The clinical use of Advanced Therapy Medicinal Products (ATMPs) in humans may be associated with specific risks to the patient and to third parties. These risks are determined by various risk factors, which are related to the quality, biological activity and application of the ATMP. Since ATMPs are very diverse in nature (i.e. gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products (sCTMPs) and tissue engineering products (TEPs)), a flexible approach to address and evaluate potential risks associated with the clinical use of ATMPs is described in the 'Risk-based approach'.
The concept of a 'Risk-based approach' has been introduced to the legislation with the revision of Annex 1, part IV of Directive 2001/83/EC as amended by Directive 2009/120 EC. The aim of the risk-based approach in the development of ATMPs is to determine the extent of quality, non-clinical and clinical data to be included in the Marketing Authorisation Application (MAA), in accordance with the scientific guidelines relating to the quality, safety and efficacy of medicinal products and to justify any deviation from the requirements of this Annex.
The application of the risk-based approach in the preparation of a MAA dossier is optional. However, in cases where the risk-based approach is being applied, the applicant is advised to follow the methodology as laid down in the present guideline.