BMT CTN 1101 Ancillary Cost-Effectiveness Analysis

描述(由申请人提供)：在等待骨髓移植的患者中，多达三分之一的患者要么找不到合适的匹配供体细胞，要么需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。为了满足这一未得到满足的医疗需求，目前正在通过一项多中心、第三阶段的随机开放标签前瞻性比较试验来研究两种新的供体细胞来源，该试验是在恶性血液病患者的降低强度调节(RIC)后进行双无关脐血(DUCB)移植和人类白细胞抗原半相合相关骨髓(haplo-BM)移植(BMT CTN 1101)。这项研究将检验这一假设，即dUCB或haplo-BM在两年内的无进展生存期(PFS)相似。由于CTN 1101的发现可以极大地扩大接受骨髓移植的潜在患者数量，这项研究对患者、健康保险公司和社会具有深远的影响。骨髓移植是医疗保健中最昂贵的干预措施之一。获取和储存用于dUCB的脐带血的过程非常耗费资源：一项内部分析表明，这可能会使移植成本增加高达40%。DUCB和haplo-BM移植之间的成本预测差异，再加上植入率、感染风险和PFS方面的不确定差异，造成了关于这些疗法的相对成本效益的高度不确定性，因此为经济分析提供了一个令人信服的案例。CTN 1101还提供了一个独特的机会来前瞻性地评估骨髓移植对患者和家庭的财务影响。虽然人们普遍认为BMT对家庭来说是经济负担，但BMT对经济影响的这一方面的真正范围从未被系统地研究过。鉴于BMT对患者和照顾者的深远而持久的影响，传统的调查方法(邮寄或电话调查)可能会带来问题。在这项研究中，我们建议使用新颖的基于网络的调查工具，这些工具可以在家用电脑和许多手机上完成。总之，我们的研究将首次从多个角度对BMT的经济影响提供高质量的全面评估：患者和家庭、健康保险公司和社会。因为从移植开始的前瞻性评估对于确保我们的记录的准确性和完整性至关重要，所以这项辅助研究代表着一个时间敏感的机会来开始这项平行的经济学评估。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评估dUCB和haplo-BM作为RIC骨髓移植替代供体细胞来源的成本效益。临床决策者可以使用这项分析的数据来(1)使用这些替代方案对移植的预算影响做出重要预测，以及(2)协助做出符合患者和使用有限资源的支付者的最佳利益的保险覆盖决定。

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.