• Anirban Basu, Aaron N Winn, Kate M Johnson, Boshen Jiao, Beth Devine, Jane S Hankins, Staci D Arnold, M A Bender, and Scott D Ramsey.
• The Comparative Health Outcomes, Policy & Economics (CHOICE) Institute, Department of Pharmacy; Department of Health Systems and Population Health; and Department of Economics, University of Washington, Seattle, Washington (A.B.).
• Ann. Intern. Med. 2024 Feb 1; 177 (2): 155164155-164.

BackgroundSickle 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.ObjectiveTo evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs).DesignComparative 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 SourcesCenters for Medicare & Medicaid Services claims data, 2008 to 2016; published literature.Target PopulationPersons eligible for gene therapy.Time HorizonLifetime.PerspectiveU.S. health care sector and societal.InterventionGene therapy versus common care.Outcome MeasuresIncremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves.Results Of Base Case AnalysisAt 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 AnalysisResults were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival.LimitationThe short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study.ConclusionGene 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 SourceNational Heart, Lung, and Blood Institute.

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