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: 8447342
• 负责人: XIANGLIN DU
• 金额: $ 29.03万
• 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/8447342
• 关键词: 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 已有证据表明，化疗可以有效延长患者的生存期 各种恶性肿瘤大量临床试验和汇总分析的一个关键发现是， 乳腺癌化疗的疗效已被证明随着年龄的增长而下降，从27% 将50岁以下妇女的10年死亡率按比例降低到14% 50-59岁的妇女和60-69岁的妇女享受8%的福利，70岁或以上的妇女没有福利。这些 即使在对合并结果进行检查时， 在1998年、2001年、2005年和2008年的近期试验中，这些结果是惊人的，因为 对于卵巢癌患者，随着年龄的增长，化疗的疗效并没有降低。 癌症，以及男性和女性肺癌和结肠癌。大量临床试验表明 化疗在不同年龄组的肺癌患者中同样有效， 结肠癌以及卵巢癌妇女，包括70岁或以上的人群。因此，我们认为， 美国国立卫生研究院（NIH）和其他 卫生当局建议对70岁以下的乳腺癌妇女进行化疗， 而对于结肠癌、卵巢癌和肺癌患者，没有年龄限制的建议。在 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 治疗的效果然而，临床试验的参与者往往不能代表 社区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用补充， 确定专科中心受控条件下的疗效是否转化为现实世界 在社区中的有效性，并提供有价值的洞察社区治疗的好处 不能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，无法进行随机试验，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，我们认为， 迫切需要了解化疗的有效性是否会随着年龄的增长而降低 仅用于乳腺癌（如临床试验所示），或随着年龄的增长，肺癌，结肠直肠癌， 卵巢癌和化疗是否符合成本效益的不同年龄组和肿瘤部位。 该建议的具体目的是：1）确定化疗的阶段特异性疗效 在对照临床试验中观察到的有效性被转化为现实世界中延长生存期的有效性， 65岁或以上的乳腺癌、卵巢癌、肺癌和结直肠癌社区居民患者; 2） 确定化疗的有效性和年龄增长之间的关联（即，到 确定化疗是否对所有年龄组的这些肿瘤患者同样有效）; 3） 确定各种化疗药物或联合方案的比较有效性 延长生存期的益处及其与年龄和肿瘤类型的相关性; 4）确定是否和 患者和肿瘤特征以及医院和医生因素在多大程度上影响 化疗的有效性与年龄的关系; 5）确定成本- 根据年龄和肿瘤类型的化疗效果。我们的方法是在全国范围内， 超过550，000名被诊断患有乳腺癌、卵巢癌、肺癌和乳腺癌的患者的基于人群的队列研究。 1991年至2005年65岁或以上的结直肠癌（2006年癌症病例更多， 2007年从2011年初的新数据链接）从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。的大量 患者将使我们能够检查化疗的比较有效性， 亚群这项拟议的研究将审查以下方面的比较效益和成本效益： 化疗与真实的世界中四种主要肿瘤的年龄增长相关 社区设置，这是以前从未测试过的。拟议的创新使用 全国范围内和基于人口的医疗保险索赔数据，提供有关特定 化疗药物或方案的有效性分析还将允许进行化疗特异性有效性分析。