Can FDA replicate Operation Warp Speed for rare diseases? Not yet, but Peter Marks has some ideas

As COVID-19 continues to dominate the FDA’s bandwidth, Peter Marks, M.D., Ph.D., director of the agency’s Center for Biologics Evaluation and Research (CBER), is playing financial whack-a-mole to figure out where resources should flow. Topping his priority list is more money to regulate the budding cell and gene therapy field, an effort he hopes can at least partly mirror the regulator’s response to the pandemic. 

Marks' comments came Tuesday on a call with the Alliance for a Stronger FDA, a lobbying group that advocates on behalf of the agency and its regulatory efforts, to discuss CBER’s budget priorities. Marks touched on a number of topics ranging from the need to bolster the agency’s overall IT infrastructure to improving staff retention. But most notable were his calls to speed up the center’s cell and gene therapy review efforts, which he said have failed to meet his expectations on account of the workload. 

“We are clearly in a position that we are not giving the kind of feedback that I'm comfortable with in real-time … to both vaccine developers, and particularly for those in the gene and cell therapy field,” he said. 

The issue is made more pressing by the fact that gene therapy is still a burgeoning field. The famed CRISPR-Cas9 technology that won Emmanuelle Charpentier, Ph.D., and Jennifer Doudna, Ph.D., a Nobel Prize was discovered a decade ago, with therapies based on this innovation only now progressing through the clinic. In other words, if there’s already a staffing crunch on reviewers, the problem is going to be exacerbated unless CBER can get more money to shore up its review effort. 

Marks said he’s not naive to the fact that Congress will likely ask him to show what he can do with more money. His answer, in part, centers on what CBER has been able to do with COVID-19 vaccine manufacturers. As part of Operation Warp Speed, the famed federal effort (PDF) to rapidly develop and progress these vaccines in 2020, the regulator maintained a consistent dialogue between sponsors and manufacturers leading up to regulatory submission.

While such detailed and sustained dialogue couldn’t currently be instituted agencywide, it could make for a valuable pilot project to prove its worth to Congress, Marks said.

“Parents of patients, of kids who have severe diseases, they don't want to hear about Type A, Type B and Type C meeting timelines,” he said. “They want to know what we're doing to truly make a difference in trying to bring something better to their children's lives.”

The solution is, painfully, both simple and difficult to accomplish: hiring and retaining more staff. The agency needs more permanent full-time employees across all disciplines, Marks said.

“So there [is] a whole host of growth that is required,” he said. “And without that, we're not going to be able to meet, I think, the need here.” 

That need, according to Marks, is to improve the agency’s response time to sponsors. He specifically noted improving on the 60-day and 75-day response times for Type B and Type C meetings, respectively, and said getting back to “good” should not be the bar. 

“I don't think that's what we want to do with something that we care about as much as gene therapy,” he said. “I think we want to do better than that.” 

Marks' comments came the same day that CBER's sister unit the Center for Drug Evaluation and Research (CDER) announced the launch of its Accelerating Rare disease Cures program. The initiative is designed to speed up and increase the development of effective and safe treatment options to address the unmet needs of patients with rare diseases.

In its first year, the program will focus on strengthening internal and external partnerships with stakeholders and will engage with external experts to help identify solutions for disease drug development, CDER said.