Post-authorization pharmacovigilance for hemophilia in Europe and the USA: Independence and transparency are keys

Abstract

The regulatory process is currently designed to ensure drug safety during the pre-marketing process. Due to limitations of the pivotal studies owing to their short duration and small number of highly selected cases, some adverse drug reactions (ADRs) are not detected, particularly the rarest and those which appear a long time after exposure. Thus it is essential to foster pharmacovigilance run totally independently from marketing authorization holders in order to attain transparent surveillance. This challenge is relevant for hemophilia drugs, after recent advances offered innovative replacement and non-replacement medicines that warrant monitoring due to the occurrence of predicted but also unexpected and rare ADRs. This article contains an overview of pharmacovigilance systems in the European Union and USA, describing the main ADRs related to traditional and recently available hemophilia medications and suggests how to involve stakeholders other than marketing authorization holders for an independent and transparent pharmacovigilance targeted to detect not only early ADRs but also those emerging post-marketing.

Keywords: Concizumab; Emicizumab; Fiturisan; Gene therapy; Monoclonal antibody; Thrombosis.