BMT CTN 1101 Ancillary Cost-Effectiveness Analysis

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DESCRIPTION (provided by applicant): Up to a third of patients awaiting a bone marrow transplant will either not find suitably matched donor cells or will encounter a waiting period to find a match that puts them at risk of succumbing to their disease. To address this unmet medical need, two novel sources of donor cells are now being studied through a multi- center, Phase III, randomized, open label, prospective, comparative trial of double unrelated umbilical cord blood (dUCB) versus HLA-haploidentical related bone marrow (haplo-BM) transplantation after reduced intensity conditioning (RIC) in patients with hematologic malignancies (BMT CTN 1101). The study will test the hypothesis that progression-free survival (PFS) at two years is similar for dUCB or haplo-BM. Because the findings from CTN 1101 could greatly expand the potential number of patients who are offered BMT, the study has profound implications for patients, health insurers, and society. BMT is one of the most expensive interventions in health care. The process of acquiring and storing cord blood for dUCB is very resource intensive: an internal analysis suggests it can increase the costs of transplantation by up to 40%. The projected differences in cost between dUCB and haplo-BM transplantation-combined with uncertain differences in engraftment, infection risk and PFS--creates a high degree of uncertainty regarding the relative cost-effectiveness of these therapies, thus providing a compelling case for an economic analysis. CTN 1101 also provides a unique opportunity to prospectively evaluate the financial impact of BMT on patients and families. Although it is widely acknowledged that BMT is financially burdensome for families, the true scope of this aspect of BMT's economic impact has never been systematically studied. Given the profound and prolonged impact of BMT on patients and caregivers, traditional survey methods (mailed or telephone- based surveys) can pose problems. In this study, we propose to use novel web-based survey tools that can be completed from home computers and many mobile phones. In summary, our study will be the first to provide a high quality, comprehensive evaluation of the economic impact of BMT, from multiple perspectives: patients and family, health insurers, and society. Because prospective evaluation from the start of transplant is critical to ensure the accuracy and completeness of our records, this ancillary study represents a time-sensitive opportunity to begin this parallel economic evaluation. The overall objective is to collect sufficient data to estimate the cost-effectiveness of dUCB versus haplo-BM as alternative donor cell sources for RIC BMT from the societal and health insurer perspectives. Clinical decision-makers can use data from this analysis to (1) make important projections of the budget impact of transplantation using these alternatives and, (2) assist with insurance coverage decisions that are in the best interest of both patients and payers working with limited resources.