RT Journal Article
A1 DeMartino, Patrick C.
A1 Haag, Meredith B.
A1 Caughey, Aaron B.
A1 Roth, Joshua A.
T1 A budget impact analysis of gene therapy for sickle cell disease: an updated analysis
JF Blood Advances
JO Blood Adv
YR 2024
DO 10.1182/bloodadvances.2024013093
VO 8
IS 17
SP 4658
OP 4661
SN 2473-9529
AB TO THE EDITOR:In December 2023, the US Food and Drug Administration (FDA) approved 2 gene therapies for sickle cell disease (SCD). The data demonstrated substantial short-term clinical improvement.1,2 Approximately half of the 100 000 individuals in the United States with SCD are Medicaid enrollees.3 Allogeneic hematopoietic stem cell transplant (HSCT) is a well-described cure for the hemoglobinopathy, although its use is limited due to donor availability and toxicity.4Prices for gene therapies have made headlines, whereas the payment timing and uncertainty in treatment durability compound the challenge for payers.5 The 2 new approvals for SCD apply to a population far larger than any existing commercial gene therapy.
RD 11/15/2025
UL https://doi.org/10.1182/bloodadvances.2024013093