Annals of Internal MedicineDistributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States

Unraveling the Economic Equilibrium: Gene Therapy in Sickle Cell Disease

A novel study investigating the economic and equitable value of gene therapy in sickle cell disease (SCD) patients underscores the potential of this treatment approach from an equity perspective, while challenging its cost-effectiveness.

Key Points:

• The study used conventional cost-effectiveness analysis (CEA) and distributional CEA (DCEA) to compare gene therapy with the standard of care (SOC) in SCD patients.
• Gene therapy, administered at age 12, yielded 25.5 discounted lifetime quality-adjusted life-years (QALYs) for females and 24.4 for males, at costs of $2.8 million per patient.
• Standard of care (SOC) resulted in 15.7 (females) and 15.5 (males) discounted lifetime QALYs, costing $1.0 million (females) and $1.2 million (males).
• The incremental cost-effectiveness ratio (ICER) was $176,000 per QALY for the entire SCD population.
• To meet DCEA standards and favor gene therapy, the inequality aversion parameter must be 0.90 for the full SCD population.

Additional Points:

• Sensitivity analysis showed that SOC was preferred in 100.0% (females) and 87.1% (males) of 10,000 probabilistic iterations at a willingness-to-pay threshold of $100,000 per QALY.
• To meet conventional CEA standards, the cost of gene therapy would need to be less than $1.79 million.
• The study used benchmark equity weights, not SCD-specific weights, to interpret DCEA results.

Related Link:

• Novel Gene Therapy Shows Positive Initial Results in Sickle Cell Patients Treated in Clinical Trial at Cleveland Clinic Children’s

Did You Know?
In sickle cell disease, the peculiar “sickle” shape of red blood cells isn’t just a morphological oddity. It’s a case of protein misbehavior – the sickle shape is due to abnormal hemoglobin (HbS) distorting the normally flexible red cells into rigid sickles under low-oxygen conditions, leading to vaso-occlusion and a cascade of clinical complications. This makes SCD a prime candidate for gene therapy interventions, potentially transforming disease management, even if challenges around cost-effectiveness remain.