We will review challenges in translation required to develop the ATMPs pharmaceutical product (cell and gene therapy products) from nonclinical to the clinical stage, as well as the challenges related to the determination of proof-of-concept, how to choose clinical dose from nonclinical studies and address the nonclinical questions required to demonstrate ATMP product safety before going into the first-in-human study. Presenters will also discuss how European Regulatory Agencies help the development of ATMP pharmaceutical products.
Key learning points :
Understand in-depth the product in relation to the intended use
Justify the study designs, test model(s) (in vitro, ex vivo, and/or in vivo), and/or absence of specific studies
Use integrated approach covering CMC/NC/Clinical aspects critical for ATMPs pharmaceutical product
Adapt QbD principles such as the development of an adapted Control Strategy based on Risk Assessment: it is possible and encouraged
Engage early dialogue with EMA and National agencies: strongly encouraged for successful such development