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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Draft guidance consultation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 Voxelotor is not recommended, within its marketing authorisation, for treating haemolytic anaemia caused by sickle cell disease, with or without hydroxycarbamide, in people 12 years and older.

1.2 This recommendation is not intended to affect treatment with voxelotor 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. For children or young people, this decision should be made jointly by the clinician, the child or young person, and their parents or carers.

Why the committee made these recommendations

Usual treatments for haemolytic anaemia caused by sickle cell disease include hydroxycarbamide (also known as hydroxyurea) or regular blood transfusions. Voxelotor can be taken on its own or with hydroxycarbamide. There is an unmet need for effective treatments for people with sickle cell disease. The condition is more common in people from African, Caribbean, Middle Eastern or South Asian family backgrounds, who tend to have poorer health outcomes than people from other family backgrounds.

The clinical evidence suggests that a higher proportion of people taking voxelotor have increased haemoglobin levels compared with people having usual treatment. However, although there is likely to be some benefit of increasing haemoglobin levels, the impact on long term outcomes is not certain, and the key trial was short.

All of the cost-effectiveness estimates are above the range that NICE considers an acceptable use of NHS resources. And the estimates are uncertain because some of the data included in the economic model was not supported by evidence. So, even taking into consideration the unmet need and health inequalities, voxelotor is not recommended.