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    Has all of the relevant evidence been taken into account?

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

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Draft guidance consultation

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1 Recommendations

1.1

Marstacimab is not recommended, within its marketing authorisation, for preventing bleeding episodes (prophylaxis) caused by severe haemophilia A (congenital factor VIII [8] deficiency) or severe haemophilia B (congenital factor IX [9] deficiency) in people 12 years and over, weighing at least 35 kg, without factor inhibitors (anti-factor antibodies).

1.2

This recommendation is not intended to affect treatment with marstacimab that was started in the NHS before this guidance was published. People having treatment outside this 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 or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.

Why the committee made this recommendation

People with severe haemophilia A usually have prophylaxis with factor 8 replacement therapy or emicizumab to prevent bleeding episodes. People with severe haemophilia B have prophylaxis with factor 9 replacement therapy.

Evidence from a clinical trial shows marstacimab reduces the number of bleeding episodes a person has compared with factor 8 or 9 prophylaxis. There is no trial directly comparing marstacimab with emicizumab. An indirect comparison suggests marstacimab reduces bleeding episodes by a similar number as emicizumab. But the evidence for this is highly uncertain.

There are also important uncertainties in the economic model and it is not possible to determine the most likely cost effectiveness estimates for marstacimab. So, marstacimab is not recommended.

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