Targeted Oral Anticancer Agents: Patterns of Indicated and Off-Label Use, the Associated Factors, and Economic Implications

项目总结 靶向口服抗癌药物(TOAM)过去一直是肿瘤药物开发的重点 二十年了。尽管这些新药有助于提高几种癌症的存活率，而且 由于便于管理，高药价引起了人们对其负担能力的担忧，甚至可能较低 高额的自付费用推动了治疗的坚持。关于利用和利用的最新知识 TOAM的依从性很大程度上建立在对慢性粒细胞白血病患者的研究基础上。它仍然存在 不清楚这种知识是否可以直接转移到其他癌症，特别是口腔和口腔癌 静脉注射抗癌药物作为可行的治疗选择。 这项研究的具体目的是：(1)考察标签外与标签外的TOAM的使用模式 使用；(2)评估TOAM使用对癌症护理总成本、非激素成本的影响 系统治疗，以及自付费用；(3)调查与启动相关的因素 TOAM和开始治疗的时间；以及(4)探索治疗切换和 开始使用TOAM进行系统治疗的患者中的停药及其相关因素。 我们将使用从三个大公司提取的癌症患者的不同队列来解决我们的研究问题 索赔数据库：SEER-Medicare、MarketScan和医疗保健成本机构数据。 我们将进行一系列研究，以更好地了解TOAM在现代癌症护理服务中的作用 系统研究了TOAM的利用模式、经济影响及影响因素。 这些新疗法的开始和停止，特别关注患者的经济因素 和提供者。我们的研究结果将告知患者、临床医生和政策制定者以下方面的程度 TOAM的哪些标签外处方在现实世界中实践以及TOAM的长期经济影响 使用并提供关键信息来指导保险单的改革或重新设计，以避免创建 经济上不鼓励开出或使用TOAM。归根结底，我们的研究目标是改善 TOAM的可及性、可负担性、有效性和安全性。

PROJECT SUMMARY Targeted oral anticancer medications (TOAMs) have been the focus of oncology drug development in the past two decades. Although these novel agents contribute to survival improvement in several cancers and are convenient to administer, high drug prices have raised concerns on their affordability and possibly low treatment adherence driven by high out-of-pocket expenses. Current knowledge on the utilization and adherence of TOAMs is largely built upon studies of patients with chronic myelogenous leukemia. It remains unclear whether such knowledge is directly transferrable to other cancers, especially those with both oral and intravenous anticancer medications as viable treatment options. The specific aims of the study are: (1) to examine the utilization patterns of TOAMs as indicated vs. off-label use; (2) to estimate the impact of TOAM use on the overall costs of cancer care, costs of non-hormonal systemic therapies, as well as out-of-pocket payment; (3) to investigate the factors associated with the initiation of a TOAM and time to treatment initiation; and (4) to explore patterns of treatment switches and discontinuation and the associated factors among patients who started their systemic therapy with a TOAM. We will address our research questions using various cohorts of cancer patients extracted from three large claims databases: the SEER-Medicare, MarketScan, and Health Care Cost Institution data. We will conduct a series of studies to better understand the role of TOAMs in modern cancer care delivery by systematically studying the utilization pattern and economic impact of TOAMs as well as factors affecting the initiation and discontinuation of these novel therapies, paying special attention to financial factors for patients and providers. The findings from our study will inform patients, clinicians, and policy makers of the extent to which off-label prescription of TOAM is practiced in the real-world and the long-term economic impact of TOAM use, and provide critical information to guide reform or re-design of insurance policies to avoid creating financial disincentive to prescribe or use TOAM. Ultimately, the goal of our research is to improve the accessibility, affordability, effectiveness, and safety of TOAMs.