1. In this cost analysis, compared to standard care, it was found that gene therapy for sickle cell disease (SCD) is cost-effective at a societal level based on two simulation models.
2. Acceptable value-based prices (VPBs) for SCD gene therapy ranged from $1 million to $2.5 million.
Evidence Rating Level: 2 (Good)
Study Rundown: SCD is a genetic condition characterized by sickling red cells leading to recurrent vaso-occlusion and hemolysis and precipitating acute pain crises, ischemia, and progressive organ damage. Approximately 100,000 Americans are living with SCD; most are of African descent. Emerging gene therapies can potentially offer broadly available long-term cures, but their expected high prices result in concerns about their cost-effectiveness. The current study was a comparative analysis using two simulation models, the University of Washington Model for Economic Analysis of Sick Cell Cure (UW-MEASURE) and the Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model (FH-HISCORE), to assess the cost-effectiveness of gene therapy versus common care regimen of hydroxyurea and transfusions. Through these models, gene therapy was found to be cost-effective at a price of $2 million from healthcare, societal, and quality-of-life perspectives. The acceptable VPBs for gene therapy ranged from 1-2.5 million dollars. Although the study was based on limited evidence of the efficacy and durability of SCD gene therapy, it offered an initial evaluation of its potential cost-effectiveness and estimates of the acceptable costs to guide policy decisions.
In-Depth [cost analysis]: The current study utilized the UW-MEASURE and FH-HISCORE to simulate the costs of implementing gene therapy for SCD. These models were then applied to a data set comprised of all individuals with SCD enrolled under Medicaid and Medicare from 2008 to 2016 who fit the inclusion criteria for the published clinical trial of SCD gene therapy of LentiGlobin. Outcome estimates for SCD progression under a common care regimen of hydroxyurea and transfusion regarding acute, subacute, and chronic outcomes were developed using this claims database. The main outcomes of the study were the incremental cost-effectiveness ratios (ICERs), calculated as cost per quality-adjusted life-year (QALY), and VBPs. Both models projected fewer pain crises with gene therapy over the lifetime compared to common care as well as improved life expectancy (17.4 years with UW-MEASURE and 17.0 years with FH-HISCORE) compared to common care. Presuming a price of $2 million, the total incremental cost of gene therapy to the healthcare sector was estimated at $2,298,780 from the UW-MEASURE model and $2,178,228 from FH-HISCORE. The incremental lifetime societal cost was estimated at $1,498,971 and $1,568,094, respectively. Accordingly, the ICERs of gene therapy compared against common care for the healthcare sector were $193,000 per QALY from UW-MEASURE and $427,000 per QALY from FH-HISCORE, while the ICERs from the societal perspective were $126,000 per QALY and $281,000 per QALY, respectively. Using the cost-effectiveness threshold of $100,000 per QALY, the acceptable societal VBPs predicted by the two models ranged from $1 million to $2.5 million, depending on whether the threshold was equity-informed. Sensitivity analysis found consistent conclusions when accounting for the costs of myeloablative conditioning before gene therapy, effect on caregivers, and effect on long-term survival. These results provided an initial assessment of the therapy’s cost-effectiveness in guiding industry and policy decisions in the future.
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