TY - JOUR
T1 - The MSOAC approach to developing performance outcomes to measure and monitor multiple sclerosis disability
AU - for the Multiple Sclerosis Outcome Assessments Consortium (MSOAC)
AU - LaRocca, Nicholas G.
AU - Hudson, Lynn D.
AU - Rudick, Richard
AU - Amtmann, Dagmar
AU - Balcer, Laura
AU - Benedict, Ralph
AU - Bermel, Robert
AU - Chang, Ih
AU - Chiaravalloti, Nancy D.
AU - Chin, Peter
AU - Cohen, Jeffrey A.
AU - Cutter, Gary R.
AU - Davis, Mat D.
AU - DeLuca, John
AU - Feys, Peter
AU - Francis, Gordon
AU - Goldman, Myla D.
AU - Hartley, Emily
AU - Kapoor, Raj
AU - Lublin, Fred
AU - Lundstrom, Gary
AU - Matthews, Paul M.
AU - Mayo, Nancy
AU - Meibach, Richard
AU - Miller, Deborah M.
AU - Motl, Robert W.
AU - Mowry, Ellen M.
AU - Naismith, Rob
AU - Neville, Jon
AU - Panagoulias, Jennifer
AU - Panzara, Michael
AU - Phillips, Glenn
AU - Robbins, Ann
AU - Sidovar, Matthew F.
AU - Smith, Kathryn E.
AU - Sperling, Bjorn
AU - Uitdehaag, Bernard M.J.
AU - Weaver, Jerry
N1 - Publisher Copyright:
© The Author(s), 2017.
PY - 2018/10/1
Y1 - 2018/10/1
N2 - Background: The Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was formed by the National MS Society to develop improved measures of multiple sclerosis (MS)-related disability. Objectives: (1) To assess the current literature and available data on functional performance outcome measures (PerfOs) and (2) to determine suitability of using PerfOs to quantify MS disability in MS clinical trials. Methods: (1) Identify disability dimensions common in MS; (2) conduct a comprehensive literature review of measures for those dimensions; (3) develop an MS Clinical Data Interchange Standards Consortium (CDISC) data standard; (4) create a database of standardized, pooled clinical trial data; (5) analyze the pooled data to assess psychometric properties of candidate measures; and (6) work with regulatory agencies to use the measures as primary or secondary outcomes in MS clinical trials. Conclusion: Considerable data exist supporting measures of the functional domains ambulation, manual dexterity, vision, and cognition. A CDISC standard for MS (http://www.cdisc.org/therapeutic#MS) was published, allowing pooling of clinical trial data. MSOAC member organizations contributed clinical data from 16 trials, including 14,370 subjects. Data from placebo-arm subjects are available to qualified researchers. This integrated, standardized dataset is being analyzed to support qualification of disability endpoints by regulatory agencies.
AB - Background: The Multiple Sclerosis Outcome Assessments Consortium (MSOAC) was formed by the National MS Society to develop improved measures of multiple sclerosis (MS)-related disability. Objectives: (1) To assess the current literature and available data on functional performance outcome measures (PerfOs) and (2) to determine suitability of using PerfOs to quantify MS disability in MS clinical trials. Methods: (1) Identify disability dimensions common in MS; (2) conduct a comprehensive literature review of measures for those dimensions; (3) develop an MS Clinical Data Interchange Standards Consortium (CDISC) data standard; (4) create a database of standardized, pooled clinical trial data; (5) analyze the pooled data to assess psychometric properties of candidate measures; and (6) work with regulatory agencies to use the measures as primary or secondary outcomes in MS clinical trials. Conclusion: Considerable data exist supporting measures of the functional domains ambulation, manual dexterity, vision, and cognition. A CDISC standard for MS (http://www.cdisc.org/therapeutic#MS) was published, allowing pooling of clinical trial data. MSOAC member organizations contributed clinical data from 16 trials, including 14,370 subjects. Data from placebo-arm subjects are available to qualified researchers. This integrated, standardized dataset is being analyzed to support qualification of disability endpoints by regulatory agencies.
KW - MS disability
KW - clinical trial database
KW - data standards
KW - performance outcome measures
KW - regulatory qualification
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U2 - 10.1177/1352458517723718
DO - 10.1177/1352458517723718
M3 - Article
C2 - 28799444
AN - SCOPUS:85042465617
SN - 1352-4585
VL - 24
SP - 1469
EP - 1484
JO - Multiple Sclerosis Journal
JF - Multiple Sclerosis Journal
IS - 11
ER -