• George Goshua, Cecelia Calhoun, Satoko Ito, Lyndon P James, Andrea Luviano, Lakshmanan Krishnamurti, and Ankur Pandya.
• Section of Hematology, Yale University School of Medicine, New Haven, Connecticut (G.G., C.C., S.I.).
• Ann. Intern. Med. 2023 Jun 1; 176 (6): 779787779-787.

BackgroundGene therapy is a potential cure for sickle cell disease (SCD). Conventional cost-effectiveness analysis (CEA) does not capture the effects of treatments on disparities in SCD, but distributional CEA (DCEA) uses equity weights to incorporate these considerations.ObjectiveTo compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.DesignMarkov model.Data SourcesClaims data and other published sources.Target PopulationBirth cohort of patients with SCD.Time HorizonLifetime.PerspectiveU.S. health system.InterventionGene therapy at age 12 years versus SOC.Outcome MeasuresIncremental cost-effectiveness ratio (ICER) (in dollars per quality-adjusted life-years [QALYs] gained) and threshold inequality aversion parameter (equity weight).Results Of Base Case AnalysisGene therapy versus SOC for females yielded 25.5 versus 15.7 (males: 24.4 vs. 15.5) discounted lifetime QALYs at costs of $2.8 million and $1.0 million (males: $2.8 million and $1.2 million), respectively, with an ICER of $176 000 per QALY (full SCD population). The inequality aversion parameter would need to be 0.90 for the full SCD population for gene therapy to be preferred per DCEA standards.Results Of Sensitivity AnalysisSOC was favored in 100.0% (females) and 87.1% (males) of 10 000 probabilistic iterations at a willingness-to-pay threshold of $100 000 per QALY. Gene therapy would need to cost less than $1.79 million to meet conventional CEA standards.LimitationBenchmark equity weights (as opposed to SCD-specific weights) were used to interpret DCEA results.ConclusionGene therapy is cost-ineffective per conventional CEA standards but can be an equitable therapeutic strategy for persons living with SCD in the United States per DCEA standards.Primary Funding SourceYale Bernard G. Forget Scholars Program and Bunker Endowment.

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