Toxicity Monitoring on Phase III Trials with Administrative Data

描述(由申请人提供)：美国国家癌症研究所(NCI)资助的合作肿瘤学小组试验在过去50年中将癌症儿童的总体存活率从10%提高到85%，并为患有恶性肿瘤的成年人设定了护理标准。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。该应用程序建议改进毒性监测，估计与治疗相关的资源利用和成本，并通过合并来自儿童肿瘤学小组(COG)和儿科健康信息系统(PHIS)管理数据库的数据，作为回答重要临床流行病学问题的平台，这些数据库用于最近第三阶段临床试验中接受从头治疗的急性髓细胞白血病(AML)患者。支持这一应用的四个假设：(1)我们假设参加最近和当前COG AML试验的95%以上的患者可以在PHIS中确定；(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合并数据将描述按ARM方案进行的标准化治疗成本，并使成本差异分析成为可能。这一应用具有方法学上的进步和在合作组肿瘤试验中使用这一方法学改进毒性监测和结果分析的意义。此外，该应用程序作为合作组临床试验数据与管理数据的首次合并，具有很高的创新性。最后，在这一应用中所采取的方法不仅适用于其他儿童恶性肿瘤，而且也适用于一般的成人癌症。因此，该应用程序可能会对癌症儿童和成人的护理产生很大影响。

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.