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）：化疗已被证明可有效延长患有各种恶性肿瘤的患者的生存期。大量临床试验和汇总分析的一项重要发现是，化疗对乳腺癌的疗效已被证明随着年龄的增长而降低，从 50 岁以下女性的 10 年死亡率按比例降低 27%，到 50-59 岁女性的 14% 和 60-69 岁女性的 8%，而对于 70 岁或以上的女性没有任何益处。即使在 1998 年、2001 年、2005 年和 2008 年的最新试验中纳入了更多患者，对汇总结果进行检查后，这些结果仍然保持不变。这些结果令人震惊，因为对于患有卵巢癌的女性以及患有肺癌和结肠癌的男性和女性来说，随着年龄的增长，化疗的疗效并未观察到下降。大量临床试验表明，化疗对于不同年龄组的肺癌和结肠癌男性和女性以及卵巢癌女性（包括 70 岁或以上人群）同样有效。因此，美国国立卫生研究院（NIH）和其他卫生当局的循证化疗临床指南建议70岁以下的乳腺癌女性接受化疗，而对于结肠癌、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金标准。然而，临床试验的参与者通常不能代表社区中的患者群体，而且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手段，确定专科中心受控条件下的疗效是否转化为社区中的真实有效性，并提供无法从临床试验中获得的治疗在社区实践中的益处的宝贵见解。此外，在某些情况下，由于伦理或后勤问题而无法进行随机试验，进行良好的观察性研究可能有助于确定各种药物疗法的比较有效性。因此，迫切需要了解以下信息：化疗的有效性是否仅随着乳腺癌的年龄而降低（如临床试验所示），或者对于肺癌、结直肠癌和卵巢癌也随着年龄而降低，以及化疗对于不同年龄组和肿瘤部位是否具有成本效益。本申请的具体目的是： 1) 确定对照临床试验中观察到的化疗的阶段特异性疗效是否转化为延长 65 岁或以上社区居民乳腺癌、卵巢癌、肺癌和结直肠癌患者生存期的现实有效性； 2) 确定化疗有效性与年龄增长之间的关联（即确定化疗对于所有年龄组的肿瘤患者是否同样有效）； 3) 确定各种化疗药物或联合治疗方案在延长生存期方面的具体益处的比较效果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生因素是否以及在何种程度上影响化疗效果与年龄之间的关系； 5) 根据年龄和肿瘤类型确定化疗的成本效益。我们的方法是从 17 个监测、流行病学和最终结果 (SEER) 登记处确定 1991 年至 2005 年间超过 550,000 名年龄在 65 岁或以上被诊断患有乳腺癌、卵巢癌、肺癌和结直肠癌的患者（根据 2011 年初的新数据链接，2006 年至 2007 年癌症病例更多），确定一个全国性的、以人群为基础的大型队列，占占美国人口的 25%。大量患者将使我们能够检查化疗在许多亚群中的相对有效性。这项拟议的研究将在现实世界的社区环境中检查化疗与四种主要肿瘤年龄增长相关的比较和成本效益，这以前从未进行过测试。拟议的全国性和基于人口的医疗保险索赔数据的创新使用，提供了有关特定化疗药物或方案的信息，也将允许进行特定于化疗的有效性分析。 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延长 65 岁或以上社区居民乳腺癌、卵巢癌、肺癌和结直肠癌患者生存期方面的相对有效性和成本效益；并确定化疗对于所有年龄组的这些肿瘤患者是否同样有效。