Cost-Effective Analysis Identifies Allotransplantation as an Equitable Therapy for Pediatric Patients with SCD

Investigators conducted cost-effectiveness analyses of allotransplantation therapy for pediatric patients with sickle cell disease (SCD) in the United States and found that this treatment is an “equitable” option. The results were presented at the 65th ASH Annual Meeting & Exposition.

“Prior research has shown that even if gene therapy is conventionally cost-ineffective versus the standard of care (SOC), it can be a justifiable treatment option for a historically marginalized patient population in the United States, as demonstrated by equity-weighted cost-effectiveness,” the investigators wrote. “For allotransplantation and gene therapy, neither conventional nor distributional (ie, equity-weighted) cost-effectiveness is known.”

Therefore, the researchers constructed a Markov model to assess the cost-effectiveness of allotransplantation, gene therapy, and SOC for pediatric patients with SCD. Data from 11 years of US commercially insured patients informed the analysis. The SOC included various treatments, and costs for allotransplantation and graft-versus-host disease were based on prior literature. Quality-adjusted life-years (QALYs) were determined using lifetime simulations. Conventional cost-effectiveness was assessed with an incremental cost-effectiveness ratio (ICER).

The study also conducted a distributional cost-effectiveness analysis to address health inequities, comparing results to commonly used equity weights in the United States (range, 0.5-3.0). The maximum starting age for allotransplantation was set at 12 years, and gene therapy was assumed to cost $2.45 million with 100% effectiveness.

Regarding the results of their analyses, the investigators state, “Allotransplantation also appears to be an equitable therapeutic option for pediatric patients with sickle cell disease, regardless of conventional cost-ineffectiveness and as compared to SOC.”

Costs associated with each therapy were:

• $1.9 million for allotransplantation
• $2.8 million for gene therapy
• $1.0 million for SOC

Discounted lifetime QALY for each option was:

• 1 for allotransplantation
• 5 for gene therapy
• 7 for SOC

The ICERs and equity weight thresholds for allotransplantation (compared with SOC) were $138,000/QALY and 0.5, while for gene therapy (compared with allotransplantation), they were $253,000/QALY and 4.5, respectively.

“The consideration of patient-population-specific equity offers an important opportunity to derive transparent benchmarks for intertwined equity- and value-informed decision making. Quantitative consideration of equity in the context of conventional cost-ineffectiveness can be an important tipping point in justification of funding,” the researchers concluded.

Reference

Goshua G, Ito S, Chetlapalli K, et al. Allotransplantation and gene therapy equity for children with sickle cell disease: distributional cost-effectiveness of allotransplantation vs gene therapy vs standard-of-care in pediatric patients with sickle cell disease in the United States. Abstract #490. Presented at the 65th American Society of Hematology Annual Meeting; December 9-12, 2023; San Diego, California.