Economic Evaluation of Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy in Transfusion-Dependent β-Thalassemia

Background: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation.

Objective: This study investigates the cost-effectiveness of betibeglogene autotemcel (‘beti-cel’; LentiGlobin for β-thalassemia) one-time, gene addition therapy compared to lifelong SoC for TDT.

Study design: Microsimulation model simulated the lifetime course of TDT based on a causal sequence in which transfusion requirements determine tissue iron levels, which in turn determine risk of iron overload complications that increase mortality. Clinical trial data informed beti-cel clinical parameters; effects of SoC on iron levels came from real-world studies; iron overload complication rates and mortality were based on published literature.

Setting: USA; commercial payer perspective.

Participants: TDT patients age 2–50.

Interventions: Beti-cel is compared to SoC. Main outcome measure: Incremental cost-effectiveness ratio (ICER) utilizing quality-adjusted lifeyears (QALYs).

Results: The model predicts beti-cel adds 3.8 discounted life years (LYs) or 6.9 QALYs versus SoC. Discounted lifetime costs were $2.28 M for beti-cel ($572,107 if excluding beti-cel cost) and $2.04 M for SoC, with a resulting ICER of $34,833 per QALY gained.

Conclusion: Beti-cel is cost-effective for TDT patients compared to SoC. This is due to longer
survival and cost offset of lifelong SoC.