Comparative Effectiveness and Cost Effectiveness of Cancer Chemotherapy

癌症化疗的比较效果和成本效益

• 批准号: 8611911
• 负责人: XIANGLIN DU
• 金额: $ 29.36万
• 依托单位: UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-04-01 至 2017-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8611911
• 关键词: Comparative; Effectiveness; Cost; Cancer; Chemotherapy; Comparative; Effectiveness; Cost; Cancer; Chemotherapy

Project Abstract Chemotherapy has been documented to be efficacious in prolonging survival for patients with various malignancies. One critical finding from numerous clinical trials and pooled analyses is that the efficacy of chemotherapy for breast cancer has been shown to decrease with age, from a 27% proportional reduction in 10-year mortality for women less than 50 years of age to 14% for women ages 50-59 years and 8% for women ages 60-69 years, and no benefit for women 70 years or older. These findings have remained constant even when the pooled results were examined with the addition of more patients from more recent trials in 1998, 2001, 2005 and 2008. These results are striking because there has been no decreasing efficacy of chemotherapy observed with advancing age for women with ovarian cancer, and for men and women with lung cancer and colon cancer. Numerous clinical trials have shown that chemotherapy is equally efficacious across different age groups of men and women with lung and colon cancer as well as women with ovarian cancer, including populations 70 years or older. Therefore, evidence-based clinical guidelines on chemotherapy from the National Institutes of Health (NIH) and other health authorities recommend chemotherapy for women with breast cancer less than 70 years old, whereas there are no age-restricted recommendations for patients with colon, ovarian and lung cancer. In other words, chemotherapy is strongly recommended for patients of all age groups with colon, ovarian and lung cancer. Randomized controlled clinical trials are considered to be the gold standard for determining the efficacy of a therapy. However, participants in clinical trials often do not represent a cross-section of patients in the community, and older patients were significantly under-represented in clinical trials. Population-based observational studies can therefore be useful adjuncts to randomized controlled trials in determining whether efficacy under controlled conditions in specialty centers translates into real-world effectiveness in the community and provide valuable insight into the benefit of a therapy in community practices that cannot be obtained from the clinical trials.Furthermore, in some circumstances where the randomized trials are not possible due to ethical or logistical issues, well-conducted observational studies can potentially serve to determine the comparative effectiveness of various drug therapies. Therefore, there is a critical need for information on whether the effectiveness of chemotherapy decreases with age for breast cancer only (as shown in clinical trials), or also decreases with age for lung, colorectal, and ovarian cancer and on whether chemotherapy is cost effective by different age groups and by tumor site. The specific aims of this proposal are to: 1) determine whether the stage-specific efficacy of chemotherapy observed in controlled clinical trials is translated into real-world effectiveness in prolonging survival among community dwelling patients with breast, ovarian, lung and colorectal cancer at age 65 years or older; 2) determine the association between the effectiveness of chemotherapy and advancing age (i.e., to determine whether chemotherapy is equally effective for all age groups of patients with these tumors); 3) determine the comparative effectiveness of various chemotherapy agents or combination regimen-specific benefits in prolonging survival and their associations with age and tumor type; 4) determine whether and to what extent patient and tumor characteristics as well as hospital and physician factors affect the relationship between the effectiveness of chemotherapy and age; and 5) to determine the cost- effectiveness of chemotherapy by age and tumor type. Our approach is to identify a large nationwide, population-based cohort of over 550,000 patients diagnosed with breast, ovarian, lung and colorectal cancer at age 65 years or older in 1991 through 2005 (with more cancer cases in 2006- 2007 from the new data linkage in early 2011) from the 17 Surveillance, Epidemiology, and End Results (SEER) registries, accounting for over 25% of the U.S. population. The large numbers of patients will enable us to examine the comparative effectiveness of chemotherapy in many subpopulations. This proposed study will examine the comparative- and cost-effectiveness of chemotherapy in association with advancing age across four major tumors in the real world community setting, which has never been tested before. The proposed innovative use of the nationwide and population-based Medicare claims data, which provide information on specific chemotherapy drug or regimens, will also allow for chemotherapy-specific effectiveness analysis.

项目摘要 化学疗法已被证明可以有效地延长 各种恶性肿瘤。来自众多临床试验和合并分析的一个关键发现是 化学疗法对乳腺癌的功效已显示出随着年龄的增长而降低的，从27％ 女性年龄少于50岁的妇女的10年死亡率比例降低到14％ 60-69岁的女性为50-59岁，为70岁以上的妇女没有好处。这些 即使在添加更多的情况下检查了汇总结果，调查结果仍然保持恒定 1998年，2001年，2005年和2008年最近试验的患者。这些结果令人惊讶，因为那里 随着卵巢女性的增长，观察到的化学疗法的疗效并没有降低 癌症，以及患有肺癌和结肠癌的男性和女性。许多临床试验已显示 这种化学疗法在不同年龄段的男性和女性患有肺和肺和女性的群体中同样有效 结肠癌以及卵巢癌的女性，包括70岁或以上的人群。所以， 国家卫生研究院（NIH）和其他的基于证据的临床化学疗法临床指南 卫生当局建议针对不到70岁的乳腺癌女性化疗， 尽管没有针对结肠，卵巢癌和肺癌患者的年龄限制建议。在 换句话说，强烈建议对所有年龄段的患者，卵巢和卵巢和 肺癌。随机对照临床试验被认为是确定的黄金标准 治疗的功效。但是，临床试验的参与者通常不代表 在临床试验中，社区中的患者和老年患者的代表性明显不足。 因此 确定专业中心的受控条件下的疗效是否转化为现实世界 在社区中的有效性，并为社区疗法的利益提供宝贵的见解 无法从临床试验中获得的实践。在某些情况下， 由于道德或后勤问题，不可能进行随机试验，该试验良好的观察性研究 可以潜在地确定各种药物疗法的比较有效性。所以， 关于化学疗法的有效性是否随着年龄的增长而降低的迫切需要信息 仅用于乳腺癌（如临床试验中所示），或者随着肺，结直肠癌和 卵巢癌以及化学疗法是否具有不同年龄组和肿瘤部位的成本效益。 该提案的具体目的是：1）确定化学疗法的特定阶段功效是否 在对照临床试验中观察到的在延长延长生存的现实世界有效性 65岁或以上的社区住宅患者患有乳房，卵巢癌，肺和大肠癌； 2） 确定化学疗法和进步年龄的有效性之间的关联（即 确定化学疗法是否对所有这些肿瘤患者的年龄段同样有效）； 3） 确定各种化学疗法剂或组合方案的比较有效性 延长生存的好处及其与年龄和肿瘤类型的关联； 4）确定是否以及 患者和肿瘤特征以及医院和医师因素在多大程度上影响 化学疗法和年龄的有效性之间的关系； 5）确定成本 - 逐年和肿瘤类型的化学疗法的有效性。我们的方法是在全国范围内确定 以人群为基础的队列，有超过55万名诊断为乳房，卵巢，肺和 1991年至2005年以上65岁以上的结直肠癌（2006年的癌症病例更多 2007年，来自2011年初的新数据链接），从17种监视，流行病学和结束 结果（SEER）注册机构，占美国人口的25％以上。大量 患者将使我们能够检查许多化学疗法的比较有效性 亚群。这项拟议的研究将检查 化学疗法与现实世界中四个主要肿瘤的年龄相关联 社区环境，以前从未经过测试。提出的创新使用 全国和基于人群的Medicare索赔数据，这些数据提供有关特定的信息 化学疗法药物或方案也将允许进行化学疗法特定的有效性分析。