The guideline provides guidance on the structure and data requirements for a clinical trial application for investigational ATMPs and a perspective towards Marketing Authorisation Applications (MAA). The guideline is multidisciplinary and addresses development, manufacturing and quality control as well as non-clinical and to some extent clinical aspects for investigational ATMPs.
The scope of the evaluation and authorisation of clinical trial applications as per Regulation (EC) No 536/2014 is to ensure that the rights, safety, dignity and well-being of subjects are protected and prevail over all other interests; and that it is adequately designed to generate reliable and robust data…
