Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.
Ann Intern Med
; 177(2): 155-164, 2024 02.
Article
en En
| MEDLINE
| ID: mdl-38252942
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. OUTCOMEMEASURES:
Incremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves. RESULTS OF BASE-CASEANALYSIS:
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 SENSITIVITYANALYSIS:
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.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Análisis de Costo-Efectividad
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Anemia de Células Falciformes
Tipo de estudio:
Health_economic_evaluation
Límite:
Aged
/
Humans
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Male
País como asunto:
America do norte
Idioma:
En
Año:
2024
Tipo del documento:
Article