Comparative modeling of multiple myeloma across myeloma control continuum: prevention, treatment, and disparity reduction

跨骨髓瘤控制连续体的多发性骨髓瘤的比较模型：预防、治疗和缩小差异

基本信息

批准号：
10831693
负责人：
GRAHAM A. COLDITZ
金额：
$ 13.89万
依托单位：
WASHINGTON UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-09-20 至 2026-08-31
项目状态：
未结题

项目摘要

PROJECT SUMMARY/ABSTRACT This application is being submitted in response to the Notice of Special Interest identified as NOT-CA-23-044. Multiple myeloma (MM) is a common and lethal hematologic malignancy. With the recent development of novel MM treatments, median survival has significantly improved; however, the long-established racial disparities in MM continue to exist. This may be attributable to the high cost of these treatments and inequitable access to care, which was further exacerbated by the recent inflation surge since 2021. The inflation has negatively impacted patients with MM, as the management of MM is the most costly among all cancer sites. To curb this inflation and reduce the financial burden of Medicare enrollees, the Inflation Reduction Act (IRA) was signed into law in 2022. Among several provisions, the cap placed on patient out-of-pocket (OOP) spending of $2,000 starting in 2025 for Medicare Part D enrollees is expected to significantly reduce the skyrocketing high OOP cost, potentially increase accessibility of novel treatments, and mitigate disparities for patients diagnosed with MM. However, to what extent these impacts remains unknown. To assess these impacts, we propose to investigate the relationship between OOP costs and treatment utilization pattern among Medicare Part D enrollees with MM (Aim 1). Furthermore, we will predict the impact of the IRA OOP cap on Part D enrollees with MM, including the impacted size of patient population, novel treatment utilization, patient survival, lifetime healthcare cost, and racial disparities in MM survival (Aim 2). The proposed supplement will utilize the established infrastructure of NCI-funded CISNET Multiple Myeloma Incubator Program (U01CA265735), including 1) CISNET-standard, evidence-based, calibrated models of natural history of MM from Washington University and Yale University modeling groups; and 2) the coordination of the Coordinating Center for comparative modeling to ensure robust and credible conclusions with transparent model assumptions, design, and parameterization, as well as high-quality reporting of the findings. This supplement will expand the two models designed to evaluate the cost-effectiveness of novel treatments to ones with the capability to evaluate external economic shocks and the corresponding economic policies. The expanded models will significantly strengthen the public health impact of the parent grant. This supplement is significant in its capability to 1) predict and quantify the impacts of the recent inflation surge and the IRA OOP cap in patients diagnosed with MM to better our understanding about the potential impacts on novel treatment utilization, MM survival, and MM health disparities; and 2) expand the models for natural history of MM to allow for the assessment of external shocks. It is also innovative in its approach used to answer the research question, including state-of-the-art, evidence-based simulation models for MM and comparative modeling to ensure robust conclusions. Successful completion of this study will provide evidence in tangible metrics to assess the impacts of the recent inflation surge and the IRA OOP cap in MM patients.

项目摘要/摘要该申请是为了响应被确定为非CA-23-044的特殊利益通知。多发性骨髓瘤（MM）是一种常见且致命的血液系统恶性肿瘤。随着小说的最新发展 MM治疗，中位生存率显着改善；但是，长期以来的种族差异 MM继续存在。这可能归因于这些治疗的高成本和无法获得自2021年以来，最近的通货膨胀率激增进一步加剧了护理。通货膨胀率负面受到MM的影响患者，因为MM的管理是所有癌症部位中最昂贵的。遏制这个通货膨胀并减轻医疗保险参与者的财务负担，签署《降低通货膨胀法》（IRA）在2022年进行法律。在几项规定中，上限放置在患者自付费用（OOP）支出2,000美元从2025年开始，医疗保险D部分将显着降低高空高OOP 成本，可能增加新治疗的可及性以及减轻诊断患者的差异毫米。但是，这些影响在多大程度上尚不清楚。为了评估这些影响，我们建议调查Medicare D部分的OOP成本与治疗利用模式之间的关系带有MM的参与者（AIM 1）。此外，我们将预测IRA OOP上限对D部分的影响 MM，包括患者人群的影响，新型治疗利用，患者生存，终生 MM生存中的医疗保健成本和种族差异（AIM 2）。拟议的补充剂将利用NCI资助的CISNET多发性骨髓瘤的既定基础设施孵化器程序（U01CA265735），包括1）顺式标准，基于证据的，校准的模型华盛顿大学和耶鲁大学建模小组的MM自然历史； 2）比较建模协调中心的协调，以确保结论可靠和可靠的结论具有透明的模型假设，设计和参数化以及高质量的报告发现。该补充剂将扩大旨在评估新颖成本效益的两种模型对具有评估外部经济冲击和相应经济能力的能力的治疗政策。扩展的模型将大大加强父母赠款的公共卫生影响。这种补充在其能力上很重要1）预测和量化最近通货膨胀的影响临时患者的激增和IRA OOP帽，以更好地了解潜在的潜力对新型治疗利用，MM生存和MM健康差异的影响； 2）扩展模型 MM的自然历史可以评估外部冲击。它的方法也是创新的回答研究问题，包括MM和MM的最先进的基于证据的仿真模型比较建模以确保结论可靠。这项研究的成功完成将提供证据在有形指标中，评估了近期通货膨胀率和IRA OOP CAP对MM患者的影响。