EAPB: Regenerative Medicine - "Scientific Advice"

Advanced Therapy Medicinal Products (ATMPs) are tightly regulated by the European Medicines Agency (EMA) with the intention to ensure the safety, efficacy as well as the good quality of the products.

A well defined risk evaluation in order to cover the whole development process and not only quality or safety risks should be set-up. For example, tumorigenicity, genome stability, biodistribution are now among the top issues during non-clinical and clinical development and need to be addressed for stem cell-based products early. A key point is hereby an efficient communication with the regulators as early as possible to avoid detours or even worst failures.
So the scientific advice became a very powerful tool to find the right way through the jungle of guidelines, reflection papers, regulations and the expectation of the regulators.