The first artificially made treatment for people with severe types of von Willebrand disease (VWD) has been refused funding by NHS England.
This means that people with VWD who need factor cover during surgery or for bleeding complications must continue to rely on plasma-based treatment.
Last month, NHS England’s Clinical Priorities Advisory Group decided that it did not have the funds to introduce the new drug this April. However, it did approve funding for Coagadex which will have a life-changing impact on people with a factor X deficiency.
Liz Carroll, Chief Executive of The Haemophilia Society, said: “While I am delighted that people with a factor X deficiency will receive such a transformative new treatment, I am very disappointed that the first recombinant treatment for von Willebrand disease will not be funded.
“It cannot be right that more than 20 years after the first recombinant products became available for haemophilia treatment, people with severe types of VWD must still rely on plasma-based products. They need and deserve the reassurance of a recombinant treatment option.”
The on-going Infected Blood Inquiry has heard evidence from witnesses who were infected with hepatitis viruses as a result of treatment for their VWD in the 1970s and 80s. One witness told the inquiry that she was so distrustful of treatment that she “utterly and absolutely refused” to allow her son, who also has VWD, to receive blood products.
Veyvondi, also known as Vonicog Alpha, works in the same way as von Willebrand factor made by the body itself, by replacing the protein needed to stop bleeding that is missing or not working.
Coagadex contains more than 94 per cent factor X, which means for the first time those with a deficiency can receive targeted treatment. Until now the only option was to take treatments designed for other conditions meaning people with factor X deficiency were taking extra proteins that they didn’t need, potentially increasing the risk of blood clots.
The Haemophilia Society had argued for Veyvondi and Coagadex to be adopted.