Department of Pathophysiology and Transplantation, Angelo Bianchi Bonomi Haemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Università degli Studi di Milano, Luigi Villa Foundation, Milan, Italy.
Haemophilia. 2014 May;20 Suppl 4:137-44. doi: 10.1111/hae.12415.
Haemophilia therapy is experiencing an unprecedented expansion in the number and novelty of clotting factor concentrates. Every product must be licensed by regulatory authorities, primarily on the basis of its safety and efficacy profiles. The low prevalence of haemophilia, and other inherited bleeding disorders, presents a significant challenge to patient recruitment for preauthorization clinical trials, especially given the low frequency of inhibitory antibodies, the major adverse event related to clotting factor exposure. Other challenges include a lack of harmonization between the major regulatory authorities in certain key areas, the selection of laboratory monitoring methodologies and the difficulty in obtaining high-quality phase IV safety data following authorization. These aspects will be reviewed in this session, which will also highlight the roles played by the World Federation of Hemophilia and International Society on Thrombosis and Haemostasis in the promotion of these discussions.
血友病疗法在凝血因子浓缩物的数量和新颖性方面正在经历前所未有的扩展。每个产品都必须经过监管机构的许可，主要依据其安全性和疗效特征。血友病和其他遗传性出血性疾病的低患病率给患者招募预先授权的临床试验带来了重大挑战，尤其是考虑到抑制性抗体的低频率，这是与凝血因子暴露相关的主要不良事件。其他挑战包括在某些关键领域主要监管机构之间缺乏协调、实验室监测方法的选择以及在获得授权后获得高质量的 IV 期安全性数据的困难。本环节将对这些方面进行审查，同时还将强调世界血友病联盟和国际血栓与止血学会在促进这些讨论方面所发挥的作用。
