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

项目摘要/摘要 本申请是针对被确认为NOT-CA-23-044的特殊利益通知而提交的。 多发性骨髓瘤(MM)是一种常见的致命性血液系统恶性肿瘤。随着小说的最新发展 MM治疗后，中位存活率显著提高；然而，长期存在的种族差异 MM继续存在。这可能是因为这些治疗的费用很高，而且不公平地获得 自2021年以来，最近的通胀飙升进一步加剧了这一问题。通货膨胀率为负 受多发性骨髓瘤影响的患者，因为多发性骨髓瘤的治疗是所有癌症部位中最昂贵的。为了遏制这种情况 通货膨胀和减轻医疗保险参保人的经济负担，签署了通货膨胀降低法案(IRA) 将于2022年成为法律。在几项规定中，患者自掏腰包(OOP)支出上限为2,000美元 从2025年开始，联邦医疗保险D部分的参保人预计将显著降低飙升的OOP 成本，潜在地增加了新治疗的可及性，并缓解了被诊断为 然而，这些影响在多大程度上仍不清楚。为评估这些影响，我们建议 医保D部分OOP费用与治疗利用模式的关系研究 参加MM(目标1)的学员。此外，我们将预测IRA OOP上限对D部分参与者的影响 对于MM，包括受影响的患者群体大小、新的治疗利用情况、患者生存时间、生命周期 医疗费用和多发性骨髓瘤存活率的种族差异(目标2)。 拟议的补编将利用NCI资助的CiNet多发性骨髓瘤的既定基础设施 孵化器计划(U01CA265735)，包括1)基于证据的校准模型 来自华盛顿大学和耶鲁大学建模小组的MM的自然历史；以及2) 协调比较建模协调中心，以确保得出可靠和可信的结论 具有透明的模型假设、设计和参数化，以及高质量的 调查结果。本补充资料将扩展两个模型，旨在评估新的成本效益 对有能力评估外部经济冲击和相应经济冲击的处理 政策。扩展后的模型将显著加强父母拨款对公众健康的影响。 这份补充资料在1)预测和量化最近通货膨胀的影响方面具有重要意义 诊断MM患者的浪涌和IRA OOP帽，以更好地了解其潜在的作用 对新疗法的使用、多发性骨髓瘤存活率和多发性骨髓瘤健康差异的影响；以及2)扩展 MM的自然历史，以便评估外部冲击。它在使用的方法上也是创新的 回答研究问题，包括最先进的基于证据的MM和 比较模型，以确保得出可靠的结论。这项研究的成功完成将提供证据 在有形指标中评估最近通胀激增和多发性骨髓瘤患者的IRA OOP上限的影响。

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.