Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia

Abstract

<b>Background</b>: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation. <b>Objective</b>: 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. <b>Study design</b>: 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. <b>Setting</b>: USA; commercial payer perspective <b>Participants</b>: TDT patients age 2-50 <b>Interventions</b>: Beti-cel is compared to SoC. <b>Main outcome measure</b>: Incremental cost-effectiveness ratio (ICER) utilizing quality-adjusted life-years (QALYs) <b>Results</b>: 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. <b>Conclusion</b>: Beti-cel is cost-effective for TDT patients compared to SoC. This is due to longer survival and cost offset of lifelong SoC.