Human cell and gene therapy research has advanced dramatically in recent years and opened the door to potential treatments for diseases once considered incurable. On January 15, FDA Commissioner Scott Gottlieb, M.D., and Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER), issued a joint statement announcing plans to keep pace with the rapidly growing and evolving field through new policy guidance and other assistance. According to the statement, FDA is turning its attention and additional resources toward these therapies in 2019 due to a “large upswing” in the number of cell and gene therapy investigational new drug (IND) applications. Based on an assessment of the more than 800 cell-based and gene therapy INDs current on file with the agency, FDA projects that it will receive more than 200 cell and gene therapy INDs per year by 2020, and will approve 10 to 20 such products per year by 2025.
To accommodate the uptick and to ensure regulation of firms that may be operating outside of regulatory compliance, the statement sets forth FDA’s planned actions to support cell and gene therapy product development in 2019: