1 Recommendations

1.1

Voxelotor, with or without hydroxycarbamide, is recommended as an option for treating haemolytic anaemia caused by sickle cell disease in people 12 years and over. It is recommended only if:

  • people are ineligible for, or intolerant of hydroxycarbamide, or

  • hydroxycarbamide alone is insufficiently effective.

    Voxelotor is only recommended if the company provides it according to the commercial arrangement.

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 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

There is an unmet need for effective treatments for sickle cell disease, and health inequalities affect people with the condition. Usual treatment options for haemolytic anaemia caused by sickle cell disease are hydroxycarbamide (also known as hydroxyurea) and regular blood transfusions. For this evaluation, the company positioned voxelotor 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 already had hydroxycarbamide.

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 some of the health inequalities associated with sickle cell disease and the unmet need for effective treatments. Greater uncertainty in the clinical-effectiveness estimates could be accepted as a reasonable adjustment for the substantial disadvantages identified for people with sickle cell disease. So, a higher cost-effectiveness estimate than usual could be considered acceptable. When taking these additional factors into account, the most plausible cost-effectiveness estimates are below what NICE considers an acceptable use of NHS resources. So, voxelotor is recommended.