Center for Biologics Evaluation and Research

You can actually go and watch it live to understand that commentary.

But basically, they want to establish a more standardized approach like fit for purpose regulatory review across both CDER and CBER and kind of tailored to specific and unique constraints of rare disease drug development.

They're signaling a coordinated institutional commitment via the FDA Rare Disease Innovation Hub, it's called, to align across review divisions.

acknowledging the statistical and enrollment challenges with small sample sizes, and really, you know, trying to codify the flexibility that they've always signaled.

So there's been a lot of chaos at the FDA in the Trump administration.

In the recent months, we had the guy who was head of what's called CBER, which is the biologics and some of the vaccines, the mRNA vaccines.

He was pushed out of the FDA, a guy named Vinay Prasad.

And then later afterwards, the FDA commissioner was also pushed out, a guy named Marty Makary.

I'm going to talk about my research during COVID-19 and not only my research, but the research at the time, what we knew when.

And then my work at the FDA, going from being a special assistant to senior advisor for the FDA commissioner and the Center for Biologics, CBER director, and then being promoted to being the head of the Center for Drug Evaluation and Research, CEDAR, where I had about 5,000 employees under me from December 2025 through May of 2026 in the circumstances under which I left the agency.

And we're gonna talk about some specific products and I need to limit myself to talking about publicly available data and then some decisions that have been made after I left the FDA because I'm a little restricted into what I can talk about because I can't talk about any internal communications or deliberations that happened during the FDA or the specifics about the decisions that were made during my time there.

So before COVID-19, I was doing research with my PhD in Denmark and looking at the large population study that causes a visual impairment in adults, looking at the association between the initiation of the preschool vision screening program in Denmark and the prevalence of amblyopia or lazy eye.

You know, the law says adequate and well-controlled studies, and the word studies is plural.

In February, Marty Macari, who was then Commissioner, Vinay Prasad, who was then head of the Center for Biologics, published in the New England Journal of Medicine.

And they said a single pivotal trial plus confirmatory evidence would be the new default standard for approval.

How is industry reading this and what do you see going on in real practice with FDA? Mark, let me ask you and then I'm going to turn tomorrow and then we're going to take a quick break.

But, uh, yeah.

Um, you know, another one that comes to mind is the new guy who's head of, um, CBER at the FDA.

Uh, Vinay Prasad was booted out.

This new guy was a Merck executive for 20 years.

So, um, [clears throat] it is, um, seeking comment on a number of focus areas, and then as public comment, there's gonna be a public meeting the end of this month.

And then it's also gonna hold some, um, focused stakeholder sessions with patients and industry, and it's also gonna have some internal meetings with, uh, CBER and CDER review teams.

And then it envisions developing a framework of all of this input, and, um, that could possibly include, uh, new rulemaking or changes in, um, current regulations, guidance, and, um, training plans, internal training plans, and things like that.

Okay.

[laughs]

The white paper does address this.

It goes through a lengthy list of, you know, all these different rare disease-focused programs, uh, throughout the agency, including in CDER and CBER.

This framework initiative is intended to build on these prior efforts as well as take a fresh look at areas where the agency can do more.