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.

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