On March 7th 2017, the European Haemophilia Consortium (EHC) held its first Round Table of Stakeholders of the year on ‘Clinical Trials in Haemophilia’.
The event, which took place in the European Parliament, was hosted by Member of the European Parliament Mrs Norica Nicolai (Romania/ALDE), and additionally attended by MEP Dr Miroslav Mikolášik (Slovakia/EPP). Both are longtime supporters of EHCs’ efforts to improve the quality of life for the haemophilia community.
More than 50 participants were present, including patient representatives, healthcare professionals and representatives from the pharmaceutical industry. After welcoming remarks, the scientific part of the programme was kicked off with an introduction to how epidemiological data in the field of haemophilia is collected. The presentations that followed build on the topic by covering aspects such as drug and side-effects, how long-term observation of efficacy for novel therapies is carried out, the safety of these therapies, as well as the angle of previously untreated patients (PUPs) in clinical trials.
Clinical trials are of crucial importance for all patients. But one of the big dilemmas in haemophilia is what causes inhibitors – the antibodies acting against the received treatment. Those affected by inhibitors are unable to continue their regular treatment regimen and are left more vulnerable to devastating physical and psychological effects, such as disability and social isolation. It is now of great significance to observe whether new treatments will also cause inhibitors and whether they can be used to treat people with inhibitors at all.
Pictures from the day can be viewed on the EHC Facebook page.