Systematic review of economic evaluations of curative treatments for sickle cell disease

Abstract

Sickle cell disease (SCD) leads to high rates of morbidity, early mortality, and high healthcare costs. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) and gene therapy represent curative approaches for sickle cell disease, however, both approaches come with risks of treatment-related toxicities, financial toxicities, and risk of mortality. Cost-effectiveness studies evaluate the financial impact of disease interventions against the gains to quality of life achieved by an intervention. Given the multiple curative interventions in sickle cell disease, including myeloablative matched-related and non-myeloablative haploidentical allogeneic transplantation as well as the novel gene therapies, the objective of this review is to summarize the methods and results of published cost-effectiveness studies of curative therapies for SCD. We analyzed economic evaluation approaches for assessing the cost-effectiveness of curative modalities in SCD including model approaches, cost input sources, health state utility values, economic perspectives, time horizons, and cost-effectiveness outcomes. We found significant heterogeneity across all assessed aspects for cost-effectiveness of curative interventions in SCD. One common theme was that most studies reported cost-effectiveness results of curative interventions at high incremental cost effectiveness ratios, suggesting curative interventions may offer a weak value proposition to the healthcare system when compared to accepted willingness- to-pay thresholds. The value proposition is improved, particularly for gene therapies, when assessed using a societal perspective. Most analyses compared only one curative modality to standard of care suggesting a need for assessing the value across curative modalities.