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) and the European Medicines Agency (EMA).

The WFH Gene Therapy Registry (GTR) aims to collect important data on all patients who receive gene therapy, whether through a clinical trial or through a post-marketed product. The data collected will be a valuable resource for patients, physicians, researchers, manufacturers, and regulatory agencies. By providing valuable patient-reported information about their own experience with gene therapy via myGTR, a web-based patient engagement tool, patients can play an active role in helping researchers better understand gene therapy for hemophilia.

Patient safety is all of our responsibility. 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.

—Glenn Pierce, MD, PhD, WFH Vice President Medical

Gene therapy is an exciting addition to the treatment landscape for people with hemophilia, their families and their healthcare teams. However, because clinical trials are limited in terms of the follow-up duration of patients prior to regulatory approval, questions on the long-term safety and efficacy of gene therapy for hemophilia remain unanswered at the completion of clinical trial programs. These questions are best answered using data derived from large cohorts of patients who have received gene therapy. Ultimately, it will be the accumulation of data from a large number of patients enrolled in one global registry that will allow us to identify unexpected safety events associated with this new technology, monitor known safety events, and determine the durability and the variability of gene therapy products. Detecting low incident or delayed safety events—particularly in rare diseases such as hemophilia—necessitates that each PWH who receives gene therapy be followed over the long term, preferably their lifetime, in a single global registry that has, as its goal, the inclusion of all treated patients. The WFH GTR will also be used to help establish long-term follow-up as the standard of care for gene therapy.

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 WFH Founding visionary partners Biomarin, CSL Behring, Pfizer, and Spark; and WFH Collaborating partner Takeda.

SOURCE: WFH