Key Points
- Non-myeloablative haploidentical (NMAC-HID) transplant would be cost-effective for long-term SCD remission in adults
- Maximum gene therapy price versus standard care across Tanzania, Nigeria, and India range is $4,200-$22,000, increasing versus NMAC-HID
Non-myeloablative-related haploidentical allogeneic stem cell transplantation (NMAC-HID allo-HSCT) emerged as an additional treatment for achieving durable remission in SCD, a prevalent blood disorder characterized by painful vaso-occlusive crises and chronic anemia. The standard-of-care (SOC) for SCD includes hydroxyurea, pain management, and blood transfusion, but patients with SCD still lose several decades of life expectancy. Gene therapy (GT) for SCD is the other treatment for lifelong disease amelioration in SCD, with accessibility limited by cost and manufacturing capacity in the US and globally. Two recent prospective studies evaluating NMAC-HID allo-HSCT validated haploidentical allotransplantation as an efficacious and accessible treatment option in the era of gene therapy. Given ongoing price negotiation across jurisdictions for GT implementation and the absence of cost-effectiveness data comparing NMAC-HID allo-HSCT versus GT, we conducted a cost-effectiveness analysis of NMAC-HID allo-HST versus GT versus SOC for adults and children living with SCD. The primary outcomes were the incremental cost-effectiveness ratio and the net monetary benefits (NMBs) across these three strategies. The secondary outcome was the maximum cost-effective threshold price for GT compared to NMAC-HID allo-HSCT. Treatment with SOC, NMAC-HID allo-HSCT, and GT accrued 14.3, 20.1, and 22.1 QALYs at costs of $1.22 million, $1.15 million and $2.75 million respectively. NMAC-HID allo-HSCT was the cost-effective strategy versus GT in 100% of 10,000 Monte Carlo iterations across the base-case and all scenario analyses. The maximum cost-effective thresholds for GT vs SOC were $1.4 million in the United States and $4,200-$22,000 across India, Nigeria, and Tanzania, depending on willingness-to-pay.