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

• Main Authors: Anuraag R. Kansal (Author), Odette S. Reifsnider (Author), Sarah B. Brand (Author), Neil Hawkins (Author), Anna Coughlan (Author), Shujun Li (Author), Lael Cragin (Author), Clark Paramore (Author), Andrew C. Dietz (Author), J. Jaime Caro (Author)
• Format: Book
• Published: Taylor & Francis Group, 2021-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary

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 life-years (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.

Item Description

2001-6689
10.1080/20016689.2021.1922028