21 / 2020
Haemophilia is a rare congenital blood coagulation disorder, which needs life-long treatment. After research and development has recently brought about important breakthroughs in the development of several new approaches to treatment, these medicines are now approaching the marketing authorisation. Thus, the legal and regulatory requirements for the German Haemophilia Registry at the Paul-Ehrlich-Institut (DHR) are also increasing. In a recent paper, researchers at the Paul-Ehrlich-Institut describe how the DHR meets the new requirements (Journal of Clinical Medicine, online edition of 24 October 2020).
Haemophilia A and haemophilia B are rare congenital blood coagulation disorders recognisable by frequent long-lasting bleeding. For a long time, treatment was possible only by administering the missing blood coagulation factors VIII or IX. In the past ten years, however, various new therapy approaches were developed, which might mean more effective treatment and more quality of life for persons with haemophilia. These include modified blood coagulation factors with longer half-lives and antibody or RNA based therapies and gene therapeutics. Several promising products have already been authorised or will be authorised shortly. This will mean new chances and challenges at a regulatory level. These shall, among other things, be met by a stronger involvement and cooperation of the different registries within Europe.
Since 2008, the Paul-Ehrlich-Institut in cooperation with the two patient organisations Interessengemeinschaft Hämophiler e.V. (Self-help organisation of haemophiliacs, IGH) and Deutsche Hämophiliegesellschaft (German Haemophilia Society, DHG) and the medizinische Gesellschaft für Thrombose und Hämostaseforschung (medical Society for Thrombosis and Haemostasis Research, (GTH) have been managing the German Haemophilia Registry Deutsches Hämophilieregister (DHR). The data collected in this registry on haemophilia and other coagulation disorders on the one hand help ensure the supply of patients with the necessary medicines, and, on the other hand, provide valuable research data in themselves for studying these rare disorders. To address the current technical, scientific, and regulatory challenges, the DHR was reorganised. Since mid-2019, it can be accessed on a new platform geared to these framework conditions. This not only assures that the DHR is up-to-date from a professional point of view but also facilitates the cooperation with other European haemophilia registers – a major step in gaining important insights into future therapies through the analysis of extensive data sets.
Updated: 18.11.2020
