Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia. Issue 1 (1st January 2021)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: Journal of market access & health policy. Volume 9:Issue 1(2021)
• Publication Date: 2021-01-01
• Subjects: Beta-thalassemia -- economic evaluation -- gene therapy; Medical policy -- Periodicals; Medical policy -- Economic aspects -- Periodicals; International trade -- Environmental aspects -- Periodicals; Health Policy -- economics; Technology Assessment, Biomedical; Marketing of Health Services; International trade -- Environmental aspects; Medical policy; Periodicals; Periodicals; Fulltext; Internet Resources; Periodicals; 362.1
• DOI: 10.1080/20016689.2021.1922028
• Languages: English
• ISSNs: 2001-6689

Abstract

ABSTRACT: 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.