Comparative Effectiveness and Cost Effectiveness of Cancer Chemotherapy

项目摘要 已有文献证明化疗能有效地延长卵巢癌患者的生存时间。 各种恶性肿瘤。大量临床试验和综合分析的一个关键发现是， 乳腺癌的化疗效果随着年龄的增长而下降，从27%下降到 50岁以下妇女10年死亡率按比例降低至14% 50-59岁，60-69岁女性8%，70岁或70岁以上女性不享受任何福利。这些 即使在检查了汇总的结果并添加了更多 来自1998年、2001年、2005年和2008年较新试验的患者。这些结果令人震惊，因为 卵巢癌患者的化疗效果并未随年龄增长而降低 癌症，以及患有肺癌和结肠癌的男性和女性。大量的临床试验表明 化疗在不同年龄段的肺癌患者和女性患者中同样有效 结肠癌以及患有卵巢癌的妇女，包括70岁或以上的人口。因此， 美国国立卫生研究院(NIH)和其他机构关于化疗的循证临床指南 卫生当局建议70岁以下乳腺癌患者进行化疗， 然而，对于结肠癌、卵巢癌和肺癌患者，没有年龄限制的建议。在……里面 换句话说，强烈建议所有年龄段的结肠癌、卵巢癌和卵巢癌患者进行化疗。 肺癌。随机对照临床试验被认为是确定 一种疗法的疗效。然而，临床试验的参与者通常并不代表 社区中的患者和老年患者在临床试验中的代表性明显不足。 因此，以人群为基础的观察性研究可以作为随机对照试验的有益补充。 确定专科中心受控条件下的疗效是否转化为现实世界 社区中的有效性，并为社区中的治疗的益处提供有价值的见解 无法从临床试验中获得的实践。此外，在某些情况下， 由于伦理或后勤问题，随机试验是不可能的，进行良好的观察性研究 可以潜在地用于确定各种药物疗法的比较有效性。因此， 迫切需要有关化疗效果是否随年龄增长而降低的信息。 仅适用于乳腺癌(如临床试验所示)，或也随年龄增长而减少，适用于肺癌、结直肠癌和 以及不同年龄段和肿瘤部位的化疗是否具有成本效益。 这项建议的具体目的是：1)确定化疗的分期疗效 在对照临床试验中观察到的在延长患者存活方面的实际效果 65岁或以上的乳腺癌、卵巢癌、肺癌和结直肠癌社区居住患者；2) 确定化疗的有效性与年龄增长之间的关联(即 确定化疗对所有年龄段的这些肿瘤患者是否同样有效)；3) 确定不同化疗药物或联合方案的比较效果 延长生存期的益处及其与年龄和肿瘤类型的关系；4)决定是否和 患者和肿瘤的特征以及医院和医生的因素在多大程度上影响 化疗效果与年龄的关系；5)确定费用-- 年龄和肿瘤类型对化疗效果的影响。我们的方法是在全国范围内找出一个大型的， 超过550,000名被诊断为乳房、卵巢、肺和 1991至2005年65岁或以上的结直肠癌(2006年癌症病例较多- 2007年，来自2011年初的新数据链接)，来自17个监测、流行病学和结束 结果(SEER)登记，占美国人口的25%以上。大量的 患者将使我们能够检查化疗在许多方面的比较效果 亚群。这项拟议的研究将审查以下项目的比较效益和成本效益 在现实世界中，化疗与年龄增长有关的四种主要肿瘤 社区环境，这是以前从未测试过的。拟议的创新使用 全国性和基于人口的联邦医疗保险索赔数据，这些数据提供了关于特定情况的信息 化疗药物或方案，也将允许进行化疗的特定效果分析。

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.