Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States | Annals of Internal Medicine
Background: Gene therapy is a potential cure for sickle cell disease (SCD). Conventional cost-effectiveness analysis (CEA) does not capture the effects of treatments on disparities in SCD, but distributional CEA (DCEA) uses equity weights to incorporate these considerations. Objective: To compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA. Design: Markov model. Data Sources: Claims data and other published sources. Target Population: Birth cohort of patients with SCD. Time Horizon: Lifetime. Perspective: U.S. health system. Intervention: Gene therapy at age 12 years versus SOC. Outcome Measures: Incremental cost-effectiveness ratio (ICER) (in dollars per quality-adjusted life-years [QALYs] gained) and threshold inequality aversion parameter (equity weight). Results of Base-Case Analysis: Gene therapy versus SOC for females yielded 25.5 versus 15.7 (males: 24.4 vs. 15.5) discounted lifetime QALYs at costs of $2.8 million and $1.0 m