Exagamglogene autotemcel for treating transfusion-dependent beta-thalassaemia in people 12 years and over

When NICE recommends a treatment as an option for use with managed access, NHS England will make it available according to the conditions in the managed access agreement. This means that, if a patient has transfusion-dependent beta-thalassaemia and a haematopoietic stem cell transplant is suitable but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available, and the healthcare professional responsible for their care thinks that exagamglogene autotemcel is the right treatment, it should be available for use, in line with NICE's recommendations and the criteria in the managed access agreement. Further information can be found in the Innovative Medicines Fund principles.

Funding for this treatment will be available through the Innovative Medicines Fund when positive final draft guidance is released.

The Welsh ministers have issued directions to the NHS in Wales on implementing NICE technology appraisal guidance when the drug or treatment, or other technology, is approved for use with managed access. When a NICE technology appraisal guidance recommends the use of a drug or treatment, or other technology, for use with managed access, the NHS in Wales must usually provide funding and resources for it within 2 months of the first publication of the final draft guidance or agreement of a managed access agreement by the NHS in Wales, whichever is the later.