OBJECTIVES : To evaluate the cost effectiveness of beti-cel versus long-term non-curative treatments with transfusions and iron chelation therapy (SoC) in patients, aged ≥12 years, with TDT and non-β⁰/β⁰ genotype and eligible for allogeneic HCST but without a HLA-matched donor, in France.

METHODS : A discretely integrated condition event model adapted to the French setting was used to estimate costs and benefits of beti-cel versus the current SoC over a lifetime horizon. Clinical input data were sourced from clinical trials of beti-cel. For SoC, complication and mortality rates related to iron overload were derived from published sources and costs were estimated based on French data. Utilities were estimated from a UK Chart review by applying French preferences methods and from literature. An innovative payment scheme for beti-cel was considered. The base case analysis was performed according to the HAS guidelines perspective (all payers without indirect costs). Future costs and clinical outcomes were discounted at 2.5%. Sensitivity and scenario analyses were performed.

RESULTS : From the collective perspective, compared to SoC, beti-cel gene therapy was associated with a gain of 4.59 (26.90 versus 22.31) life years (LYs) and 6.41 (20.80 versus 14.39) quality-adjusted life years (QALYs). Direct lifetime costs of SoC were €1,207,166, while those of beti-cel were €314,141 higher, resulting in an incremental cost-effectiveness ratio (ICER) of €48,998/QALY. When indirect costs due to loss of productivity, including unemployment, were included, the ICER was €427/QALY. Parameters with the greatest impact on ICER in sensitivity analyses were time horizon, discount rate, and perspective. Given a willingness to pay of €100,000/QALY, beti-cel has an 84% cost-effectiveness probability compared to the current SoC.

CONCLUSIONS : Even in the context of no official threshold in France, this modeling study suggests that beti-cel can represent a cost-effective treatment option for TDT’ indication patients.