Chapter 7. European regulation applicable to stem cell–based medicines

The development of advanced therapy medicinal products based on the use of stem cells now represents one of the most significant developments in contemporary clinical research. Human stem cells offer the potential to replace deficient cells and to act on the biological functioning of a patient’s body, thereby enabling the development of individualised therapeutic strategies characteristic of regenerative and personalised medicine. The conversion of human biological materials into therapeutic products through complex scientific and industrial processes confers on stem cells a dual medical and economic dimension, raising significant ethical concerns. All stages of the life cycle of these products – from development to marketing – are governed by a specific, complex and demanding European regulatory framework, which must continuously adapt to scientific and technological advances. Clinical research conducted on these products raises key issues, notably those relating to respect for the free and informed consent of both donors and recipients, heightened requirements for traceability, safety and transparency, as well as the challenge of ensuring equitable access to costly therapies within resource-constrained healthcare systems once these products reach the market. Although the clinical trials undertaken to develop these medicines have led to major biotechnological advances, they remain inseparable from substantial scientific, clinical and ethical challenges linked to the complexity of their manufacturing processes, their biological instability, and the need for long-term follow-up of participants. Against this background, the article provides an analysis of the European regulatory framework applicable to stem cell-based advanced therapy medicinal products, while highlighting the persistent tensions between scientific innovation, ethical requirements, patient safety and the economic sustainability of this therapeutic model.