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

靶向口服抗癌药物：适应症和标签外使用模式、相关因素和经济影响

• 批准号: 10304880
• 负责人: Ya-Chen Tina Shih
• 金额: $ 34.99万
• 依托单位: UNIVERSITY OF TX MD ANDERSON CAN CTR
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-12-06 至 2023-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10304880
• 关键词: Address; Adherence; Affect; Antineoplastic Agents; Area; Attention; Breast Cancer Cell; Cancer Patient; Chronic; Chronic Myeloid Leukemia; Clinical; Data; Databases; Diagnosis; Disease Progression; Disincentive; Drug Costs; Drug usage; Economics; Effectiveness; Environment; Financial Hardship; Goals; Guidelines; Health Care Costs; Health Services Research; Home; Income; Institutes; Institution; Insurance; Intravenous; Knowledge; Label; Laws; Life; Literature; Malignant Neoplasms; Medical Oncology; Medicare; Modernization; Oncologist; Oncology; Oral; Patients; Pattern; Pharmaceutical Preparations; Policies; Policy Maker; Price; Provider; Renal Cell Carcinoma; Research; Role; Safety; Scientific Advances and Accomplishments; Series; Systemic Therapy; Testing; Therapeutic; Time; Tyrosine Kinase Inhibitor; Uninsured Medical Expense; United States Food and Drug Administration; anti-cancer; barrier to care; base; cancer care; cancer cell; cancer survival; cancer type; care costs; care delivery; chemotherapy; chronic myeloid leukemia cell; clinical application; cohort; cost; design; drug development; economic impact; economic implication; experience; health economics; improved; innovation; multidisciplinary; neuroendocrine cancer; novel; novel therapeutics; off-label use; parity; payment; rare cancer; research study; targeted cancer therapy; treatment adherence; trend

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.

项目摘要 靶向口服抗癌药（TOAMS）过去一直是肿瘤药物开发的重点 尽管这些新型药物有助于几种癌症的生存改善，并且是 方便管理，高级药品价格引起了人们对其可承受能力的担忧，可能的较低 高自付费用驱动的治疗依从性。当前有关利用的知识和 TOAMS的依从性在很大程度上是基于对慢性粒细胞性白血病患者的研究。它仍然存在 尚不清楚这种知识是否直接转移给其他癌症，尤其是口腔和口服的癌症 静脉注射抗癌药作为可行的治疗选择。 该研究的具体目的是：（1）检查TOAMS的利用模式，如所示与标签外 使用; （2）估计使用TOAM的影响对癌症护理的整体成本的影响，非激素的成本 系统性疗法以及自付费用； （3）研究与该计划相关的因素 托姆和治疗计划的时间； （4）探索治疗开关的模式和 开始使用TOAM进行全身治疗的患者中的停药和相关因素。 我们将使用从三个大型的癌症患者中提取的各种癌症患者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Toams在现代癌症护理中的作用 系统地研究TOAMS的利用模式和经济影响以及影响的因素 这些新型疗法的启动和停用，特别注意患者的财务因素 和提供者。我们研究的发现将告知患者，临床医生和政策制定者 在现实世界中实践了哪种标签的托姆处方以及toam的长期经济影响 使用并提供关键信息来指导改革或重新设计保险单，以避免创建 财务不利于开处方或使用Toam。最终，我们研究的目的是改善 TOAMS的可访问性，可用性，有效性和安全性。