MONTREAL, Jan. 18, 2023 To ensure patient safety remains at the forefront of the work of the World Federation of Hemophilia (WFH), we have developed a registry to monitor the long-term safety and efficacy of gene therapy for people with hemophilia: the WFH Gene Therapy Registry (GTR). The launch of the WFH GTR coincides with the approval of gene therapy for hemophilia by both the United States Food and Drug Administration (FDA)¹ and the European Medicines Agency (EMA)².
This worldwide endeavour aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product. Clinical data from around the world will be combined in this registry and used to answer critical questions on the long-term safety and efficacy of gene therapy in hemophilia. Incorporating data in one global registry is the best approach to enable detection of low incident and delayed safety events in a rare disease such as hemophilia. The data collected will be a valuable resource for patients, physicians, researchers, manufacturers, and regulatory agencies.
“Patient safety is all of our responsibility,” explains Glenn Pierce, MD, PhD, WFH Vice President, Medical. “Collecting data in one global registry—the WFH GTR—is essential to ensure that rare adverse events, in a small patient population over a large geographical area, will be detected.”
The WFH GTR was developed in collaboration with the International Society on Thrombosis and Haemostasis (ISTH), the European Haemophilia Consortium (EHC), the US National Hemophilia Foundation (NHF), the American Thrombosis and Hemostasis Network (ATHN), Patient-Reported Outcomes Burdens and Experiences (PROBE) study group, manufacturers, and experts in the field of gene therapy. The WFH is proud to partner with the United BioSource Corporation and Lifelink Systems in the technological development of the WFH GTR.
The WFH GTR is now ready to enroll all PWH who have received gene therapy, through a clinical trial or through a marketed product. Hemophilia treatment centres and national registries interested in participating in the WFH GTR should contact us at GTR@wfh.org.
The Gene Therapy Registry is supported by:
Founding visionary partners: Biomarin, CSL Behring, Pfizer, Spark
Collaborating partner: Takeda
¹FDA approval: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b
²EMA approval – Roctavian / HemA: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-severe-haemophilia. Hemgenix / HemB: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b
About hemophilia and other bleeding disorders
In people with bleeding disorders, the blood clotting process doesn’t work properly, with the result that they can bleed for longer than normal, and some people may experience spontaneous bleeding into joints, muscles, or other parts of their bodies which can lead to developmental and permanent mobility issues. The overwhelming majority of people living with inherited bleeding disorders around the world still do not have access to diagnosis, treatment and care.
About the World Federation of Hemophilia
The World Federation of Hemophilia (WFH) is a non-profit organization dedicated to improving and sustaining care for people with inherited bleeding disorders around the world. Our vision of Treatment for All is for a world where all people with inherited bleeding disorders have access to care, regardless of their type of bleeding disorder, gender, or where they live.
We work in partnership with healthcare providers (HCPs), governments, and our global network of national member organizations (NMOs) in 147 countries. We provide our NMOs and healthcare providers with the knowledge and tools they need to identify, support and treat people living with bleeding disorders in their communities, while promoting global advocacy and collaboration to achieve our common goals.
To find out more about the WFH, please visit www.wfh.org.