Use of Geographic Variations to Estimate Effectiveness with Non-Randomized Data

描述（由申请人提供）：在美国，不同地理区域的医疗支出和服务使用存在巨大差异。这种变化的很大一部分与使用尚未在随机试验中研究的服务有关。这种服务的巨大差异导致人们呼吁使用观察数据进行研究，以衡量医疗的相对有效性。由于患者组之间可能存在未观察到的差异，因此在非随机化环境中估计治疗有效性需要仔细收集和分析数据。越来越多的研究人员正在利用服务使用的地理差异来推断其对个体患者的有效性，假设患者特征在地理区域之间是平衡的。然而，很少有人了解的设置中，生态变化可以用来获得有效的估计治疗效果的非随机研究。使用监测流行病学和最终结果（SEER）-医疗保险数据库中诊断为结直肠癌和前列腺癌的老年患者群体的数据，我们将开发，评估和应用先进的统计方法来估计癌症干预措施的有效性。癌症为这种分析提供了一个理想的环境，因为它是一种普遍和严重的健康状况，并且正在不断开发和推广新的和昂贵的治疗方法。这些目标将通过以下三个具体目标来实现：（1）开发和评价统计方法，以使用地理居住地来模拟治疗随机化来估计治疗有效性;（2）应用这些方法来估计诊断为结直肠癌和前列腺癌的患者的干预措施的比较有效性;以及（3）开发和传播软件，以促进在比较有效性研究中使用适当的统计方法。这些结果将提供有关干预措施有效性的信息，这些干预措施在最近的医学研究所的一项研究中得到了强调，这是比较有效性研究的优先事项，也提供了一种方法，其他研究人员可以用来了解其他疾病的治疗效果。 公共卫生相关性：该项目将提供有关结肠直肠癌和前列腺癌干预措施的宝贵信息，这些干预措施在最近的医学研究所关于比较有效性的优先事项的研究中得到了强调。此外，开发的方法将适用于癌症和其他疾病的广泛干预措施，为未来的比较有效性研究和最终改变治疗指南提供框架。

DESCRIPTION (provided by applicant): There is enormous variation in medical spending and use of services across geographic areas in the United States. A large portion of this variation has been linked to use of services that have not been studied in randomized trials. This wide variation in services of unknown benefit has led to calls for research using observational data to measure the comparative effectiveness of medical treatments. Estimating treatment effectiveness in non-randomized settings requires careful data collection and analysis because of possible unobserved differences between patient groups. Increasingly, researchers are exploiting geographic variation in use of services to infer their effectiveness in individual patients, under the assumption that patient characteristics are balanced across geographic areas. However, little is understood about settings in which ecological variation can be used to obtain valid estimates of treatment effects in non-randomized studies. Using data from population-based cohorts of elderly patients diagnosed with colorectal and prostate cancer in the Surveillance Epidemiology, and End Results (SEER)-Medicare database, we will develop, evaluate, and apply advanced statistical methods to estimate the effectiveness of cancer interventions. Cancer provides an ideal setting for such an analysis because it is a prevalent and serious health condition, and new and expensive treatments of unknown benefit are continuously being developed and diffused. These goals will be accomplished through the following three specific aims: (1) develop and evaluate statistical methods to estimate treatment effectiveness using geographic residence to mimic randomization to treatment; (2) apply these methods to estimate the comparative effectiveness of interventions for patients diagnosed with colorectal and prostate cancer; and (3) develop and disseminate software to promote the use of appropriate statistical methods in comparative effectiveness studies These results will provide information about the effectiveness of interventions highlighted in a recent Institute of Medicine study on priorities for comparative effectiveness research and also provide a methodology that other researchers can use to learn about the effectiveness of treatments in other diseases. PUBLIC HEALTH RELEVANCE: This project will provide valuable information on interventions in colorectal and prostate cancer highlighted in a recent Institute of Medicine study on priorities for comparative effectiveness. In addition, the methods developed will be applicable to a broad range of interventions for cancer and other diseases, providing a framework for future comparative effectiveness studies and ultimately changes in treatment guidelines.