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  • Question on Document

    Has all of the relevant evidence been taken into account?

  • Question on Document

    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Draft guidance consultation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

Maralixibat is not recommended, within its marketing authorisation, for treating cholestatic pruritus in Alagille syndrome in people 2 months and over.

1.2

This recommendation is not intended to affect treatment with maralixibat 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 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 these recommendations

Alagille syndrome is a rare genetic condition that often causes cholestatic pruritus (itching), which usually starts in early childhood. Most people with the syndrome have cholestasis. This causes a build-up of bile acids in the liver and skin, which can lead to cirrhosis of the liver and pruritus. There are no treatments that are licensed for cholestatic pruritus in Alagille syndrome available in the NHS. Standard care includes treatments for pruritus that are not licensed for use in this syndrome. Some people have a liver transplant because of constant severe pruritus. The company has positioned maralixibat as an add-on treatment to standard care.

Clinical trial evidence suggests that the pruritus of Alagille syndrome is more likely to respond to maralixibat plus standard care than to placebo plus standard care alone. But it is not clear how effective maralixibat is at reducing the pruritus in the longer term, and whether it would help people live longer compared with standard care alone. There is also no evidence on maralixibat use in adults.

There are also many uncertainties in the company's economic model, so the cost-effectiveness estimates are uncertain. Even when considering the condition's severity, and the effect of maralixibat on quality and length of life, the most likely cost-effectiveness estimates are above the range NICE normally considers an acceptable use of NHS resources. So, maralixibat is not recommended.