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Mult Scler. 2018 Oct;24(11):1469-1484. doi: 10.1177/1352458517723718. Epub 2017 Aug 11.

The MSOAC approach to developing performance outcomes to measure and monitor multiple sclerosis disability.

Author information

1
National Multiple Sclerosis Society, New York, NY, USA.
2
Critical Path Institute, Tucson, AZ, USA.
3
Biogen, Cambridge, MA, USA.
4
University of Washington, Seattle, WA, USA.
5
NYU Langone Medical Center, New York, NY, USA.
6
University at Buffalo, Buffalo, NY, USA.
7
Cleveland Clinic, Cleveland, OH, USA.
8
Kessler Foundation, West Orange, NJ, USA.
9
Genentech, San Francisco, CA, USA.
10
UAB School of Public Health, Birmingham, AL, USA.
11
Teva Pharmaceuticals, Green Valley, PA, USA.
12
Faculty of Medicine and Life Sciences, Hasselt University, Hasselt, Belgium.
13
Novartis Pharmaceuticals, East Hanover, NJ, USA.
14
University of Virginia, Charlottesville, VA, USA.
15
University College London, London, UK.
16
Icahn School of Medicine at Mount Sinai, New York, NY, USA.
17
Imperial College London, London, UK.
18
McGill University, Montreal, QC, Canada.
19
University of Alabama at Birmingham, Birmingham, AL, USA.
20
Johns Hopkins University, Baltimore, MD, USA.
21
Washington University in St. Louis, St. Louis, MO, USA.
22
Sanofi Genzyme, Cambridge, MA, USA.
23
Acorda Therapeutics, Inc., Ardsley, NY, USA; KES Business Consulting LLC, Lyme, CT, USA.
24
VU University Medical Center, Amsterdam, The Netherlands.

Abstract

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.

KEYWORDS:

MS disability; clinical trial database; data standards; performance outcome measures; regulatory qualification

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