Landmark endorsement of a global registry: The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard.

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has publicly endorsed the World Federation of Hemophilia (WFH) Gene Therapy Registry as the global standard for consolidating international data on individuals with hemophilia who receive gene therapy. This endorsement highlights the importance of the registry in ensuring patient safety and uniting all stakeholders involved in hemophilia treatment. The registry collects comprehensive, long-term data on individuals with hemophilia who undergo gene therapy and aims to assess the safety, efficacy, and quality of life of patient recipients. The registry follows strict international regulations and standards of data privacy and has a multi-stakeholder governance structure to ensure wide representation. The funding for the registry comes from the manufacturers of gene therapy products, and industry representatives are included in the governance committees but are not involved in decision-making regarding data analysis or reporting. The endorsement by the CHMP is significant as gene therapy products for hemophilia are being approved for use in multiple countries, and the registry provides a centralized repository of standardized global data for robust analyses and identification of safety events. The registry benefits patients, hemophilia treatment centers, national registries, industry partners, regulatory agencies, and Health Technology Assessment organizations. Overall, the endorsement emphasizes the importance of collaboration among all stakeholders in ensuring the safety of individuals with hemophilia who receive gene therapy. [Extracted from the article]