The U.S. Food and Drug Administration (FDA), as the regulatory authority for the registration and approval of drugs, has so far approved four gene therapeutics for the treatment of rare diseases such as autoimmune diseases and genetic disorders in order to ensure new therapeutic concepts as an option for the intervention of these diseases. However, there are still 900 new products in clinical trials on hold for which applications have been submitted to the authorities. According to experts at the FDA authority, this is a positive development, but these gene therapy products must also be subject to clear regulatory guidelines, which are limited by the FDA as general conditions because product development and testing of these complex groups of active ingredients must be controlled and assessed differently from conventional drugs. A certain degree of uncertainty, therefore, remains even after completion of the development and review process. The development of science in this area of innovation is so rapid that questions about these complex groups of active ingredients, such as product safety, mechanism of action, safe modes of application and, last but not least, therapeutic efficacy, must be tested over a long period of time. The FDA authority, therefore, offers the developers of these products binding guidelines that enable them to answer critical questions in the research and development of their innovative products. In order to provide even more meaningful assessments, the authority has currently published six final guidelines, which contain specifications on the manufacture and quality of gene therapy products, so that a minimum of uncertainty remains at the time of approval and the marketing of the product. Another set of FDA guidelines has also already been drafted. This is intended to facilitate the review of different products for the same indication and make them comparable. In this context, molecular structural features are the main focus of the evaluation.
