On November 28th, 2017, the European Haemophilia Consortium (EHC) held its third and final for the year Round Table of Stakeholders on ‘Usage and measurement of extended half-life (EHL) coagulation factor concentrates and non-substitutional therapies.’
The event took place at the Sofitel Hotel in Brussels, Belgium and was chaired by Professor Mike Makris, who is the Director of the Sheffield Haemophilia and Thrombosis Centre in the UK and a member of the EHC Medical Advisory Group.
Over 40 participants representing medical researchers, health care professionals, patient organisations and the pharmaceutical industry gathered to discuss the novel therapies on the market. As it is well known, the optimal standard for haemophilia treatment is prophylactic substitution therapy, but current products still have significant shortcomings, such as frequent intravenous infusions and the risk of developing inhibitors. The emergence of extended half-life (EHL) factor concentrates and non-substitutional therapies bring long-awaited improvements in treatment but also present many questions on safety, inhibitor development, bleeding rates, etc. To this end, the Round Table programme focused on current use of EHL and non-substitutional therapies and the monitoring of their safety and efficacy. The event ended with a comparative assessment of standard treatment vs novel technologies and a panel discussion on what haemophilia treatment will look like in 2027.
Below you can find documents provided during the event. All pictures from the event can be viewed on the EHC Facebook page.
- Speakers’ biographies
- List of Participants
- Round Table report
- Current use of EHL and non-substitutional therapies by Professor Roger Schutgens
- Monitoring efficacy: Measuring EHL and non-substitutional therapies by Dr Annette Bowyer
- Monitoring safety: Pharmacovigilance programmes for EHL and non-substitutional therapies by Mrs Caroline Voltz-Girolt
- Assessing value: Comparative assessment of standard treatment vs novel technologies by Dr Niamh O’Connell
- Panel discussion: what will haemophilia treatment look like in 20207?
You can also find an article summarising the discussions held during the event in the EHC December 2017 Newsletter, available here.