Gene therapy provides more quality-adjusted life-years versus non-myeloablative haploidentical allogeneic stem cell transplantation in sickle cell disease, but at a much higher cost.The cost of gene therapy would have to be reduced by about two-thirds to be as cost-effective as transplantation.This study supports the idea that patients with sickle cell disease have two curative-intent options.
Non-myeloablative haploidentical allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a more cost-effective strategy compared with gene therapy for treating sickle cell disease (SCD), researchers found.
Their cost-effectiveness analysis demonstrated that while gene therapy provides more quality-adjusted life-years (QALYs) than non-myeloablative haploidentical allo-HSCT (22.1 vs 20.1), that comes at a much higher cost -- $2.75 million versus $1.15 million, reported George Goshua, MD, of Yale University School of Medicine in New Haven, Connecticut, and colleagues in Blood.
The incremental net monetary benefit of transplantation was $1,403,000 versus gene therapy, while the incremental cost-effectiveness ratio for gene therapy was $818,000/QALY compared with transplantation.
A cost threshold analysis determined that based on results from two prospective phase II trials, the price of gene therapy in the U.S. would have to be reduced by 66% to 71% to be as cost-effective as non-myeloablative haploidentical allo-HSCT. Substantially greater reductions would be needed in lower-income countries.
