August 16, 2023
FDA biologics center director Peter Marks said the agency plans to encourage sponsors to use the accelerated approval pathway, particularly for rare disease treatments, as the agency also moves ahead with an Operation Warp Speed-like pilot for rare diseases later this year.
In an expansive interview with Endpoints News, Marks said he expects to see more accelerated approval applications in the rare disease gene therapy space as well as potentially in cell therapies. Marks said the rare disease Operation Warp Speed effort, which he announced in February, will “start small” to make sure the agency can work closely with the first round of participants.
Marks spoke with Endpoints on Aug. 15. This interview has been substantially edited for length and clarity.
Lia DeGroot: I wanted to start with a question about the Office of Therapeutic Products. You’ve brought on Nicole Verdun to lead that office and are staffing up. How are things going?
Peter Marks: It’s great to have her on board because that stability will help us get to our hiring goals. People like to know who their bosses are going to be.
We’re making reasonable progress toward getting our reviewer positions filled, including physicians and CMC reviewers. It’s a little early to tell how far we’re going to get by the end of the year. I’d always love if we had more people wanting to come work at the agency.
DeGroot: A few months ago, there was a bit of a backlog with cell and gene therapy applications. Is there still?
Marks: There’s probably a slight backlog still. We’re still trying to do better with the timeliness of the feedback that we’re giving. We’re trying to make sure we get through the backlog and then cut down the time it’s taking to give people things like interact meetings and some of the more helpful meetings that we don’t have quite the same statutory timelines on as type A, B, C meetings. That’s what staffing up helps with.
There is something that is still a valid concern, which is that we’re doing more written responses than industry would like. We’re doing that simply because of bandwidth.
DeGroot: I wanted to switch gears a little bit to accelerated approvals. FDA is now working with some more authorities around requiring confirmatory trials to have started before decisions. Any progress on implementing that?
Marks: Yeah, although it’s a work in progress, we are very actively working on making sure sponsors understand that we are serious about wanting good evidence, as required by the FDORA, that we be able to have compelling evidence that these clinical trials are either underway, enrolling or completed before we actually take our regulatory action, or at least enrollment is completed.
Some of this that we’re doing right now is trying to understand what we need, when. When someone submits something for accelerated approval, what will we be requiring?
If you’re going to be submitting for accelerated approval, we’ll need to have reasonable evidence that you’re serious about conducting your clinical trial for confirmation. The exact form of that seriousness is something that we’re still working on.
DeGroot: I’m also wondering how difficult it might be for some applicants to be able to carry out those studies. What are you hearing from industry?
DeGroot, L. (2023, August 16). Q&A: FDA’s Peter Marks ready to encourage more accelerated approvals for rare diseases. Endpoints News. https://endpts.com/qa-fdas-peter-marks-ready-to-encourage-more-accelerated-approvals-for-rare-diseases/