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

Author:

Roth Joshua A12,Bensink Mark E1,O’Donnell Paul V34,Fuchs Ephraim J5,Eapen Mary6,Ramsey Scott D7

Affiliation:

1. Public Health Sciences Division, Fred Hutchinson Cancer Research Centre, 1100 Fairview Avenue North, Seattle, WA 98109, USA

2. Group Health Research Institute, Group Health Cooperative, 1730 Minor Avenue, Seattle, WA 98101, USA

3. Clinical Research Division, Fred Hutchinson Cancer Research Centre, 1100 Fairview Avenue North, Seattle, WA 98109, USA

4. Department of Medicine, University of Washington, WA, USA

5. John Hopkins University School of Medicine, 1650 Orleans Street, Baltimore, MD 21287, USA

6. Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA

7. Public Health Sciences Division, Fred Hutchinson Cancer Research Centre, 1100 Fairview Avenue North, Seattle, WA 98109, USA.

Abstract

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.

Publisher

Future Medicine Ltd

Subject

Health Policy

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