ASH 2022 | Distributional cost-effectiveness analysis of gene therapy vs SOC in patients with SCD in the US

The approval of gene therapy in the United States (US) could enable patients with sickle cell disease (SCD) to achieve lifelong disease remission without the toxicity of allogeneic transplantation. However, many project that gene therapy will be a cost-ineffective investment for the US. To address this, George Goshua, MD, MSc, Yale University School of Medicine, New Haven, CT, presents results from the first distributional cost-effectiveness analysis (DCEA), which quantifies the tradeoffs between traditional cost-effectiveness outcomes and health equity for gene therapy in SCD. The analysis used a Markov simulation model of patients with a diagnosis of mild, moderate, or severe SCD to examine the cost-effectiveness of gene therapy versus patients treated with standard-of-care (SOC). The DCEA accounted for health disparities in SCD, and based on an ability to reduce these, favors gene therapy over SOC. The incremental cost-effectiveness ratio (ICER) for SCD gene therapy was determined as $144,000/ quality-adjusted life year (QALY), suggesting that gene therapy is not a good value for money when compared to the cost-effectiveness threshold of $100,000/QALY. Overall, Dr Goshua believes that gene therapy can be used as an equitable therapeutic strategy for patients with SCD, assuming there’s similar therapeutic efficacy of gene therapy across disease severity. This press briefing took place at the 64th ASH Annual Meeting and Exposition congress in New Orleans, LA.

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