September 11, 2017

How FDA will shift its approach to gene therapies, according to its commissioner

Daily Briefing

    FDA Commissioner Scott Gottlieb on Thursday said the agency is looking to update the way it evaluates applications for gene and cell therapy drugs.

    Gottlieb said the agency already has about 626 active investigational applications for new drugs related to cell and gene therapy. He explained, "A lot of our policies, and yes, our regulatory framework, are fashioned to a much different paradigm of science and drug development," adding, "So we're challenged to adapt those policies." Gottlieb continued, "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 [it's] evaluating."

    To do so, Gottlieb said FDA will focus on having early scientific conversations with small and startup biotechnology companies, adding that such companies "sometimes don't have a full understanding of what it will take" to file drug applications and get them approved. Gottlieb explained, "It's often the smaller companies or individual researchers who are working with the most novel technology platforms," and FDA in some instances is "able to help significantly streamline the early development process by eliminating unnecessary preclinical tests" that can make it costly for small companies to get their drugs approved.

    Further, Gottlieb said FDA might shift the process for evaluating cell and gene therapy drugs to more closely align with how it evaluates medical devices, including by focusing more on post-market evaluations. According to Politico's "Pulse," FDA's medical device approval process typically is shorter than its drug approval process.

    Gottlieb said in the coming months he plans to further detail the steps FDA is taking "to adapt [its] regulatory principles to the new challenges [it] face[s] in properly evaluating a very different set of scientific opportunities" (Burton, Wall Street Journal, 9/7; Diamond, "Pulse," Politico, 9/8; Gottlieb remarks, 9/7).

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