Hemoglobinopathies

In this study, researchers from the Center for Biomedical Intervention at the Massachusetts Institute of Technology and the Department of Pharmaceutical and Health Economics at the University of Southern California’s School of Pharmacy investigated the potential cost-effectiveness (CE) of a durable cell or gene therapy cure for SCD from the US healthcare sector perspective. They developed a lifetime Markov model to evaluate the CE of standard-of-care therapy vs a hypothetical single-administration durable treatment provided at birth to infants with SCD. Based on their findings, the authors concluded that “[a] hypothetical cell or gene therapy cure for SCD is likely to be cost-effective from the US healthcare sector perspective,” noting that “[l]arge upfront costs of a single administration cure are offset by significant downstream gains in health for patients treated early in life.”