– Progress toward new tool to speed pace of MS clinical trials
UPDATE - Originally released March 22, 2017. A fifth paper has now been published
- Five papers have been published that review research and methods behind four clinical outcome measures that detect changes that occur in walking, vision, cognition and manual dexterity experienced by people with MS.
- Together, the four measures provide snapshots of how an individual’s physical and mental functions may remain stable or change over time, and their use may improve the ability to detect benefits of therapies in clinical trials.
- The papers are part of ongoing efforts of the National MS Society-supported international MS Outcome Assessments Consortium (MSOAC) to speed the pace of MS clinical trials by developing a new tool set that can detect the benefit of potential treatments that slow or reverse progressive disability in people with MS.
- The five papers have been published in Multiple Sclerosis Journal, and their full content may be read by anyone. (Links provided below.)
The international MS Outcome Assessments Consortium
(MSOAC) was launched after a National MS Society-supported task force met in late 2011 to determine how to address the need for better measures of MS-related disability in clinical trials. It became clear that collaborating with the Critical Path Institute
(C-Path) would create a clearer path forward. C-Path is a nonprofit partnership with the FDA, whose aim is to accelerate the pace and reduce the costs of medical product development by creating new standards for evaluating the safety and effectiveness of therapies.
Participants in MSOAC include representatives from academic institutions, pharmaceutical companies, the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the National Institutes of Health. Over the 4-year, $3 million project, MSOAC has been conducting literature review and perspectives of people with MS, along with data from clinical trials, to evaluate measures that could be used to detect and evaluate the disability status of individuals with MS in terms of walking, vision, cognition and manual dexterity. Another key activity has been to collect data from 16 MS clinical trials involving 14,370 trial participants, then standardize and analyze the data to identify the optimal measures for inclusion in the outcome measures tool.
An early achievement of the project was the development of new MS data standards – called CDISC standards
. This permits the harmonization of data collected across different studies, and will be required by the FDA for future clinical trials.
Another milestone was reached in April 2016
, when MSOAC opened up to qualified investigators access to a subset of the data containing nearly 2500 (de-identified) patient records from the placebo arms of nine MS clinical trials to help answer research questions.
The recent publication of the journal papers is another important milestone in this effort.
The papers review published evidence for the validity of the four measures and the extent to which they can detect changes that are “clinically meaningful” (reflecting functions that may impact an individual’s activities of daily living). Four papers and an accompanying editorial were published in Multiple Sclerosis Journal early online on February 16, 2017. A fifth paper that chronicles the methods used by MSOAC was published early online on August 11, 2017. Each of these papers is openly available to all readers:
- Editorial: The measure tells the tale: Clinical outcome measures in multiple sclerosis Hanneke E Hulst, Alan J Thompson, Jeroen JG Geurts
- Validity of the Symbol Digit Modalities Test as a cognition performance outcome measure for multiple sclerosis Ralph HB Benedict, John DeLuca, Glenn Phillips, and others
- Validity of low-contrast letter acuity as a visual performance outcome measure for multiple sclerosis Laura J Balcer, Jenelle Raynowska, Rachel Nolan, and others
- Validity of the timed 25-foot walk as an ambulatory performance outcome measure for multiple sclerosis Robert W Motl, Jeffrey A Cohen, Ralph Benedict, and others
- The Nine-Hole Peg Test as a manual dexterity performance measure for multiple sclerosis Peter Feys, Ilse Lamers, Gordon Francis, and others
- NEW: The MSOAC approach to developing performance outcomes to measure and monitor multiple sclerosis disability Nicholas G LaRocca, Lynn D Hudson, Richard Rudick, and others
In the coming months, the MSOAC is finalizing the application that includes the clinical trials data and proposed tool, and will submit the information to the FDA and EMA for regulatory approval. Upon qualification of the tool set for use as a primary outcome measure in clinical trials, it will be made freely available, and details describing how to use the tool set will be published.
Read more about the MSOAC