Therapeutic Area Data Standards for Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC)
Therapeutic Area Data Standards for Autosomal Dominant Polycystic Kidney Disease: A Report From the Polycystic Kidney Disease Outcomes Consortium (PKDOC)
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DOI：
10.1053/j.ajkd.2015.04.044
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发表时间：
2015-10-01
影响因子：
13.2
通讯作者：
Broadbent, Steve
中科院分区：
文献类型：
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作者：
Perrone, Ronald D.;Neville, Jon;Broadbent, Steve
数据标准为一致理解和交换数据提供了一种结构，并使跨研究的数据整合成为可能，以便进行综合分析。目前还没有适用于肾脏疾病的数据标准。我们描述了首个临床数据交换标准联盟(CDISC)由多囊肾病结果联盟(PKDOC)制定的常染色体显性遗传性多囊肾病(ADPKD)数据标准的开发过程。详细描述了公共数据元素的定义和从长期ADPKD登记中使用的病例报告表格创建ADPKD特定数据标准、观察队列(多囊肾病放射成像研究联合会[CRISP]1和2)以及随机临床试验(HALT-PKD)。这一数据标准经过了广泛的审查，包括一段全球公众评议期，现已作为第一个针对PKD的数据标准在线提供(www.cdis.org/Treatform)。到2016年底，所有疾病领域的新电子提交将要求使用标准数据结构和术语的临床试验数据提交。这一数据标准将允许将现有数据映射和汇集成一个共同的数据集，并为ADPKD的未来研究、数据共享和长期登记提供基础。该数据集还将用于支持将肾总体积作为临床试验中使用的预后生物标志物的调节资格。ADPKD的共识数据标准的可用性有可能促进临床试验的启动，并增加观察性研究和已完成临床试验之间的数据共享和汇总，从而提高我们对疾病进展和治疗的理解。(C)2015年，由国家肾脏基金会公司提供。
Data standards provide a structure for consistent understanding and exchange of data and enable the integration of data across studies for integrated analysis. There is no data standard applicable to kidney disease. We describe the process for development of the first-ever Clinical Data Interchange Standards Consortium (CDISC) data standard for autosomal dominant polycystic kidney disease (ADPKD) by the Polycystic Kidney Disease Outcomes Consortium (PKDOC). Definition of common data elements and creation of ADPKD-specific data standards from case report forms used in long-term ADPKD registries, an observational cohort (Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease [CRISP] 1 and 2), and a randomized clinical trial (Halt Progression of Polycystic Kidney Disease [HALT-PKD]) are described in detail. This data standard underwent extensive review, including a global public comment period, and is now available online as the first PKD-specific data standard (www.cdisc.org/therapeutic). Submission of clinical trial data that use standard data structures and terminology will be required for new electronic submissions to the US Food and Drug Administration for all disease areas by the end of 2016. This data standard will allow for the mapping and pooling of available data into a common data set in addition to providing a foundation for future studies, data sharing, and long-term registries in ADPKD. This data set will also be used to support the regulatory qualification of total kidney volume as a prognostic biomarker for use in clinical trials. The availability of consensus data standards for ADPKD has the potential to facilitate clinical trial initiation and increase sharing and aggregation of data across observational studies and among completed clinical trials, thereby improving our understanding of disease progression and treatment. (C) 2015 by the National Kidney Foundation, Inc.