PBI16 Betibeglogene Autotemcel GENE Therapy (BETI-CEL) Is Cost-Effective Versus Standard of Care in Patients with Transfusion-Dependent B-Thalassemia (TDT) in France

Improving outcomes and quality of life for patients with transfusion-dependent β-thalassemia: recommendations for best clinical practice and the use of novel treatment strategies

“…The impact of gene therapy on patient QoL remains to be fully determined and should be further evaluated in future clinical trials of beti-cel. However, data from the phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) studies showed that patients under 18 years of age who achieved transfusion independence had an improved EuroQoL 5 dimensions (Youth) (EQ-5D-Y) score (67 [range 50-96] versus 92.5 [85][86][87][88][89][90][91][92][93][94][95]) after 12 months [67,90]. In addition, HSCT has been shown to have a positive impact on both physical and emotional aspects of HRQoL in patients with TD β-thalassemia and, given GVHD was the most common cause of impaired HRQoL in these patients, a similar or greater benefit would be expected from gene therapy [67].…”

“…As has been previously described for HSCT, gene therapy could theoretically be cost-effective if it allows patients to achieve transfusion independence and avoid the high monthly costs associated with long-term transfusions, ICT, and related healthcare expenses; however, the cost of gene therapy is considerably higher than that of HSCT. A recent health economic analysis of beti-cel therapy versus standard of care for TD β-thalassemia in France showed that beti-cel was costeffective with an incremental cost-effectiveness ratio of EUR 49 per quality-adjusted life-year (QALY), rising to EUR 427/QALY when indirect costs such as unemployment and loss of productivity were included [92].…”