Objective:

To compare the cost-effectiveness of non-myeloablative haploidentical allogeneic stem cell transplantation (NMAC-HID allo-HSCT), gene therapy, and standard care for adults with sickle cell disease.

Approach:

Key Findings:

• Gene therapy provided the highest projected health benefit at 22.1 quality-adjusted life years (QALYs) but at a cost of $2.75 million.
• NMAC-HID allo-HSCT resulted in 20.1 QALYs at a cost of $1.15 million.
• Standard care yielded 14.3 QALYs at a cost of $1.22 million.
• The US threshold price for gene therapy was estimated to be $627,000 to $740,000, a significant reduction from current pricing.
• In lower-income countries, gene therapy thresholds ranged from $24,000 to $35,000.

Interpretation:

NMAC-HID allo-HSCT was found to deliver the best clinical value for cost compared to gene therapy and standard care.

Limitations:

• Long-term safety and efficacy data for gene therapy and NMAC-HID allo-HSCT are limited.
• The model lacked patient-level data across trials.
• Assumed patients had a viable donor and no prohibitive donor-specific antibodies.
• Did not include costs for fertility preservation.
• Relied on public gene therapy list prices instead of negotiated prices.

Conclusion:

Cost-effectiveness data should inform policy decisions without limiting patient choice, and all treatment options should be discussed with patients.

Sources:

• Yale School of Medicine Press Release
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