[European pharmacovigilance of advanced therapy medicinal products]

Advanced therapy medicinal products (ATMPs) are playing an increasingly important role in therapeutics, and sometimes represent the only hope of improving or even curing some pathologies. Because of their biological origin, composition, mode of action, and their frequent technical complexity, ATMPs are potentially generating new risks. Moreover, clinical trial data generally concern only a very small number of patients. The pharmacovigilance of ATMPs must adapt to these constraints and to these new identified or potential risks to be authorized according to an European centralized procedure. The conception and the assessment of the Risk Management Plan are essential to complete routine pharmacovigilance with post-authorization studies and additional risk minimisation measures adapted to the identified safety concerns. This article provides an overview of existing ATMPs and their specific regulatory pharmacovigilance requirements, involving the role of the European committees. It aims to describe the essential elements of the risk management plan of ATMPS, with a focus on delayed adverse reactions. The particular case of ATMPs prepared in the context of « hospital exemption », is also addressed, with the example of ATMPs for adoptive immunotherapy.