Design of a cost-effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer

Background: BMT CTN 1101 is a Phase III randomized controlled trial evaluating the comparative effectiveness of double unrelated umbilical cord blood (dUCB) versus HLA-haploidentical related donor bone marrow (haplo-BM) donor cell sources for blood or bone marrow transplantation (BMT) in patients with hematologic malignancies. Herein, we present the rationale, design and methods of the first cost-effectiveness analysis to be conducted alongside a BMT trial. Methods: Consenting patients will provide health insurance information to allow calculation of direct medical costs from reimbursement records, and will provide out-of-pocket costs, time costs and health-related quality of life measures through an online survey. These outcomes will inform a cost-effectiveness analysis comparing dUCB and haplo-BM donor cell sources from patient, payer and societal perspectives. Conclusion: Novel approaches may significantly change the cost, outcomes or availability of BMT. The results of this analysis will be the first to provide a comprehensive evaluation of the comparative effectiveness of these approaches from multiple perspectives.