Gene Therapy for Sickle Cell Disease Cost Effective in Reducing Health Disparities


A DCE analysis weighed gender-, age-, and disease-severity-specific annual costs as opposed to traditional cost-effectiveness models.

A distributional cost-effectiveness analysis (DCEA) has revealed that gene therapy may be an equitable treatment strategy for patients with sickle cell disease (SCD) in the US.

The results of the analysis were presented at the 64th American Society of Hematology (ASH) Annual Meeting, December 10-12, 2022, in New Orleans, Louisiana, by George Goshua, MD, MSc, Assistant Professor of Medicine, Yale School of Medicine.

“Cost effectiveness analysis conventionally does not cover the impact of health inequities, but DCEA does. This is a new methodology out of the University of York, and we are the first to apply it in the hematologic oncologic space,” Goshua said during his presentation.

Goshua and colleagues built a Markov simulation model of patients with mild, moderate, or severe SCD and used 10 years of real-world data including gender-, age-, and disease-severity-specific annual costs and transition probabilities between severities to analyze cost-effectiveness. These factors were weighted as inequality aversion parameters as part of the DCEA. To analyze traditional cost-effectiveness, they used an incremental cost-effectiveness ratio (ICER) and a cost-effectiveness threshold of $100,000/quality-adjusted life-years (QALYS). They picked a base-case gene therapy assumed to cost $2.1 million, cause 100% disease remission, and treat patients with a minimum age of 12 years.

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The researchers found that gene therapy was cost-effective when considering equitable health care and reducing disparities in health care treatments. Specifically, the base-case gene therapy at age 12 yields 25.5 discounted lifetime QALYs at a cost of $2.4 million compared to standard-of-care (SOC) which yields 16.0 QALYS at $1.1 million. While the ICER is $144,000/QALY for gene therapy and $100,000/QALY for SOC, when applying the inequality aversion parameters in the DCEA, the threshold is 1.7 which is within the normal US range (0.5-3.0) and well below the UK range (11.0-28.9), representing a favorable, disparity-reducing, cost-effectiveness of gene therapy.

“While gene therapy may exceed conventional cost-effectiveness standards, if we actually care about equity and under the DCEA and using typical equity weights in the US, we can see that this can be truly an equitable therapy for our patients. Future studies to determine these equity weights across diseases and disparities are needed if this is where we want to move to as a field and in clinical medicine in general if we care about equity – these are the studies that can inform equitable and value-based benchmarks,” Goshua concluded his presentation.

Click here to read more coverage of the ASH 2022 meeting.

Goshua G, Calhoun C, Cheng V, et al. Gene therapy equity in sickle cell disease: Distributional cost-effectiveness analysis (DCEA) of gene therapy vs. standard-of-care in patients with sickle cell disease in the United States. Presented at: 64th ASH Annual Meeting, December 10-12, in New Orleans, Louisiana. Abstract #581.
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