How can the safety of biotherapy products be ensured for patients?

• Published in: Therapie Vol. 59; no. 3; p. 305
• Main Authors: Brailly, Hervé, Postaire, Eric, Zorzi, Pierrette
• Format: Journal Article
• Language: English
• Published: France 01.05.2004
• Subjects: Biological Products - therapeutic use; Clinical Trials as Topic - standards; Humans; Safety; Stem Cell Transplantation; Transplantation, Autologous
• ISSN: 0040-5957
• DOI: 10.2515/therapie:2004059

The topic of the round table being very broad, it was agreed with the participants that the discussion should be focused on autologous cell therapy (CT) used in tissue repair, immunomodulation or gene transduction. Autologous CT is actually comprised of both very innovative procedures as well as of products used in routine clinical practice. In France, the regulatory framework for CT has now been finalised, underlining the fact that a CT product (CTP) is intrinsically linked to the process used in its preparation. The objective of this round table was to define the essential prerequisites for the development of therapies involving ex vivo cell preparations and, more specifically, to address the issues associated with patient safety during the course of product development. The different stages of CTP development were considered: (i) requirements for CTP manufacturing in order to guarantee product quality; (ii) nonclinical development, and selection of appropriate animal models to provide the proof of the concept and support the definition of CTP specifications; and (iii) clinical development and methodological specificities of CTP development. The specific problems associated with the use of ancillary therapeutic products (ATPs) during the development of a CTP were discussed. Actions were proposed to ensure long-term supply of ATPs, which may be a significant bottleneck for the development of CT in France. Eventually, new regulatory provisions will come into force in the area of biovigilance, and it was deemed necessary for healthcare and industry professionals to be appropriately involved in the implementation phase.