Regulation of Genetic Therapy - The American Experience

Abstract

Gene therapies intended for human use in the United States of America are evaluated by two national organisations, the Food & Drug Administration (FDA) and the Recombinant Advisory Committee (RAC) of the National Institutes of Health (NIH) and by one local institution specific mechanism, the Institutional Review Board (IRB). At the FDA’s Center for Biologics Evaluation and Research (CBER), all gene therapies, just like all new and investigational therapeutics in the US, are evaluated based upon the science, medicine and manufacturing issues. The RAC, a body composed of scientists, physicians, lawyers and ethicists, has a more focused review responsibility to evaluate gene therapy protocols that utilize National Institutes of Health funds. The RAC reviews protocols that may occur at their particular institution. This board has the responsibility of protecting the rights and welfare of human subjects. The local board has the opportunity to oversee the individual investigator and the equity of the protocol for the individual human subject. Gene therapy protocols in the US should have IRB review before they are sent to the national level for evaluation.