FDA has a variety of patient safety programs and procedures in place for assessing and promoting the safety of investigational and approved new drugs and biological agents. For investigational products, FDA requires sponsors to report serious and unexpected adverse events associated with use of a product as soon as possible and within 15 calendar days. Sponsors must report any unexpected fatal or life-threatening experience associated with the use of the product as soon as possible, and within seven days. Prompt reporting allows the agency to analyze reports and take immediate action as indicated. Sponsors summarize other events in annual reports and information amendments that facilitate our periodic comprehensive overview of the effects of a product, including safety. FDA review teams either review related products or meet periodically to share information and discern trends.

Through experience in the review and regulation of gene therapy products, FDA has identified several concerns and issues raised by gene therapy products that differ from those typically raised by more traditional products. Because of the specific issues raised by gene therapy and because continuing and expanding research in gene therapy is creating new demands on oversight systems, FDA has pursued the development of a comprehensive Gene Therapy Patient Tracking System (GTPTS) to help ensure the appropriate oversight and safe development of these therapies.