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-thalasse...

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Autores principales:	Anuraag R. Kansal, Odette S. Reifsnider, Sarah B. Brand, Neil Hawkins, Anna Coughlan, Shujun Li, Lael Cragin, Clark Paramore, Andrew C. Dietz, J. Jaime Caro
Formato:	article
Lenguaje:	EN
Publicado:	Taylor & Francis Group 2021
Materias:	beta-thalassemia economic evaluation gene therapy Public aspects of medicine RA1-1270 Business HF5001-6182
Acceso en línea:	https://doaj.org/article/d6cb861054544eae9f3fd0425864a1ec
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spelling	oai:doaj.org-article:d6cb861054544eae9f3fd0425864a1ec2021-11-26T11:19:49ZEconomic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia2001-668910.1080/20016689.2021.1922028https://doaj.org/article/d6cb861054544eae9f3fd0425864a1ec2021-01-01T00:00:00Zhttp://dx.doi.org/10.1080/20016689.2021.1922028https://doaj.org/toc/2001-6689Background 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.Anuraag R. KansalOdette S. ReifsniderSarah B. BrandNeil HawkinsAnna CoughlanShujun LiLael CraginClark ParamoreAndrew C. DietzJ. Jaime CaroTaylor & Francis Grouparticlebeta-thalassemiaeconomic evaluationgene therapyPublic aspects of medicineRA1-1270BusinessHF5001-6182ENJournal of Market Access & Health Policy, Vol 9, Iss 1 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	beta-thalassemia economic evaluation gene therapy Public aspects of medicine RA1-1270 Business HF5001-6182
spellingShingle	beta-thalassemia economic evaluation gene therapy Public aspects of medicine RA1-1270 Business HF5001-6182 Anuraag R. Kansal Odette S. Reifsnider Sarah B. Brand Neil Hawkins Anna Coughlan Shujun Li Lael Cragin Clark Paramore Andrew C. Dietz J. Jaime Caro Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
description	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.
format	article
author	Anuraag R. Kansal Odette S. Reifsnider Sarah B. Brand Neil Hawkins Anna Coughlan Shujun Li Lael Cragin Clark Paramore Andrew C. Dietz J. Jaime Caro
author_facet	Anuraag R. Kansal Odette S. Reifsnider Sarah B. Brand Neil Hawkins Anna Coughlan Shujun Li Lael Cragin Clark Paramore Andrew C. Dietz J. Jaime Caro
author_sort	Anuraag R. Kansal
title	Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
title_short	Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
title_full	Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
title_fullStr	Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
title_full_unstemmed	Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
title_sort	economic evaluation of betibeglogene autotemcel (beti-cel) gene addition therapy in transfusion-dependent β-thalassemia
publisher	Taylor & Francis Group
publishDate	2021
url	https://doaj.org/article/d6cb861054544eae9f3fd0425864a1ec
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Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia

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