The Regulatory Process from Concept to Market

Abstract The US Food and Drug Administration (FDA) is responsible for the regulatory oversight of a wide range of products, including cellular and cell-based products or therapies regulated by the Office of Cellular, Tissue, and Gene Therapies (OCTGT) in the Center for Biologics Evaluation and Research (CBER). This chapter focuses on several key issues relevant to the development of cellular and cell-based products, including early and late phase considerations for chemistry, manufacturing and controls, pharmacology/toxicology, and clinical testing. This chapter also introduces the following topics: the regulatory process; sponsor meetings with OCTGT; the requirements for submitting an Investigational New Drug Application (IND) IND content, and FDA review of an original IND application; and special regulatory considerations such as clinical research involving children, responsibilities of sponsors and investigators and the use of standards. Knowledge of and consideration of the topics discussed in this chapter may aid in the development of cellular and cell-based therapies.