Toxicity Monitoring on Phase III Trials with Administrative Data

美国国家癌症研究所（NCI）资助的合作肿瘤组试验在过去的五十年中将癌症儿童的总生存率从10%提高到85%，并为恶性肿瘤成人患者制定了护理标准。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”。如果不能提高其效率和有效性，癌症新疗法的引入将被推迟，患者的生命将不必要地丧失。本申请旨在改善毒性监测，估计治疗相关的资源利用和成本，并作为一个平台，通过合并来自儿童肿瘤组（COG）和儿科健康信息系统（PHIS）管理数据库的数据来回答重要的临床流行病学问题，用于近期III期临床试验中治疗新发急性髓性白血病（AML）的患者。四个假设支持该应用：（1）我们假设在最近和当前的COG AML试验中招募的患者中，超过95%可以在PHIS中识别;（2）COG-PHIS合并数据将比未合并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合并数据将按方案组描述标准化治疗成本，并能够分析成本变化。该应用程序具有重要意义，无论是作为一种方法的进步，并在使用这种方法，以改善毒性监测和结果分析的合作组肿瘤学试验。此外，该应用程序具有高度创新性，首次将合作格鲁临床试验数据与管理数据合并。最后，本申请中采用的方法不仅适用于其他儿科恶性肿瘤，而且一般适用于成人癌症。因此，该应用可能对患有癌症的儿童和成人的护理产生很大影响。 公共卫生相关性：本申请旨在改善毒性监测，估计治疗相关的资源利用和成本，并作为一个平台，通过合并来自儿童肿瘤组（COG）和儿科健康信息系统（PHIS）管理数据库的数据来回答重要的临床流行病学问题，用于近期III期临床试验中治疗新发急性髓性白血病（AML）的患者。该应用程序具有重要意义，无论是作为一种方法的进步，并在使用这种方法，以改善毒性监测和结果分析的合作组肿瘤学试验。此外，本申请中采用的方法不仅应适用于其他儿科恶性肿瘤，还应适用于一般成人癌症，因此具有广泛的公共卫生影响。

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.