EMA workshop 'Haemophilia registries'
On 8 June 2018 Kathelijn Fischer and Mariette Driessens participated on behalf of HemoNED in the haemophilia registries workshop organized by the European Medicines Agency (EMA). You will soon find a report of this workshop on the EMA website.
The workshop discussed how national registers can be set up in a good way and in accordance with the revised FVIII Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products (link) in order to facilitate the long-term follow up of haemophilia therapy in a real world setting. The authorities want to replace PUP (previously untreated patient i.e. the babies with haemophilia) studies and post-marketing studies with data from registers, because it is shown that the information is collected equally reliably, but is expected to be available more quickly for patients and practitioners. An additional advantage is the prevention of the considerable burden that PUP studies entail for patients and their parents, the practitioners and the pharmaceutical companies.
The collection of data through national registers is important because it allows the normal treatment of haemophilia over a longer period of time to be monitored and the use of new products (such as PEGylated products, monoclonal antibodies and gene therapies) can be evaluated.
One of the outcomes of the workshop was that there should be a European platform where data from various national registers are collected and analysed. This is a complicated process and requires a lot of efforts from all kinds of parties, both in the Netherlands and the rest of Europe.
More information can be found in the appendix.