Regulation of cell‐based medicinal products

Abstract

The regulation of cell‐based medicinal products in the EU is governed by legislation for medicinal products in general plus dedicated regulations for advanced therapy medicinal products (ATMPs). In addition, the procurement and use of the tissues and cells used to manufacture these products are regulated by additional legislation. This presentation will focus on how a regulatory strategy for bringing a cell‐based ATMP under development into clinical trials and ultimately to marketing authorisation should be formulated around all relevant legislation and the guidelines used to implement it. A regulatory strategy should address the quality, safety and efficacy aspects of a medicinal product intended for human use, through defined strategies around CMC (chemistry, manufacturing and controls), nonclinical studies and clinical trials. In addition, regulatory routes to clinical trials and market authorisation that are appropriate to the ATMP under development should also be considered from an early stage, for example including orphan designation, paediatric investigation plans and accelerated approval schemes. Building a regulatory and product development strategy around these elements for a cell‐based ATMP will be outlined.