PB0223 - Standardized and Centralized Global Database on Gene Therapy Data – The World Federation of Hemophilia Gene Therapy Registry

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

TY

Toong Youttananukorn

Data Quality Manager
World Federation of Hemophilia
Montreal, Quebec, Canada
Barbara A. Konkle

Professor of Medicine
Washington Center for Bleeding Disorders and the University of Washington, Seattle, WA, USA
Seattle, Washington, United States
Flora Peyvandi, MD, PhD

Full Professor
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Università degli Studi di Milano, Department of Pathophysiology and Transplantation
Milan, Lombardia, Italy
MN

Mayss Naccache

Head of Gene Therapy
World Federation of Hemophilia
Montreal, Quebec, Canada
Wolfgang Miesbach, MD

Professor and Head of Coagulation Disorders and Comprehensive Care Centre
University Hospital Frankfurt, Frankfurt, Germany
Frankfurt, Hessen, Germany
BO

Brian O'Mahony

Chief Executive
Irish Haemophilia Society
Dublin, Dublin, Ireland
Michael Makris, MD

University of Sheffield
Steven W. Pipe, MD

Professor of Pediatrics and Pathology
University of Michigan
Ann Arbor, Michigan, United States
MS

Mark W. Skinner

President
Institute for Policy Advancement Ltd
Washington DC, Washington, United States

Presenting Author(s)

DC

Donna Coffin

Director
World Federation of Hemophilia
Montreal, Quebec, Canada

Co-Author(s)

GP

Glenn F. Pierce

Vice President, Medical
World Federation of Hemophilia
Montreal, Quebec, Canada

Background: The World Federation of Hemophilia (WFH) has launched the Gene Therapy Registry (GTR) aimed at collecting clinical and patient reported outcome (PRO) data on all people with hemophilia (PWH) who receive gene therapy (GT).

Aims: To develop a global, centralized database collecting long-term data on the safety and efficacy of GT.

Methods: Clinical data are collected either directly from hemophilia treatment centres (HTCs) via the GTR platform or via data transfer from National Registries. PRO data, including bleeds, treatments and health related quality of life, are collected through myGTR – a patient engagement tool (table 1).

The GTR Scientific Advisory Board is responsible for oversight of all data in the registry. Aggregated, de-identified clinical and PRO data will be disseminated to different stakeholders. Global safety data will be shared with HTC and National Registries. Regulatory agencies and Health Technology Assessment organizations can request specific data to inform their decisions. Industry partners will receive product-specific data.

Results: To foster dialogue, the GTR National Registries and HTC Consortium has been established. To address potential data gap when patients reduce their HTC visit schedule the WFH designed myGTR to be a simple tool where the patients will be prompted (4 times during the first year, 2 times per year thereafter) to answer 2 simple questions about their health status since GT infusion.

The European Medicines Agency Committee for Medicinal Products for Human Use has shown strong support for the GTR by endorsing it as the worldwide registry for consolidating data on PWH who receive GT.

Conclusion(s): The GTR streamlines data collection and sharing processes for all stakeholders. PRO data from myGTR are vital to assess quality of life and burden of disease post GT infusion. The GTR and myGTR enable the advancement in our knowledge and understanding of GT's safety, efficacy, and long-term effects.