After two rounds of public consultations, the EMA adopted the Guideline on quality, non-clinical and clinical requirements for investigational advanced therapy medicinal products in clinical trials, which will enter into force on 1 July 2025.
The guideline explains how to organise and submit information for a clinical trial application involving advanced therapy medicinal products (ATMPs). It covers both early-stage (exploratory) and later-stage (confirmatory) trials, with a perspective towards Marketing Authorisation Applications (MAA). It also offers guidance on different areas, such as product development, manufacturing, quality control, and some aspects of both non-clinical and clinical testing.
For trials involving paediatric populations, specific issues such as requiring preliminary safety data in adults, effects on reproductive health or germline expression may arise with the use of ATMPs. To this purpose, the guideline refers to other specific guidelines relevant to the clinical investigation of medicinal products in the paediatric population (e.g. ICH E11).