These are boom times for gene therapy, an innovative type of medicine that works to treat disease at the genetic level to offer a longer-lasting treatment and potentially even a cure.
The Food and Drug Administration has seen a "noticeable uptick" in the area, Dr. Peter Marks, the agency's top official overseeing gene therapy, said on Thursday at the Gene Therapy for Rare Disorders conference in Boston. Marks heads the FDA's Center for Biologics Evaluation and Research.
More than 200 applications to try gene therapies were submitted to the FDA last year, he said, with well over 800 gene-therapy research efforts ongoing.
Gene therapy has "tremendous promise," Marks said, but developers — many of which are focusing on inherited rare diseases first — also face serious challenges because of the complexity of making the treatments.
Such a large number of experimental products are in the works, in fact, that backlogs have formed. Those are "actually slowing down investigating these therapies and getting these therapies to patients," Marks said.
So "unless we get it right here in the rare-disorders space and develop the technology," Marks said, "we're not going to be able to have it grow to meet the larger need."
Here are the key suggestions he had:
- Look at incentives from the FDA. The regulator has various programs to encourage development in important medical areas. In that vein, gene-therapy startups could be eligible for a "regenerative medicine advanced therapy" designation, especially if they're working on rare diseases, Marks said. That designation, intended to speed up gene-therapy development efforts, can come with more attention from and meetings with the FDA and its top leaders, he said. According to Marks, the agency has received 94 requests about the designation as of the start of March, and 31 have been granted, most of which also had a similar but distinct "orphan product" designation.
- Check FDA guidance, but expect it to keep changing. The agency put out a "suite" of six draft recommendations around gene therapy in July, covering topics like development efforts in hemophilia, retinal disorders, and rare diseases, and it wants to put out finalized guidelines later this year, Marks said. But they should have a pretty short life span before they're updated and evolve, he said, "because the science is really moving so quickly."
- Design research "very thoughtfully." The gold standard of scientific research is a type of trial designed to take away anything that could introduce bias and influence the results. But those "randomized trials aren't going to be possible here," because biopharmaceutical companies are working with such small patient populations, Marks said. Instead, companies can use a broader standard of "well controlled" studies, he said, and the FDA is also open to them using "surrogate endpoints," a proxy measure that's supposed to connect to how well patients do but might not necessarily. "This is a place where we think it's really good to interact with the agency early and interact thoughtfully," Marks said, adding that the FDA has a program for companies to do just that.
- Think about how to smooth a difficult part of the development process. Gene therapy has been largely developed by academics at research institutions, posing problems when it comes to companies taking up those products and making them at a bigger scale. "There is a gap," Marks said. "They don't transfer well sometimes." He said that getting companies and academics to agree on a common technology could help but that there would likely be challenges.
- Small companies, speak up. Smaller startups don't always know they can ask FDA questions or ask for a supervisor review, Marks said. But companies may have a better sense of why something won't work than the FDA. "So don't be bashful about communicating with us early, often, or questioning things that don't seem right to you," he said.
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