PRO13 Economic IMPACT of Sickle Cell Disease, Treatments, and Related Complications

Abstract

Sickle cell disease (SCD) is a complex genetic disorder that begins in infancy and manifests throughout life. Patients and caregivers experience reductions in quality of life, productivity, and lifetime income. Although upfront costs of potentially curative genetic therapies will be high, improved productivity and a reduction in downstream costs will benefit patients, caregivers, and healthcare systems. Characterizing these economic impacts will aid our current understanding and inform value assessments as genetic therapies are marketed. Summarize medical literature that describes the medical and nonmedical costs of SCD burden, treatment/ complications, productivity, and lifetime income. We searched MEDLINE and EMBASE (2008-2020) and identified studies that detailed medical or nonmedical costs. Eligible studies included those that provided information about any aspect of cost for SCD individuals of all ages and their caregivers. Treatments included hydroxyurea, transfusions, voxelotor, crizanlizumab, and hematopoietic stem cell transplantation (HSCT). One reviewer screened, extracted, and quality appraised the studies. Costs were adjusted to 2019 USD. Search queries returned 479 studies. Forty-one studies contained relevant cost information - 39 detailed medical and two detailed non-medical costs. Costs for SCD individuals trended higher than costs for non-SCD individuals across several complications, ranging from childbirth ($16,007 vs $13,365) to cholecystectomy ($46,897 vs $34,846). Inpatient costs were the largest component of lifetime costs; hydroxyurea and transfusions decreased these amounts. After high first year costs ($135,568-$467,747), HSCT decreased medical costs over the ensuing several years by up to 43%. Lifetime income was significantly lower in individuals with SCD. No study captured caregiver or productivity costs, or the trajectory of costs throughout the lifespan. Evidence suggests that current therapies can reduce the economic burden of SCD, but additional estimates are warranted to fill remaining gaps and capture costs across the lifetime disease trajectory.