Kansal Anuraag R, Reifsnider Odette S, Brand Sarah B, Hawkins Neil, Coughlan Anna, Li Shujun, Cragin Lael, Paramore Clark, Dietz Andrew C, Caro J Jaime
Evidera, Inc., Waltham, MA, USA.
Visible Analytics, Oxford, UK.
J Mark Access Health Policy. 2021 Jun 7;9(1):1922028. doi: 10.1080/20016689.2021.1922028.
: Standard of care (SoC) for transfusion-dependent β-thalassemia (TDT) requires lifelong, regular blood transfusions as well as chelation to reduce iron accumulation. : 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. : 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. : USA; commercial payer perspective : TDT patients age 2-50 : Beti-cel is compared to SoC. : Incremental cost-effectiveness ratio (ICER) utilizing quality-adjusted life-years (QALYs) : 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. : Beti-cel is cost-effective for TDT patients compared to SoC. This is due to longer survival and cost offset of lifelong SoC.
输血依赖型β地中海贫血（TDT）的标准治疗（SoC）要求进行终身定期输血以及螯合治疗以减少铁蓄积。本研究调查了与TDT的终身SoC相比，一次性基因添加疗法betibeglogene autotemcel（“beti-cel”；用于β地中海贫血的LentiGlobin）的成本效益。微观模拟模型基于一种因果序列模拟了TDT的终身病程，在该序列中输血需求决定组织铁水平，而组织铁水平又决定铁过载并发症的风险，铁过载并发症会增加死亡率。临床试验数据为beti-cel的临床参数提供了依据；SoC对铁水平的影响来自真实世界研究；铁过载并发症发生率和死亡率基于已发表的文献。美国；商业支付方视角TDT患者年龄在2至50岁之间将beti-cel与SoC进行比较。使用质量调整生命年（QALYs）的增量成本效益比（ICER）该模型预测，与SoC相比，beti-cel可增加3.8个贴现生命年（LYs）或6.9个QALYs。beti-cel的贴现终身成本为228万美元（如果排除beti-cel成本则为572,107美元），SoC为204万美元，由此得出的ICER为每获得一个QALY 34,833美元。与SoC相比，beti-cel对TDT患者具有成本效益。这是由于生存期延长以及终身SoC的成本抵消。
