On February 12, in an appraisal consultation document, the UK National Institute for Health and Care Excellence (NICE) recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) gene therapy for the treatment of patients with transfusion-dependent β-thalassaemia (TDT).
Such a negative opinion would result in the treatment not being supported by the National Health Service (NHS) leaving patients with thalassaemia in the UK unable to benefit. In addition, health authorities in other countries may possibly be influenced by this decision, thus denying the choice of gene therapy to patients beyond the immediate sphere of influence of NICE.
However, the appraisal committee states that it is interested in receiving comments, indicating that this is not their final decision. The Thalassaemia International Federation (TIF), after coordination with its two UK members, the UK Thalassaemia Society (UKTS) and the North of England Bone Marrow and Thalassaemia Association (NEBATA), has prepared its own response to NICE in support of its UK patients benefit and rights.
Read the TIF Full Submission/Opinion on NICE’s Consultation Document for Zynteglo® HERE