Abstract
Advanced therapy medicinal products (ATMPs) are becoming the new kid on
the block for the treatment of a variety of indications with promising
results. Academic centres have often been at the forefront of basic
research, preclinical phases, and translational studies for the clinical
development of ATMPs for several approved drugs. Despite the academic
contribution to the basic and clinical research of ATMPs, undertaking a
full product development process is extraordinarily challenging and
demanding for academic institutions. Meeting regulatory requirements is
probably the most challenging aspect for academic development,
considering the limited experience and resources compared with
pharmaceutical companies. The aim of this review is to present and
discuss the key aspects to be considered when developing novel ATMPs
from an academic perspective, based on our own experience. Emphasis is
placed on understanding the regulatory requirements during the early
phases of the drug development process, particularly for the preparation
of a Clinical Trial Application. In general, academic centres do not
possess experience in product-related documentation (such as the
Investigational Medicinal Product Dossier), and therefore, early
interaction with regulators is crucial to understand their requirements
and receive guidance to comply with them. Tips are provided on how to
manage quality, non-clinical, clinical, and risk and benefit
documentation, based on our own experience and challenges. We believe
this review will contribute to providing key regulatory knowledge to
academic and clinical environments with the purpose of smoothing the
regulatory path of ATMPs.