The German Hemophilia Registry: Growing with Its Tasks

Journal of Clinical Medicine

Anneliese Hilger¹  Christine Keipert²  Birgit Haschberger²  Janina Hesse²  Heike Duda² 

[1] Division Haematology/Transfusion Medicine, Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, 63225 Langen, Germany;dhr Office, Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, 63225 Langen, Germany;

关键词: hemophilia;    patient registries;    gene therapy;    EHL clotting factor concentrates;    nonreplacement therapies;    inhibitors;   

DOI  :  10.3390/jcm9113408

来源: DOAJ

【 摘 要 】

Hemophilia is a rare heredity bleeding disorder that requires treatment for life. While few therapeutic options were available in the past, multiple recent breakthroughs have fundamentally altered and diversified hemophilia therapy, with even more new therapeutic options forthcoming. These changes are mirrored by significant regulatory and legal changes, which have redefined the role of hemophilia registries in the European Union (EU). This dual paradigm shift poses new regulatory, scientific but also structural requirements for hemophilia registries. The aim of this manuscript is to enumerate these significant challenges and to demonstrate their incorporation into the redesign of the German Hemophilia Registry (Deutsches Hämophilieregister, dhr). To identify the spectrum of hemophilia therapies and the degree of regulatory changes, a horizon screening was performed. Consequently, a core dataset for the dhr was defined by harmonization with regulatory guidelines as well as other hemophilia registries and by heeding the needs of different stakeholders (patients, clinicians, regulators, and scientists). Based on this information, a new registry structure was established, which is optimized for capturing data on new and established hemophilia therapies in a changing therapeutic and regulatory landscape

【 授权许可】

Unknown