BMT CTN 1101 Ancillary Cost-Effectiveness Analysis

描述（由申请人提供）：多达三分之一的等待骨髓移植的患者要么找不到合适的供体细胞，要么要等待一段时间才能找到匹配的细胞，这使他们面临死于疾病的风险。为了解决这一未满足的医疗需求，两种新的供体细胞来源正在通过一项多中心、III期、随机、开放标签、前瞻性、比较试验进行研究，即双非相关脐血（dUCB）与hla -单倍体相关骨髓（haploo - bm）移植后降低强度调节（RIC）对血液恶性肿瘤患者（BMT CTN 1101）。该研究将验证dUCB或单倍型乳腺癌2年无进展生存期（PFS）相似的假设。由于CTN 1101的研究结果可以极大地扩大接受BMT治疗的潜在患者数量，因此该研究对患者、医疗保险公司和社会都具有深远的意义。BMT是医疗保健中最昂贵的干预措施之一。为dUCB获取和储存脐带血的过程非常耗费资源：一项内部分析表明，这可能使移植成本增加多达40%。dUCB和单倍骨髓移植之间预计的成本差异，加上移植、感染风险和PFS方面的不确定性差异，对这些疗法的相对成本效益产生了高度的不确定性，从而为经济分析提供了一个令人信服的案例。CTN 1101也提供了一个独特的机会来前瞻性地评估BMT对患者和家庭的经济影响。虽然人们普遍认为BMT给家庭带来了经济负担，但BMT对经济影响的真正范围从未得到系统研究。鉴于BMT对患者和护理人员的深远和长期影响，传统的调查方法（邮寄或电话调查）可能会造成问题。在这项研究中，我们建议使用新颖的基于网络的调查工具，这些工具可以通过家用电脑和许多移动电话完成。总之，我们的研究将首次从患者、家庭、健康保险公司和社会等多个角度，对BMT的经济影响进行高质量、全面的评估。因为从移植开始的前瞻性评估对于确保我们记录的准确性和完整性至关重要，这项辅助研究代表了一个时间敏感的机会，可以开始这种平行的经济评估。总体目标是收集足够的数据，从社会和健康保险公司的角度估计作为RIC BMT替代供体细胞来源的dUCB与单倍体骨髓移植的成本效益。临床决策者可以使用该分析的数据来(1)对使用这些替代方案的移植的预算影响做出重要预测，(2)协助做出符合患者和有限资源的付款人的最佳利益的保险覆盖决策。

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. PUBLIC HEALTH RELEVANCE: The Bone Marrow Transplant (BMT) Clinical Trails Network (CTN) is investigating the best graft source for adult patients requiring alternative donor cells sources for transplantation. Differences in the cost and quality of life provided by these alternatives make this proposed parallel cost- effectiveness study an important addition to the parent study. Clinical decision-makers and patients 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.