Gene Therapy – FDA Takes Steps Toward Clarifying Scope of Orphan Drug Exclusivity
Gene therapy is the focus of concentrated industry attention, with numerous products in development to treat a variety of serious chronic diseases. The U.S. Food and Drug Administration (FDA or the agency) is taking active steps to facilitate this development. In July 2018 alone, the agency issued six draft guidance documents on gene therapy. In addition, in August 2018, FDA and the National Institutes of Health (NIH) announced efforts to streamline review of gene therapy protocols by, among other things, eliminating the regular required review of every protocol by the NIH Recombinant Advisory Committee (RAC), a federal advisory committee established in 1974 to advise on research related to the manipulation of nucleic acids.
This article originally appeared in the December 2018/January 2019 issue of Update Magazine and is posted with permission from FDLI.