Presentation Details
| Gathering and Disseminating Standardized Gene Therapy Data – The World Federation of Hemophilia Gene Therapy Registry Barbara A.Konkle1, Flora Peyvandi2, Mayss Naccache3, Wolfgang Miesbach4, Brian O'Mahony5, Steven W.Pipe6, Mark W.Skinner7, Donna Coffin3, Glenn F.Pierce3. 1University of Washington, Seattle, WA, USA.2Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.3World Federation of Hemophilia, Montreal, QC, Canada.4University Hospital Frankfurt, Frankfurt, Germany.5Irish Haemophilia Society, Dublin, Ireland.6University of Michigan, Ann Arbor, MI, USA.7Institute for Policy Advancement Ltd, Washington, DC, USA |
Abstract
Introduction In this era of transformative biotechnology, the World Federation of Hemophilia (WFH) launched the Gene Therapy Registry (GTR) aimed at gathering comprehensive data on all people with hemophilia (PWH) who receive gene therapy worldwide. Methods The GTR is a prospective, observational, and longitudinal registry designed to standardize and centralize global data collection by establishing a single, unified data repository. Data entry occurs once, either directly into the GTR platform or through data transfer from National Registries. The GTR will disseminate specific de-identified data to various stakeholders. The GTR Scientific Advisory Board accesses global data for monitoring safety and efficacy. Hemophilia treatment centres (HTC) and National Registries will receive aggregated global safety data. Regulatory agencies and Health Technology Assessment organizations can request specific data to inform their decisions, while industry partners will receive product-specific data. Results The WFH is engaged with a broad network of HTCs and National Registries to establish mutually beneficial collaborations with the GTR. In North America specifically, the WFH GTR is collaborating with both the American Thrombosis and Hemostasis Network (ATHN) and the Canadian Bleeding Disorders Registry (CBDR) to ensure a common set of data is captured and easily linked to the GTR data set. De-identified data will be transferred from ATHN and the CBDR to the GTR on a regular basis. In the United States, hemophilia treatment centres can also enter data directly in the GTR, for patients not enrolled in ATHN. Similar collaborations have been established with other countries, including: Australia, Brazil, Ireland, Japan, France, Germany, Saudi Arabia, Spain, Sweden, the Netherlands, and the United Kingdom. Based on the GTR protocol, core data set and methodology, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has shown strong support for the GTR, endorsing it as the worldwide registry for consolidating all international data on PWH who receive gene therapy and encouraging collaboration of all HTCs and National Registries. The CHMP also emphasized the value of the GTR for post approval safety and efficacy studies of gene therapies, recommending its use as a planned data source for mandated Phase IV studies. Conclusion The GTR simplifies the data entry process, facilitates efficient data sharing, and provides valuable information to all stakeholders, advancing our understanding of gene therapy's safety, efficacy, and long-term effects, ultimately contributing to improved patient care and treatment outcomes.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.