Data Coordinating Center for the Sickle Cell Disease Clinical Research Network

描述(由申请人提供)： 在RFA HL-05-006，镰状细胞疾病临床研究网络中描述的数据协调中心(DCC)的职责。NERI提供以下高度相关的专业知识和经验。(1)拟议的PI、Co-PI、网络经理和协议经理在协调镰状细胞疾病、地中海贫血和其他领域的多地点临床试验和流行病学研究方面均有10年以上的经验。(2)DCC团队包括在镰状细胞疾病方面拥有丰富临床和研究经验的医生，以及与DCC团队合作超过10年的医生。(3)DCC包括一支强大的统计学家团队，他们设计、监测和分析了许多镰状细胞病和其他领域的临床试验和流行病学研究。(4)NERI开发了一个基于Oracle的专有数据管理系统--ADEPT，该系统已成功地用于许多多中心试验，包括国际试验，并支持基于网络的数据输入。(5)NERI还开发了一种计算机化的随机化系统，支持多种随机化方案(简单、排列区块、分层随机化等)。并与数据管理系统完全协调。(6)NERI在建立和运行每个患者的报销系统方面经验丰富，包括支持多个报销时间表。(7)NERI和DCC团队在识别和协调中心实验室、终点裁决小组和其他中央阅读小组(例如MRI和MRA)方面经验丰富。(8)DCC小组包括衡量生活质量和成本效益的专门知识、确定疾病遗传风险因素的统计方面的经验、关于儿童的研究经验以及设计和执行等价性和非劣效性试验的经验。 NERI团队还建议根据先前在临床试验网络中的经验，纳入以下功能。(1)拟在15个临床中心设立病人登记处，以评估病人的特征和每项试验的可用人数是否足够，以便在启动复杂的试验方案之前提供数据收集和管理的即时培训工具，并提供持续的病人资源数据库。(2)将在ADEPT中实施一个基于网络的数据采集和协议管理系统，该系统将实时向卫生与公众服务部的CCS和方案办公室提供自动在线报告，并将充分支持按患者向网站支付费用，并提供自动报告以触发支付。 NERI为DCC组建了一支经验丰富的团队，并提供数据管理系统和其他资源，以成功协调将由镰刀细胞病临床研究网络进行的多项平行试验和其他研究。(摘要结束)

DESCRIPTION (provided by applicant): In addressing the responsibilities of the Data Coordinating Center (DCC) described in the RFA HL-05-006, Sickle Cell Disease Clinical Research Network. NERI offers the following highly relevant expertise and experience. (1) The proposed PI, Co-Pi, Network Manager and Protocol Manager each have more than 10 years of experience in coordinating multi-site clinical trials and epidemiological studies, in sickle cell disease, thalassemia and other areas. (2) The DCC team includes physicians with extensive clinical and research experience in sickle cell disease, physicians with whom the DCC Team has worked for more than 10 years. (3) The DCC includes a strong team of statisticians who have designed, monitored and analyzed numerous clinical trials and epidemiological studies in sickle cell disease and other areas. (4) NERI has developed a proprietary, Oracle-based data management system - ADEPT - that has been used successfully in numerous multi-center trials, including international trials and that supports web-based data entry. (5) NERI has also developed a computerized randomization system that supports many randomization schemes (simple, permuted blocks, stratified randomization, etc.) and which is fully coordinated with the data management system. (6) NERI is experienced at establishing and running per-patient reimbursement systems, including support of multiple schedules for reimbursement. (7) NERI and the DCC team are experienced at identifying and coordinating central laboratories, endpoint adjudication panels and other central reading panels (e.g. MRI and MRA). (8) The DCC team includes expertise in measuring quality of life and cost effectiveness, experience in the statistical aspects of identifying genetic risk factors for disease, experience in research on children and experience in designing and executing equivalence and noninferiority trials. The NERI team also proposes to incorporate the following features, based on prior experience in clinical trials networks. (1) A registry of patients at the 15 Clinical Centers is proposed to assess the characteristics of the patients and the adequacy of the numbers available for each trial, to provide an immediate training vehicle for data acquisition and management before initiating a complex trial protocol, and to provide an ongoing patient resource database. (2) A web-based data acquisition and protocol management system will be implemented in ADEPT that will provide automated on-line reports, in real time, to the CCs and Program Office at NHLBI, and that will fully support per-patient payments to the sites with automated reports for triggering the payments. NERI has assembled a highly and appropriately experienced team for the DCC and offers the data management system and other resources needed to successfully coordinate the multiple parallel trials and other studies that will be undertaken by the Sickle Cell Disease Clinical Research Network. (End of Abstract)