Regulation for Gene and Cell Therapy Medicinal Products in Europe

An important step in the regulation of cell and gene therapy medicinal products, which are classified as advanced therapy medicinal products (ATMPs) in the European Union, has been made with Regulation 1394/2007/EC. By this regulation a new committee, the Committee for Advanced Therapies, has been established to ensure appropriate coverage of scientific and regulatory aspects of ATMPs. In addition, novel regulatory tools specific for ATMPs such as the classification, certification, and hospital exemption were introduced to support the development of this product class. By nature, ATMPs are a special class of medicinal products with characteristics different to conventional drugs and even other biologicals. Hence, regulatory requirements for manufacturing and clinical evaluation need to be tailored to the type and design of the ATMP, as well as to its manufacturing process and clinical indication. This chapter summarizes the general regulatory pathway for ATMPs as well as the currently applicable ATMP-specific procedures. In addition, the regulatory requirements regarding manufacturing, quality, and nonclinical and clinical testing for cell and gene therapy medicinal products are discussed. Important aspects of the environmental risk assessment, a provision for ATMPs containing genetically modified organisms, are also reviewed.