still essential.