Impact of Advanced Therapy Medicinal Products status in European regulations on their production cost: The case of hematopoietic stem cells.

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The therapeutic promises of somatic cell and gene therapy (Advanced Therapy Medicinal Products - ATMP) and the significant current or future costs of such therapies raise questions about the impact of European health policy on these expenses. Regulation 1394/2007 currently imposes the medicinal product status on many treatments derived from cell therapy. This study aims to determine the impact of new regulations on manufacturing costs of cell therapy treatments, used as ATMP. In our study, using counterfactual reasoning, we analyze the consequences that a change of status would have on the hematopoietic stem cells (HSC) used for treatment of leukemia. Under the existing status of cell transplantation (CT, Directive 2004/23/EC), the annual production cost of these cell therapy products (CTP) in France is estimated at euro 5.6 million. If the HSC used in leukemia indication were under the medicinal product status of ATMP, the cost would rise to approximately euro 9.9 million, without accounting for expenses related to facilities, which are much higher for ATMP than for CT. This additional burden for manufacturers must be weighed against the theoretical benefits for patients, such as reduced contamination risk. Given the very low contamination levels reported by facilities using transplant standards, the extra cost seems excessive and could hinder the development of new advanced therapies. Furthermore, it raises the need to adapt existing Good Manufacturing Practice guidelines for autologous products or those made from a single donor for a single patient, ensuring safety and cost reduction, which should encourage further development of cell therapy.