FDA Adapting to Increase In Gene Therapy

09/10/2017

Food and Drug Administration Commissioner Scott Gottlieb said the FDA is hurrying to modernize the way it evaluates a flood of new drug applications in the fast-growing fields of gene and cell therapy.   The agency will take to begin to grapple with the new science, focusing on conducting early scientific conversations with startup and small biotechnology companies.  Dr. Gottlieb said they "sometimes don't have a full understanding of what it will take to get" applications filed and approved. "It's often the smaller companies or individual researchers who are working with the most novel technology platforms," he said. He estimated the agency already has at least 626 active investigational new-drug applications related to gene and cell-therapy products, and the FDA needs to modernize alongside the new platforms that being re evaluating

Small companies sometimes overestimate the amount of information needed to file an application and so too many of the costs of development therefore get front-loaded, increasing the cost of advancing new science.  The FDA's review staff is sometimes able to help significantly streamline the early development process by eliminating unnecessary preclinical tests.  Some of the therapies at issue include CAR T-cell treatment, in which a patient's immune cells are altered and magnified in the laboratory and reinfused in the patient in order to create a treatment that is most effective against that patient's specific cancer.  The first such treatment involving genetic altering of a patient's cells to combat cancer was approved by the FDA last month. Novartis AG's new medicine, called Kymriah, was approved to treat children and young adults with an aggressive type of leukemia.