Washington, D.C. — The advent of federal regulatory guidelines for developing gene therapies—which transplant new genes into people’s cells in order to correct disorders—is a crucial moment in the narrative of the still-novel treatment type. The United States Food and Drug Administration’s (FDA) draft guidance is called Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, and it comes in response to more than 900 investigational new drug (IND) applications in the gene-therapy space. The goal, aside from facilitating development with a consistent regulatory framework, is to increase the number of gene therapies available.
“As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs,” said Peter Marks, director of the Center for Biologics Evaluation and Research at the FDA, in a press release. “Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.”
The FDA has already noted that post-market clinical trials are going to be critical to ensuring safety, given how novel gene therapies are.
The new guidelines bode well for both product developers and patients. It helps illuminate the FDA’s point of view with regard to “sameness”—a point of view that encourages creating multiple gene-therapy products for the same condition. The patient upshot is that multiple approved treatments for the same disease could create a more competitive market with more options and lower prices.