Phillip Noguchi, M.D ., Food and Drug Administration, Rockville, Maryland Clinical trials using gene therapy products seems, at first blush, similar to the conduct of clinical trials with any FDA regulated drug or biologic. Certainly these trials incorporate fundamental concepts such as adherence to the agreed upon clinical protocol, timely reporting of adverse events, independent monitor of the trial, appropriate informed consent process and documentation.
Gene therapy is complicated.
Not as complicated as launching a space shuttle, but coordination of efforts, and communications of problems in a timely fashion becomes directly of importance to human subjects who may consider participation in a gene therapy clinical trial. As a biological product, the active product may have toxicities but the manufacturing process may also contribute to adverse events. The numbers of human subjects treated is still relatively small, so that trends are not easily seen at this time.