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    Has all of the relevant evidence been taken into account?

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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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1 Recommendations

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1 Recommendations

1.1

Efanesoctocog alfa is not recommended, within its anticipated marketing authorisation, for treating and preventing bleeding episodes in people with haemophilia A (congenital factor VIII deficiency).

1.2

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, the company asked for efanesoctocog alfa to be considered only for people with severe haemophilia A. This does not include everyone who it is anticipated to be licensed for.

Current treatment for severe haemophilia A includes factor VIII replacement therapies (such as efmoroctocog alfa) or emicizumab to prevent bleeding, and on-demand factor VIII replacement therapies to treat bleeding.

The results from 1 clinical trial show fewer bleeding episodes for people having ongoing efanesoctocog alfa compared with on-demand efanesoctocog alfa. There is limited clinical-effectiveness evidence from direct comparisons of efanesoctocog alfa with any other treatment for severe haemophilia A. An indirect comparison suggests that efanesoctocog alfa may reduce the number of bleeding episodes compared with efmoroctocog alfa. The results of an indirect comparison of efanesoctocog alfa with emicizumab are unreliable, so whether one works better than the other is unknown.

It is not possible to reliably estimate the cost effectiveness of efanesoctocog alfa because:

  • of the uncertainties in the clinical-effectiveness evidence and economic modelling

  • the company did not provide evidence comparing it with all relevant factor VIII replacement therapies.

So, efanesoctocog alfa is not recommended.