Economic Evaluations of Sickle Cell Disease-Modifying Therapies in Adult Patients

Abstract

Throughout history, sickle cell disease (SCD) has faced unequal healthcare treatment, impacting African Americans disproportionately due to difficulties in obtaining high-quality medical services. Although advancements in medicine have resulted in longer lifespans, these improvements may not fully meet the complex requirements of the growing number of elderly individuals dealing with the illness. Objectives: The objectives of this study are to (1) to investigate how the type of insurance influences the use of crizanlizumab and voxelotor in adult patients with SCD; (2) to evaluate the financial burden and therapeutic benefits of SCD therapies in mature individuals; and (3) to determine the financial impact and feasibility of using SCD treatments in U.S. Medicaid programs that have the highest rates of SCD. Methods: A comprehensive review of patient records was undertaken at the adult SCD clinic at Howard University Hospital, spanning from 2019 to 2023. The review focused on patients prescribed crizanlizumab and voxelotor, and extracted demographic data and insurance information. Logistic regression used to predict crizanlizumab or voxelotor usage A cost-utility analysis compared the cost-effectiveness of SCD-modifying therapies to automated red blood cell exchange in adult patients, using a lifetime horizon and taking into account societal costs. This model was then used to estimate the total budgetary impact of these therapies versus automated red blood cell exchange over a one-year period, from the perspective of U.S. Medicaid programs with the highest incidence of SCD, and exploring the potential benefits of an annuity payment model. Results: A total of 54 patients with were included, SCD, with an average age of 39.4 years, and the vast majority were single (90.7%). Notably, patients with dual eligibility had a significantly higher average age (43 years) compared to those with only Medicaid coverage(40 years). After controlling for other factors, the results showed that patients with Medicare coverage were nearly 10 times more likely to receive crizanlizumab compared to those with Medicaid coverage. The cost utility analysis indicated that automated red blood cell exchange (aRBCX) may be a cost-effective strategy compared to other SCD-modifying therapies, with a cost per quality-adjusted life-year (QALY) below $100,000, depending on the severity of SCD and the likelihood of death due to vaso-occlusive crisis. The cost-utility model predicted that hydroxyurea had the lowest lifetime costs, approximately $1,039,311, while voxelotor had the highest, approximately $3,252,674. Also, aRBCX resulted in the highest QALY and the fewest acute pain episodes over a lifetime. In terms of cost-effectiveness, l-glutamine had the lowest incremental cost-effectiveness ratio compared to aRBCX. Furthermore, the analysis demonstrated that voxelotor had the largest annualized budget impact, adding approximately $95,702.40 per patient compared to aRBCX, while hydroxyurea offered significant short-term cost savings of approximately $41,519.23 per patient relative to aRBCX. Conclusions: Medicaid serves as a vital safety net for individuals afflicted with SCD, with roughly half of adult sufferers relying on Medicaid or similar programs for their health insurance needs. Despite this, the adoption of interventions that can alter the course of SCD remains surprisingly limited. In stark contrast, aRBCX has consistently shown more favorable results in adult patients compared to these disease-modifying therapies.