1 Recommendations

1 Recommendations

1.1 Crizanlizumab is recommended as an option for preventing recurrent sickle cell crises (vaso-occlusive crises) in people agedĀ 16 or over with sickle cell disease only if the conditions in the managed access agreement are followed.

1.2 This recommendation is not intended to affect treatment with crizanlizumab 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

Treatments to prevent sickle cell crises include hydroxycarbamide (also known as hydroxyurea), which is taken as a tablet, or regular blood transfusions. Crizanlizumab is a treatment injected into the vein (intravenous, or IV) that people agedĀ 16 or over can take on its own or alongside hydroxycarbamide.

The clinical evidence suggests that people taking crizanlizumab have fewer sickle cell crises in a year than if they have best supportive care with or without hydroxycarbamide. However, because the trial was short and included only a small number of people on the licensed dose of the drug, the long-term benefits are uncertain.

There is also uncertainty about the cost-effectiveness estimates because some of the inputs used in the model do not reflect the clinical evidence. The most likely cost-effectiveness estimate is above what NICE normally considers a cost-effective use of NHS resources. Therefore, the committee could not recommend crizanlizumab for routine use in the NHS.

However, there is an unmet need for effective treatments for people with sickle cell disease. They also face health inequalities because the condition is not well understood, results in disability, and is more common in people of African or African-Caribbean family origin, who tend to have poorer health outcomes than other ethnicities. Access to crizanlizumab may help address these inequalities. Because of this, crizanlizumab is recommended for people with sickle cell disease and recurrent vaso-occlusive crises if more data is collected using a managed access agreement, to address the uncertainties in the evidence. This recommendation will be reviewed based on the data collected.

  • National Institute for Health and Care Excellence (NICE)