“The CHMP supports the WFH Gene Therapy Registry (GTR) as the worldwide registry for consolidating all international data on individuals with hemophilia who receive gene therapy and encourages collaboration of hemophilia treatment centres and national registries worldwide.”
The CHMP saw particular value in the utilisation of the WFH GTR for post-approval safety or efficacy studies of gene therapies. Consequently, they recommend its use as the planned data source for mandated Phase IV studies for new hemophilia treatments. This significant endorsement underscores the value of the GTR in harmonizing global, standardized data on PWH who receive gene therapy.
The EMA qualification process is a voluntary, scientific pathway designed to evaluate and endorse novel methods and tools used in the development and assessment of medicines within the European Union (EU). This procedure focuses on validating innovative techniques and technologies that can improve the evaluation and development of medical treatments.
The WFH initiated the qualification process for the GTR in August 2022. After thorough discussions between the WFH and the EMA regarding all aspects of the GTR—including the core dataset, data management and oversight of safety data, enrolment plan, governance, and stakeholder engagement strategies—the CHMP issued a letter of support, urging all countries to participate in the GTR, either through the participation of individual hemophilia treatment centres (HTCs) or through the transfer of data from established national registries.
Upon demonstrating its capability to collect and report data with a study context, the WFH will submit the GTR for evaluation to attain full qualification, marking another significant milestone in its pursuit of advancing hemophilia treatment and patient care.