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: 8187687
• 负责人: XIANGLIN DU
• 金额: $ 29.61万
• 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 2015-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8187687
• 关键词: Comparative; Effectiveness; Cost; Cancer; Chemotherapy

DESCRIPTION (provided by applicant): 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 application 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. PUBLIC HEALTH RELEVANCE: The main objectives of this project are to determine the comparative effectiveness and cost effectiveness of various chemotherapy regimens in prolonging survival among community dwelling patients with breast, ovarian, lung and colorectal cancer at age 65 or older; and to determine whether chemotherapy is equally effective for all age groups of patients with these tumors.

描述（由申请人提供）：化疗已被证明对各种恶性肿瘤患者有效延长生存期。从大量临床试验和综合分析中得出的一个重要发现是，化疗对乳腺癌的疗效已被证明随着年龄的增长而下降，50岁以下女性的10年死亡率按比例降低27%，50-59岁女性的死亡率降低14%，60-69岁女性的死亡率降低8%，而70岁及以上女性则没有任何益处。即使在1998年、2001年、2005年和2008年的试验中加入了更多的患者，对合并结果进行检验时，这些发现仍然保持不变。这些结果是惊人的，因为化疗的效果并没有随着年龄的增长而下降，无论是卵巢癌女性患者，还是肺癌和结肠癌的男性和女性患者。大量临床试验表明，化疗对不同年龄段的肺癌、结肠癌患者和卵巢癌患者同样有效，包括70岁以上的人群。因此，来自美国国立卫生研究院（NIH）和其他卫生当局的基于证据的化疗临床指南建议对70岁以下的乳腺癌患者进行化疗，而对结肠癌、卵巢癌和肺癌患者则没有年龄限制的建议。换句话说，强烈建议所有年龄段的结肠癌、卵巢癌和肺癌患者接受化疗。随机对照临床试验被认为是确定治疗效果的黄金标准。然而，临床试验的参与者往往不能代表社区患者的横截面，老年患者在临床试验中的代表性明显不足。因此，基于人群的观察性研究可以作为随机对照试验的有用辅助，以确定在专业中心控制条件下的疗效是否转化为社区的实际疗效，并提供有价值的见解，了解无法从临床试验中获得的治疗在社区实践中的益处。此外，在一些由于伦理或后勤问题而无法进行随机试验的情况下，进行良好的观察性研究可以潜在地用于确定各种药物治疗的相对有效性。因此，迫切需要了解化疗的有效性是否仅随着乳腺癌的年龄而下降（如临床试验所示），或者肺癌、结直肠癌和卵巢癌的有效性是否也随着年龄而下降，以及化疗是否在不同年龄组和肿瘤部位具有成本效益。本应用程序的具体目的是：1)确定在对照临床试验中观察到的化疗的分期特异性疗效是否转化为延长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生存的实际有效性；2)确定化疗有效性与年龄增长之间的关系（即确定化疗是否对所有年龄组的这些肿瘤患者都同样有效）；3)确定各种化疗药物或联合方案特异性获益在延长生存期方面的比较有效性及其与年龄和肿瘤类型的关系；4)确定患者和肿瘤特征以及医院和医师因素是否以及在多大程度上影响化疗效果与年龄的关系；5)根据年龄和肿瘤类型确定化疗的成本-效果。我们的方法是从17个监测、流行病学和最终结果（SEER）登记处（占美国人口的25%以上）中确定一个大型的、全国性的、以人群为基础的队列，其中超过55万名1991年至2005年65岁或以上诊断为乳腺癌、卵巢癌、肺癌和结直肠癌的患者（从2011年初的新数据链接来看，2006年至2007年癌症病例更多）。大量的患者将使我们能够检查化疗在许多亚群中的相对有效性。本研究将在现实世界的社区环境中研究化疗与年龄增长相关的比较和成本效益，这在以前从未进行过测试。提出的创新使用全国和基于人群的医疗保险索赔数据，提供特定化疗药物或方案的信息，也将允许化疗特异性有效性分析。