The value-based price of transformative gene therapy for sickle cell disease: a modeling analysis.

ABSTRACT: Sickle cell disease (SCD) is an inherited, progressively debilitating blood disorder. Emerging gene therapies (GTx) may lead to a complete remission, the benefits of such can only be realized if GTx is affordable and accessible in the low-and middle-income countries (LMIC) with the greatest SCD burden. To estimate the health impacts and country-specific value-based prices (VBP) of a future gene therapy for SCD using a cost-utility model framework. We developed a lifetime Markov model to compare the costs and health outcomes of GTx versus standard of care for SCD. We modeled populations in seven LMICs and six high-income countries (HICs) estimating lifetime costs and disability-adjusted life-years (DALYs) in comparison to estimates of a country's cost-effectiveness threshold. Each country's

SUBMITTER: Morgan G 

PROVIDER: S-EPMC10834512 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature