TY - JOUR
T1 - Therapeutic area data standards for autosomal dominant polycystic kidney disease
T2 - A report from the Polycystic Kidney Disease Outcomes Consortium (PKDOC)
AU - Perrone, Ronald D.
AU - Neville, Jon
AU - Chapman, Arlene B.
AU - Gitomer, Berenice Y.
AU - Miskulin, Dana C.
AU - Torres, Vicente E.
AU - Czerwiec, Frank S.
AU - Dennis, Eslie
AU - Kisler, Bron
AU - Kopko, Steve
AU - Krasa, Holly B.
AU - Leroy, Elizabeth
AU - Castedo, Juliana
AU - Schrier, Robert W.
AU - Broadbent, Steve
N1 - Publisher Copyright:
© 2015 National Kidney Foundation, Inc.
PY - 2015/10/1
Y1 - 2015/10/1
N2 - 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.
AB - 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.
KW - Autosomal dominant polycystic kidney disease (ADPKD)
KW - Clinical Data Interchange Standards Consortium (CDISC)
KW - Polycystic Kidney Disease Outcomes Consortium (PKDOC)
KW - consensus data standards
KW - controlled terminology
KW - data pooling
KW - disease progression biomarker
KW - standard data structure
KW - total kidney volume (TKV)
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U2 - 10.1053/j.ajkd.2015.04.044
DO - 10.1053/j.ajkd.2015.04.044
M3 - Article
C2 - 26088508
AN - SCOPUS:84942294959
SN - 0272-6386
VL - 66
SP - 583
EP - 590
JO - American Journal of Kidney Diseases
JF - American Journal of Kidney Diseases
IS - 4
ER -