A Budget Impact Analysis of Gene Therapy for Sickle Cell Disease: The Medicaid Perspective.

ABSTRACT:

Importance

Hundreds of gene therapies are undergoing clinical testing and are likely to be priced more than $1 million per course of treatment. The association that high prices will have with insurance coverage of gene therapy remains unclear. Gene therapy for sickle cell disease has shown early success and would be one of the first gene therapies available for a relatively large population.

Objectives

To estimate the budget impact and affordability of a gene therapy for severe sickle cell disease from the perspective of US Medicaid programs with the highest prevalence of sickle cell disease while exploring the impact of an annuity payment model.

Design, setting, and participants

A budget impact analysis was performed from January 1 to May 31, 2020, for a sickle cell

SUBMITTER: DeMartino P 

PROVIDER: S-EPMC7985816 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature