Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis
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Visual Abstract. Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States
The U.S. Food and Drug Administration recently approved 2 genetic therapies for sickle cell disease. Both therapies increase the production of nonsickling red blood cells, either fetal hemoglobin or a gene therapy–derived hemoglobin similar to hemoglobin A. In addition, both therapies require collection of a patient’s stem cells, modification of the cells in the laboratory, high-dose chemotherapy to remove existing bone marrow cells, and a single-dose infusion of the patient’s modified stem cells. This article uses 2 different cost-effectiveness models to estimate whether these therapies provide enough value to justify their high costs.
Abstract
Background:
Sickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community.
Objective:
To evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs).
Design:
Comparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases.
Data Sources:
Centers for Medicare & Medicaid Services claims data, 2008 to 2016; published literature.
Target Population:
Persons eligible for gene therapy.
Time Horizon:
Lifetime.
Perspective:
U.S. health care sector and societal.
Intervention:
Gene therapy versus common care.
Outcome Measures:
Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves.
Results of Base-Case Analysis:
At an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values.
Results of Sensitivity Analysis:
Results were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival.
Limitation:
The short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study.
Conclusion:
Gene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis.
Primary Funding Source:
National Heart, Lung, and Blood Institute.
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Published In
Annals of Internal Medicine
Volume 177 • Number 2 • February 2024
Pages: 155 - 164
History
Published online: 23 January 2024
Published in issue: February 2024
Keywords
Copyright
© 2024 American College of Physicians.
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Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis. Ann Intern Med.2024;177:155-164. [Epub 23 January 2024]. doi:10.7326/M23-1520
Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States: A Cost-Effectiveness Analysis. Ann Intern Med.2024;177:155-164. [Epub 23 January 2024]. doi:10.7326/M23-1520
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