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Betibeglogene autotemcel for treating transfusion-dependent beta-thalassaemia

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1 Recommendations

1.1 Betibeglogene autotemcel is not recommended, within its marketing authorisation, for treating transfusion-dependent beta-thalassaemia (TDT) in people 12 years and older who do not have a beta0/beta0 genotype, when haematopoietic stem cell transplantation (HSCT) is appropriate but a human leukocyte antigen-matched related HSC donor is not available.

1.2 This recommendation is not intended to affect treatment with betibeglogene autotemcel 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. When the person having treatment is under 18 years, this decision should be made jointly by the clinician and the child or young person, and their parents or carers.

Why the committee made these recommendations

Current treatment for TDT includes regular blood transfusions, and iron chelation therapy for excess iron build up in the body from the transfusions. HSCT is a curative therapy that is available for a few people with the condition. But they need a matched donor and to be within a specific age range. Betibeglogene autotemcel is approved for people who do not have a beta0/beta0 genotype, and when HSCT would be appropriate but there is no suitable donor.

Clinical trials for betibeglogene autotemcel are small and people have not been followed up for very long. However, the trials suggest that, after betibeglogene autotemcel, some people either eventually do not need blood transfusions any more or need them less often.

There are uncertainties about the cost effectiveness and the preferred estimate for betibeglogene autotemcel is considerably higher than what NICE normally considers an acceptable use of NHS resources. So, betibeglogene autotemcel is not recommended.