Annals of Internal MedicineGene Therapy Versus Common Care for Eligible Individuals with Sickle Cell Disease in the United States

Gene Therapy for Sickle Cell Disease: A Cost-Effectiveness Perspective

This study provides a comprehensive analysis of the cost-effectiveness and value-based pricing for gene therapy as a treatment for sickle cell disease (SCD), a condition that significantly impacts morbidity, mortality, and healthcare costs, particularly in the US and African heritage communities. Through comparative modeling, utilizing data from the Centers for Medicare & Medicaid Services and published literature, the research evaluates the intervention’s long-term financial and health outcomes compared to common care practices.

Study Design:

• Two 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]) were used.
• Analysis based on Centers for Medicare & Medicaid Services claims data from 2008 to 2016 and published literature.
• Target population included persons eligible for gene therapy, considering a lifetime horizon from both US healthcare sector and societal perspectives.

Key Findings:

• Estimated incremental cost-effectiveness ratios (ICERs) for gene therapy were $193,000 per QALY (UW-MEASURE) and $427,000 per QALY (FH-HISCORE) from a healthcare sector perspective.
• From a societal perspective, ICERs were lower, at $126,000 per QALY (UW-MEASURE) and $281,000 per QALY (FH-HISCORE).
• Acceptable value-based prices (VBPs) for gene therapy ranged from $1 million to $2.5 million, influenced by quality-of-life considerations and equity-informed threshold values.
• Sensitivity analyses highlighted the impact of myeloablative conditioning costs, caregiver quality of life, and long-term survival benefits on cost-effectiveness outcomes.

HCN Medical Memo
The analysis indicates that gene therapy for sickle cell disease (SCD), priced under $2 million, presents a financially viable treatment option from a societal perspective. This affordability is further emphasized when considering an equity-informed threshold in cost-effectiveness analysis, highlighting the therapy’s potential to offer significant health benefits equitably across populations. This approach not only underscores the importance of integrating societal values and equity considerations into healthcare decision-making but also supports the broader adoption of gene therapy as a cost-effective solution for managing SCD.

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