Toxicity Monitoring on Phase III Trials with Administrative Data

描述（由申请人提供）：在过去的50年里，美国国家癌症研究所（NCI）资助的合作肿瘤学组试验将儿童癌症患者的总生存率从10%提高到85%，并为成人恶性肿瘤患者制定了治疗标准。然而，根据医学研究所的说法：“临床试验系统正在接近危机状态”。如果[它]不提高其效率和效果，癌症新疗法的引入将被推迟，患者的生命将不必要地丧失。该应用程序旨在通过合并儿童肿瘤学组（COG）和儿科健康信息系统（PHIS）管理数据库的数据，改善毒性监测，估计治疗相关的资源利用和成本，并作为回答重要临床流行病学问题的平台，这些数据来自最近III期临床试验中接受新生急性髓性白血病（AML）治疗的患者。这一应用的基础是四个假设：(1)我们假设超过95%参加最近和目前的COG AML试验的患者可以在公共卫生信息系统中被识别出来；(2) COG-PHIS合并后的数据比未合并的数据更准确地报告不良事件；(3) COG-PHIS合并数据可以前瞻性地监测III期试验的不良事件；(4) COG-PHIS合并后的数据将描述按方案组的标准化治疗成本，并能够分析成本变化。这一应用具有重要的意义，既可以作为一种方法学的进步，也可以在合作组肿瘤试验中使用这种方法学来改进毒性监测和结果分析。此外，该应用程序是高度创新的，因为它首次将合作小组临床试验数据与管理数据合并。最后，在本应用中采用的方法不仅适用于其他儿科恶性肿瘤，也适用于一般的成人癌症。因此，该应用程序很可能对患有癌症的儿童和成人的护理产生重大影响。

DESCRIPTION (provided by applicant): National Cancer Institute (NCI) funded cooperative oncology group trials have improved overall survival for children with cancer from 10% to 85% over the past five decades, and have set standards of care for adults with malignancies. However, according to the Institute of Medicine: "the clinical trial system is approaching a state of crisis". If [it] does not improve its efficiency and effectiveness, the introduction of new treatments for cancer will be delayed and patient lives will be lost unnecessarily. This application proposes to improve toxicity monitoring, estimate treatment associated resource utilization and costs, and serve as a platform for answering important clinical epidemiology questions by merging data from the Children's Oncology Group (COG) and the Pediatric Health Information Systems (PHIS) administrative data base for patients treated for de novo acute myeloid leukemia (AML) on recent Phase III clinical trials. Four hypotheses underpin this application: (1) We hypothesize that greater than 95% of patients enrolled on recent and current COG AML trials can be identified in PHIS; (2) COG-PHIS merged data will more accurately report adverse events than unmerged data; (3) COG-PHIS merged data can prospectively monitor a Phase III trial for adverse events; (4) COG-PHIS merged data will describe standardized costs of treatment by regimen arm and enable analyses of cost variation. This application has significance both as a methodological advancement and in the use of this methodology to improve toxicity monitoring and outcome analysis in cooperative group oncology trials. Furthermore, this application is highly innovative as the first merging of cooperative grou clinical trial data with administrative data. Finally, the approach taken in this application shoul be applicable not only to other pediatric malignancies, but also to adult cancers generally. Thus, the application is likely to have a high impact on the care of both children and adults with cancer. PUBLIC HEALTH RELEVANCE: This application proposes to improve toxicity monitoring, estimate treatment associated resource utilization and costs, and serve as a platform for answering important clinical epidemiology questions by merging data from the Children's Oncology Group (COG) and the Pediatric Health Information Systems (PHIS) administrative data base for patients treated for de novo acute myeloid leukemia (AML) on recent Phase III clinical trials. This application has significance both as a methodological advancement and in the use of this methodology to improve toxicity monitoring and outcome analysis in cooperative group oncology trials. Furthermore, the approach taken in this application should be applicable not only to other pediatric malignancies, but also to adult cancers generally, and thus have broad public health impact.