Event Report: EHC Round Table of Stakeholders on ‘Clinical Trials in Haemophilia’

Haemophilia is a rare and congenital bleeding disorder caused by a genetic defect, resulting in a lack of or insufficient coagulation factors VIII or IX in the body. In affected individuals, this causes an inability to clot blood, leading to bleeds in the joints, muscles and soft tissues. If left untreated, this can lead to disability and sometimes death. It is widely agreed that the optimal standard for haemophilia treatment is prophylactic substitution therapy, i.e. the regular infusion of the missing coagulation factor.