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. But Benjamin Franklin might note from Poor Richard's Almanac: "for want of a nail, a shoe was lost; for want of a shoe, a horse was lost", while Shakespeare might comment from Richard III "A horse! A horse! My kingdom for a horse!
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. No gene therapy product has been approved; the knowledge base of real and potential adverse events related to gene therapy products is still rapidly evolving. 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. How will a potential human subject be able to realistically evaluate the risks of gene therapy trials? How will the sponsor of such a trial be able to realistically provide to the potential human subject enough information to be considered? Given the still small numbers of treated human subjects, how can we communicate and discuss adverse events, while protecting individual patient identity?