Qualification Criteria of Gene Therapy for Haemophilia-Opinion of the EAHAD Gene Therapy Working Group

Authors: Miesbach, W; Boban, A; Chowdary, P; Coppens, M; Jimenez-Yuste, V; Klamroth, R; Mulders, G; Crato, M; Peyvandi, F

Affiliations: Medical Clinic 2, Institute of Transfusion Medicine, University Hospital Frankfurt, Frankfurt, Germany. Haemophilia Centre, Department of Haematology, University Hospital Centre Zagreb, Zagreb, Croatia. University of Zagreb School of Medicine, Zagreb, Croatia. Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK. Hemophilia Treatment Center, Department of Vascular Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam, Netherlands. Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, the Netherlands. Hematology Department, Hospital Universitario La Paz-IdiPaz, Autonoma University, Madrid, Spain. Department for Internal Medicine and Vascular Medicine, Haemophilia Treatment Center, Vivantes Hospital im Friedrichshain, Berlin, Germany. Institute of Experimental Hematology and Transfusion Medicine, University Hospital Bonn, Medical Faculty, University of Bonn, Bonn, Germany. Department of Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands. European Haemopilia Consortium, Brussels, Belgium. Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Milan, Italy. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.

Publication: Haemophilia; 2025

ABSTRACT: BACKGROUND: Following the approval of the first gene therapies for haemophilia, it is essential to develop an optimal infrastructure for the administration of gene therapy. This can be ensured by identifying the criteria for the definition of treatment centers (hub centers) and follow-up centers (spoke centers), as well as establishing effective cooperation between them. METHODS: The interdisciplinary members of the EAHAD Gene Therapy Working Group answered a survey to define requirements for centers participating in gene therapy care, addressing aspects such as product administration, coagulation parameter monitoring, and long-term safety surveillance. RESULTS: The majority support the implementation of standardized protocols. Hub centers are expected to maintain high standards of quality and flexibility, possess pharmacist expertise, ensure regulatory compliance, and have experience in gene therapy Spoke centers should be certified haemophilia centers, access to hepatologists and providing 24-h support. Prior to gene therapy, spoke centers manage initial patient interactions, while hub centers handle complex care needs. Post-therapy, both centers can monitor factor levels and liver health. However, hub centers are responsible for managing immunosuppression and facilitating specialist consultations. Collaboration between both centers is crucial for data sharing and the assessment and resolution of adverse events, emphasizing the importance of timely test results and regular liver imaging. CONCLUSION: The survey results highlight essential criteria for the safe and effective delivery of gene therapy through a structured hub-and-spoke model. These include accreditation, clinical trial experience, access to specialized healthcare professionals, and the establishment of standard operating procedures for monitoring and managing adverse events.