PSY155 - Review of National Health Technology Assessment Reports on Treatment of Severe Haemophilia A

Abstract

To review the national health technology assessment (HTA) reports in Europe comparing on-demand with prophylaxis treatment of severe haemophilia A and to assess the consistency of methodologies and conclusions. The database of the Centre for Reviews and Dissemination and websites of the national HTA bodies were searched. The methodologies, results and conclusions of the retrieved reports were extracted and compared. Two reports on treatment of hemophilia were found: SBU (Sweden), 2011 and IQWIG (Germany), 2015. Both analyzed the effects of clotting factor concentrates therapy. In the review of evidence, SBU included systematic reviews, randomised controlled trials (RCTs), controlled clinical trials (CCTs), observational studies and review- and discussion papers; IQWIG included RCTs and CCTs with a minimum of 50 patients. The effectiveness of prophylaxis versus on-demand treatment in severe haemophilia A was judged by SBU based on one RCT, eleven observational studies, one retrospective data analysis and five review- and discussion papers; by IQWIG based on 3 RCTs: one in adolescents and adults (SPINART trial) and two in children (ESPRIT and JOS trials). Quality of evidence was assessed overall as low by SBU and IQWIG, with exception of the SPINART trial (low risk of bias). SBU reported insufficient but consistent evidence that prophylaxis initiated at a young age can prevent future joint damage. IQWIG concluded positive effect of prophylaxis on the number of severe bleeds in adolescents and adults; and sign of additional benefit in children, compared to on-demand treatment. A comparison of two national HTA reports on haemophilia treatment in Europe demonstrated inconsistencies in methodological approaches and generated conclusions. A European consensus on the effectiveness assessment of therapies in rare diseases, such as haemophilia, and appropriate patient involvement would enhance common understanding of therapeutic value of treatments and potentially improve patient access in countries where access is limited.