Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent ß-thalassemia.
J Mark Access Health Policy
; 9(1): 1922028, 2021 Jun 07.
Article
em En
| MEDLINE
| ID: mdl-34178295
ABSTRACT
Background:
Standard of care (SoC) for transfusion-dependent ß-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 ß-thalassemia) one-time, gene addition therapy compared to lifelong SoC for TDT. Studydesign:
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 perspectiveParticipants:
TDT patients age 2-50Interventions:
Beti-cel is compared to SoC. Main outcomemeasure:
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Tipo de estudo:
Health_economic_evaluation
/
Prognostic_studies
Aspecto:
Patient_preference
Idioma:
En
Revista:
J Mark Access Health Policy
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Estados Unidos