FDA modernizing evaluations as gene, cell therapy fields grow

FDA Commissioner Scott Gottlieb, MD, said Thursday the agency is working to modernize the way it evaluates a wave of new drug applications derived from the rapidly growing fields of gene and cell therapy.

FDA Commissioner Scott Gottlieb, MD, said Thursday the agency is working to modernize the way it evaluates a wave of new drug applications derived from the rapidly growing fields of gene and cell therapy. Speaking before a coalition of corporate and academic research groups, Gottlieb discussed steps FDA will take to begin to deal with the new science. He noted FDA will focus on conducting early scientific conversations with startup and small biotechnology companies. Gottlieb noted 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. Gottlieb estimated FDA already has more than 625 active investigational new-drug applications related to gene and cell-therapy products. "A lot of our policies, and yes, our regulatory framework, are fashioned to a much different paradigm of science and drug development," he said. "So we're challenged to adapt those policies." He added, "Our ability to fully capitalize on new science, and maintain FDA's gold standard for product review means FDA also needs to modernize itself alongside the new platforms that we're evaluating."