Fiche publication


Date publication

février 2025

Journal

Annales pharmaceutiques francaises

Auteurs

Membres identifiés du Cancéropôle Est :
Pr BENSOUSSAN Danièle , Dr POCHON Cécile


Tous les auteurs :
Petitpain N, Prontskus V, Gauthier M, Loutterbach L, Pochon C, Dalle JH, Bensoussan D

Résumé

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 additionnal 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.

Mots clés

advanced therapies medicinal products, gene therapy, médicaments de thérapie innovante, pharmacovigilance, safety, thérapie génique

Référence

Ann Pharm Fr. 2025 02 27;: