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Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States

2023; American College of Physicians; Volume: 176; Issue: 6 Linguagem: Inglês

10.7326/m22-3272

1539-3704

George Goshua, Cecelia Calhoun, Satoko Ito, Lyndon P. James, Andrea Luviano, Lakshmanan Krishnamurti, Apurvakumar Pandya,

Prenatal Screening and Diagnostics

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.