Highlights From the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders 2025
Enhancing Global Collaboration: The Role of Data Governance in the WFH Gene Therapy Registry
Presenting Author: Mayss Naccache
Konkle BA,1 Peyvandi F,2 Naccache M,3* Youttananukorn T,3 Newman V,4 Coffin D,3 Pierce GF3
- University of Washington, Seattle, United States
- Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
- World Federation of Hemophilia, Montreal, Canada
- Garthorne Medical and Clinical Limited, Witheridge, United Kingdom
Overview
The WFH Gene Therapy Registry (GTR) is a global database enrolling all patients who receive gene therapy for hemophilia, with oversight from the WFH Scientific Advisory Board (SAB). Developed with input from EMA and FDA, it aims to determine the long-term safety, efficacy, and durability of factor VIII and IX gene therapies while assessing patient quality of life post-infusion. Data is de-identified, with ownership retained by HTCs and national registries. Access requires formal requests reviewed by SAB, ensuring confidentiality and controlled sharing with stakeholders. Safety events are reported to national authorities and entered into the GTR, with real-time alerts and regulatory follow-ups. Quarterly reviews and expert assessments ensure data accuracy, transparency, and timely stakeholder communication.
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