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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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    Are the recommendations sound and a suitable basis for guidance to the NHS?

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Draft guidance consultation

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1 Recommendations

1.1 Etranacogene dezaparvovec is not recommended, within its marketing authorisation, for treating moderately severe or severe haemophilia B (congenital factor IX deficiency) in adults without a history of factor IX inhibitors (antibodies against factor IX).

1.2 This recommendation is not intended to affect treatment with etranacogene dezaparvovec 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 clinician consider it appropriate to stop.

Why the committee made these recommendations

People with moderately severe or severe haemophilia B usually have long-term treatment with factor IX (FIX) concentrates to prevent bleeding episodes (prophylaxis) and on-demand FIX concentrates to stop bleeding during a bleeding episode. A small number of people with the condition opt to only have on-demand treatment.

Evidence from a clinical trial suggests that etranacogene dezaparvovec reduces the number of bleeding episodes a person has each year. But there is not enough evidence on how well it works in the long term.

An indirect comparison of etranacogene dezaparvovec with FIX prophylaxis treatments suggests that it improves bleeding outcomes. But there are problems with this evidence, such as differences between studies in the methods used, and the definition and measurement of bleeding outcomes. So, the indirect comparison results are highly uncertain.

The cost-effectiveness estimates for etranacogene dezaparvovec are uncertain because of uncertainties in the long-term clinical evidence and some of the assumptions used to estimate cost effectiveness. They are also above what NICE considers an acceptable use of NHS resources. So etranacogene dezaparvovec is not recommended.