New gene therapy for severe beta-thalassaemia

Posted on 19 September 2024

Research teams from the Centre for Reviews and Dissemination (CRD) and the Centre for Health Economics (CHE) assessed the clinical and cost-effectiveness of exagamglogene autotemcel for treating NHS patients with severe beta-thalassaemia.

In August 2024 the BBC reported on a new gene therapy for severe beta-thalassaemia. For qualifying patients, this one-off treatment replaces regular and life-long blood transfusions, which are burdensome to receive and come with serious side effects. The National Institute of Health and Care Excellence (NICE) decision to approve exagamglogene autotemcel for use on the NHS was informed by the report of the CRD & CHE Evidence Assessment Group. This group consisted of Rob Hodgson, Mark Corbett, Noortje Uphoff, Jasmine Deng, Joseph Lord, Helen Fulbright, and colleagues from CHE. The work was carried out under our Technology Assessment Review (TAR) programme of work, funded by the National Institute of Health and Care Research (NIHR).