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Draft guidance consultation

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1 Recommendations

1.1

Exagamglogene autotemcel (exa‑cel) is not recommended, within its marketing authorisation, for treating sickle cell disease (SCD) in people 12 years and over with recurrent vaso-occlusive crises (VOCs) who have a βS/βS, βS/β+ or βS/β0 genotype, when a haematopoietic stem cell transplant (HSCT) is suitable and a human leukocyte antigen-matched related haematopoietic stem cell donor is not available.

Why the committee made these recommendations

Standard care for SCD includes hydroxycarbamide, blood transfusions and iron chelation therapy to remove excess iron in the blood. People who are well enough can have an HSCT if available. When an HSCT is suitable but there is no available human leukocyte antigen-matched donor, exa-cel is a possible cure.

In SCD, damaged red blood cells can block blood flow to parts of the body, depriving them of oxygen and causing severe pain. This is often called a VOC. Evidence from a clinical trial suggests that exa-cel can result in people not having VOCs. But this is uncertain because the treatment was not compared with anything else, the number of people in the trial was small and it was not clear how well it will work in the long-term.

NICE requires more information to address the uncertainties in the clinical and economic evidence. There were several issues with the economic modelling, including:

  • the model structure

  • the survival estimates

  • quality-of-life estimates

  • how long the treatment effect with exa‑cel lasts

  • how often people withdraw from exa-cel treatment before having the infusion

  • the frequency of complications.

The acceptable cost-effectiveness estimate for exa-cel is higher than what NICE normally considers to be a cost-effective use of NHS resources. This is a reasonable adjustment to account for health inequalities and the innovative nature of the technology. Even when taking this into account, the cost-effectiveness estimate for exa-cel is still above this. So, it is not recommended for routine use.

Uncertainty in the cost-effectiveness evidence could be addressed through managed access, but the company has not proposed to collect data to fully address this. Also, the cost-effectiveness evidence suggests that exa‑cel is not likely to be cost-effective. So, exa‑cel is not recommended for use with managed access.