NICE UK Positive for Altuvoct in Hemophilia A

i) they have a factor VIII activity level of less than 1% (severe haemophilia A); ii) the company provides it according to the commercial arrangement. This recommendation is not intended to affect treatment with efanesoctocog alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children and young people, this decision should be made jointly by them, their healthcare professional, and their parents or carers. Why the committee made these recommendations - For this evaluation, efanesoctocog alfa was only considered for people with severe haemophilia A, in line with the evidence provided by the company. This does not include everyone who it is licensed for. Current treatment options for severe haemophilia A include ongoing treatment with factor VIII replacement therapies (including standard half-life and extended half-life therapies) or emicizumab to prevent bleeding. On-demand factor VIII replacement therapies are used to treat bleeding. The results from a clinical trial suggest that there may be fewer bleeding episodes with ongoing efanesoctocog than with previous ongoing factor VIII replacement therapy, but this is uncertain. There is limited clinical-effectiveness evidence directly comparing efanesoctocog alfa with currently available treatments for severe haemophilia A, and there are substantial limitations with the available indirect comparisons. So, it is uncertain how well efanesoctocog alfa works compared with other haemophilia A treatments. Because of uncertainties in the clinical-effectiveness evidence and the economic model, the cost-effectiveness estimates are uncertain. But, when considering all the available evidence and economic analyses, efanesoctocog alfa is a cost-effective use of NHS resources. So, it is recommended.