The World Federation of Hemophilia (WFH) has launched a new registry to help monitor the long-term safety and efficacy of hemophilia gene therapies in people with hemophilia across the globe.
The Gene Therapy Registry (GTR), which is now live, is designed to collect critical data on patients who have received a gene therapy through a clinical trial or via a product that has already come to market. By employing such a tool on an international scale, the GTR will capture even those sporadic and infrequent and safety events that might otherwise fall under the radar of global hemophilia community stakeholders.
"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."
WFH intends for the generated data to represent a valuable resource made available to patients, physicians, researchers, manufacturers, and regulatory agencies around the world.
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.
Source: Cision (PRNewswire), January 18, 2023