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）：已记录了化学疗法可以有效地延长各种恶性肿瘤患者的生存期。来自众多临床试验和汇总分析的一个关键发现是，化学疗法对乳腺癌的疗效已显示出随着年龄的增长而降低的，这是从50岁以下的10年死亡率降低的27％，到50-59岁的女性，年龄在50-59岁的女性中，对60-69岁的女性不利，对70岁或70岁的女性受益。即使在1998年，2001年，2005年和2008年对更多试验中增加了更多的患者进行检查，这些发现仍然保持恒定。这些结果令人惊讶，因为没有观察到化学疗法的疗效，而卵巢癌女性以及男性和女性肺癌和肺癌和癌症癌症的疗效也没有降低。许多临床试验表明，在不同年龄段的肺癌和结肠癌以及卵巢癌的女性（包括70岁或以上的人群）中，化学疗法同样有效。因此，国家卫生研究院（NIH）和其他卫生当局的化学疗法基于证据的临床指南建议对乳腺癌女性不到70岁的女性进行化学疗法，而对于结肠，卵巢癌和肺癌患者，没有年龄限制的建议。换句话说，强烈建议对所有年龄段，卵巢癌和肺癌的患者进行化学疗法。随机对照临床试验被认为是确定治疗功效的黄金标准。但是，参加临床试验的参与者通常不代表社区中患者的横截面，而在临床试验中，老年患者的代表性显着不足。因此，基于人群的观察性研究可以是对随机对照试验的有用辅助方法，以确定专业中心的受控条件下的功效是否转化为社区中现实世界中的有效性，并为无法从临床试验中获得的社区实践中的治疗益处提供了宝贵的见解。此外，在某些情况下，由于道德或后勤问题，无法进行随机试验，经过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。因此，关于化学疗法的有效性是否仅随着乳腺癌的年龄（如临床试验所示）而降低，或者随着肺，结直肠癌和卵巢癌的年龄而降低，并且化学疗法是否是由不同年龄组和肿瘤部位的成本效益。该应用的具体目的是：1）确定在受控临床试验中观察到的化学疗法的特异性疗效是否转化为现实世界中的有效性，以延长65岁或65岁以上的乳房，卵巢，肺和结直肠癌的社区居住患者的生存率； 2）确定化学疗法的有效性与增长年龄之间的有效性（即确定化学疗法对这些肿瘤患者的所有年龄段同样有效）； 3）确定各种化学疗法药物或联合方案特异性益处在延长生存期以及它们与年龄和肿瘤类型的关联方面的比较效率； 4）确定患者和肿瘤特征以及医院和医师因素是否会影响化学疗法和年龄的有效性之间的关系； 5）确定按年龄和肿瘤类型进行化学疗法的成本效益。我们的方法是在1991年至2005年（2006年至2007年的癌症案例中有更多的癌症病例，2011年初的癌症病例在2011年初，在2011年初的新数据链接中有更多的癌症病例）确定一个全国性的，基于人群的大量，由550,000名诊断为乳腺癌，卵巢，肺和大肠癌的患者，该患者在17个监视，流行病学和最终结果（Seeer）的监管中占25％的责任。大量患者将使我们能够检查化学疗法在许多亚群中的比较有效性。这项拟议的研究将研究化学疗法的比较和成本效益，以及在现实世界社区环境中四个主要肿瘤的年龄增长，这是从未进行过测试的。拟议在全国和基于人群的Medicare索赔数据的创新使用，这些数据提供了有关特定化学疗法药物或方案的信息，也将允许进行化学疗法特定的有效性分析。 公共卫生相关性：该项目的主要目标是确定各种化疗方案在65岁或以上的乳腺癌，卵巢，肺和大肠癌的社区住宅患者之间长期生存中的比较有效性和成本效益；并确定化学疗法是否对所有这些肿瘤患者的年龄组同样有效。