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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]

Background: The prevalence of hemophilia varies globally, with close to 100% of patients diagnosed in high-income countries and as low as 12% diagnosed in lower-income countries. These inequalities in the care of people with hemophilia exist across various care indicators., Objectives: This analysis aims to describe the clinical care outcomes of patients in the World Bleeding Disorders Registry (WBDR)., Methods: In 2018, the World Federation of Hemophilia developed a global registry, the WBDR, to permit hemophilia treatment centers to collect clinical data, monitor patient care longitudinally, and identify gaps in management and treatment., Results: As of July 18, 2022, 10,276 people with hemophilia were enrolled from 87 hemophilia treatment centers in 40 countries. Nearly half (49%, n  = 5084) of patients had severe hemophilia; 99% were male, 85% had hemophilia A, and 67% were from low-middle-income countries. Globally, the age of diagnosis for people with severe hemophilia has improved considerably over the last 50 years, from 82 months (∼7 years) for those born before 1980 to 11 months for those born after 2010, and most prominently, among people with severe hemophilia in low- and low-middle-income countries, the age of diagnosis improved from 418 months (∼35 years) for those born before 1970 to 12 months for those born after 2010. Overall, the age of diagnosis of people with hemophilia in low- and low-middle-income countries is delayed by 3 decades compared to patients in upper-middle-income countries and by 4 decades compared to patients in high-income countries., Conclusion: Data reveal large treatment and care disparities between socioeconomic groups, showing improvements when prophylaxis is initiated to prevent bleeding. Overall, care provided in low-income countries lags behind high-income countries by up to 40 years. Limitations in the interpretation of data include risk of survival and selection bias., (Crown Copyright © 2023 Published by Elsevier Inc. on behalf of International Society on Thrombosis and Haemostasis.)