DeMartino P, Haag MB, Hersh AR, et al. - By performing this budget impact analysis, the budget effect as well as affordability of a gene therapy for severe sickle cell disease was estimated from the viewpoint of US Medicaid programs with the highest prevalence of sickle cell disease while investigating the effect of an annuity payment model. A mean 1-year budget effect of $29.96 million per state Medicaid program, or $1.91 per member per month, in spending was estimated in the 10 states of interest. Based on findings, it was concluded that a substantial budget impact for many Medicaid plans is likely to be generated by a gene therapy for severe sickle cell disease while potentially affording considerable advantage to patients. Taking steps to ensure affordability and access may be required from payers. Gene therapy for sickle cell disease is possibly to offer an early demonstration of the unique economical challenges related to this emerging drug class.