The challenges of Advanced Therapy Medicinal Products manufacturing in the European Union: Strengths and limits of current regulatory tools

Luc-Sylvain Gilbert (1) , Auxane Delage (1) , Hsin-Yu Kuo (2) , Anna Couturier (2) , Aurélie Mahalatchimy (1)

1 DICE / CERIC - Droits International, Comparé et Européen / Centre d'études et de recherches internationales et communautaires (CERIC)
2 Edin. - University of Edinburgh

Advanced Therapy Medicinal Products (ATMPs) is a European classification of medicinal products based on genes, cells and tissues specifically regulated in the European Union (EU) from 2007. Any company wishing to manufacture ATMPs in the EU must hold a manufacturing authorisation issued by the national competent authority of the Member State where they carry out these activities. Ensuring the quality of medicines is a criterion to obtain ATMPs’ marketing authorisation issued by the European Commission under the “centralised procedure” after a single application to the European Medicines Agency. All ATMPs marketed in the EU must be produced in accordance with EU quality standards to ensure the quality, safety and efficacy of medicines for the patients: Good Manufacturing Practice (GMP) principles and guidelines dedicated to ATMPs enforceable since 2017, and relevant parts of the European Pharmacopeia provide the main regulatory standards to comply with. The manufacture, meeting the GMP requirements from the raw materials to the final product, is particularly challenging for ATMPs regarding their level of complexity and their specific characteristics, especially the use of substances of human origin as starting materials, and the reproducibility when using live biological samples. The ATMPs’ manufacturer has to maintain costly technical specifications capable of guaranteeing the reproducibility of the medicine’s composition in accordance with GMP guidelines and the specific requirements of the ATMP marketing authorisation. Regulatory requirements are often seen as obstacles to the development of ATMPs. Nevertheless, they mainly provide a set of solutions to overpass the technical obstacles of ATMPs’ manufacturing in order to ensure their quality for the safety of patients. It appears clearly within the various supporting regulatory tools currently available at the European level, which include both guidance documents and procedures for interactions with regulators. This poster will provide an overview of the challenges of ATMPs’ manufacturing and link them to the relevant supporting regulatory tools highlighting both their strengths and their limits in the context of ATMPs development for patients.

Domains: Humanities and Social Sciences; Law

Luc-Sylvain Gilbert, Auxane Delage, Hsin-Yu Kuo, Anna Couturier, Aurélie Mahalatchimy. The challenges of Advanced Therapy Medicinal Products manufacturing in the European Union: Strengths and limits of current regulatory tools. European Society for Gene & Cell Therapy Congress 2023, Oct 2023, Bruxelles (BEL), Belgium. ⟨halshs-04290578⟩