Annals of Internal Medicine
February 2024 Volume 177, Issue 2
Original Research
Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States – A Cost-Effectiveness Analysis
Anirban Basu, PhD, … et al.
Pages:155–164
The U.S. Food and Drug Administration recently approved 2 genetic therapies for sickle cell disease. Both therapies increase the production of nonsickling red blood cells, either fetal hemoglobin or a gene therapy–derived hemoglobin similar to hemoglobin A. In addition, both therapies require collection of a patient’s stem cells, modification of the cells in the laboratory, high-dose chemotherapy to remove existing bone marrow cells, and a single-dose infusion of the patient’s modified stem cells. This article uses 2 different cost-effectiveness models to estimate whether these therapies provide enough value to justify their high costs.