How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • 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?

  • Question on Document

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

  • Question on Document

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of age, disability, gender reassignment, pregnancy and maternity, race, religion or belief, sex or sexual orientation?
The content on this page is not current guidance and is only for the purposes of the consultation process.

Draft guidance consultation

Closed for comments This consultation ended on at   Request commenting lead permission

1 Recommendations

1.1

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

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

There is an unmet need for effective treatments for sickle cell disease, and health inequalities affect people with the condition. Usual treatment for haemolytic anaemia caused by sickle cell disease includes hydroxycarbamide (also known as hydroxyurea) or regular blood transfusions. For this evaluation, the company positioned volexotor as a second-line treatment. This does not include everyone who it is licensed for.

Clinical evidence suggests that people who have voxelotor are more likely to have an increase in haemoglobin levels compared with people who have usual treatment. Although this is likely to be beneficial, how well voxelotor works is uncertain because:

  • the key trial was short, so it is uncertain what the benefits are in the long term

  • the people in the trial did not reflect the people who would have second-line treatment with voxelotor in the NHS, because they were not able to have regular blood transfusions and did not have to have had hydroxycarbamide previously.

The cost-effectiveness estimates for voxelotor are also uncertain. This is because some assumptions used to estimate the cost effectiveness were not supported by clinical evidence. Voxelotor has the potential to partially address the health inequalities associated with sickle cell disease and the unmet need for effective treatments, so a cost-effectiveness estimate that is higher than the usual maximum for a positive decision could be considered acceptable. But, the cost-effectiveness estimates for the company's proposed second line positioning varied widely owing to high uncertainty regarding key assumptions in the economic model. It was agreed that greater uncertainty around clinical effectiveness estimates could be accepted as a reasonable adjustment for the substantial disadvantage identified for people with sickle cell disease. However, even taking this into account, the most plausible cost-effectiveness estimates are beyond what NICE considers an acceptable use of NHS resources.

It is not possible to consider use of voxelotor with managed access because the company has not provided a managed access proposal. So, voxelotor is not recommended.

The committee has requested further clarification and analyses for the fourth evaluation committee meeting to ensure that potential avenues for patient access are fully explored.