Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what discussions she has had with NHS England on the introduction of (a) gene therapy and (b) other new treatments for patients with thalassaemia.

The Department regularly discusses a range of issues with colleagues in NHS England related to patient access to new medicines. The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether all new medicines should be routinely funded by the NHS, based on an assessment of their costs and benefits. The NICE’s appraisal of the gene therapy, exagamglogene autotemcel, for treating transfusion-dependent beta-thalassaemia is currently paused, to allow the company and NHS England to enter into commercial and managed access discussions.

In November 2023, NHS England published a clinical commissioning policy that recommends that allogeneic haematopoietic stem cell transplantation for adult transfusion dependent thalassaemia should be routinely commissioned.