Substantial progress was reviewed at the second annual meeting of the National MS Society-supported MS Outcome Assessments Consortium at the U.S. Food and Drug Administration (FDA). The MSOAC is a global effort aimed at developing a new tool that will address the critical need for a more sensitive way to detect the benefit of potential treatments that slow or reverse progressive disability in people with MS. Participants included representatives from 23 academic institutions, 9 pharmaceutical companies, the FDA, the European Medicines Agency (EMA), the Consortium of MS Centers, and several MS societies.
Over the 4-year, $3 million project, MSOAC is collecting data from many MS clinical trials, which will then be standardized and analyzed to identify the optimal measures for inclusion in this tool. This will then be submitted to the FDA and EMA for regulatory approval, which would validate its acceptance for use as a primary outcome measure in clinical trials. The participation from FDA and EMA staff at the development stages of this process should help ensure that the tool meets the agencies’ requirements.
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. The task force considered whether existing measures such as the MSFC (Multiple Sclerosis Functional Composite) could be enhanced or if one or more new measures would be needed. The task force determined that a key aspect of the project would be the need to analyze data from completed clinical trials that have used parts of the MSFC as well as other measures of disability.
After considering several options for implementing the goals of the project, it became clear that collaborating with the Critical Path Institute (C-Path) would be of great value. 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.
Members reported on progress in obtaining the clinical trial data from industry and academic studies that used elements of the MSFC and other disability measures such as “low contrast visual acuity” (a highly sensitive way to evaluate visual problems). To date the team has amassed data from 5,000 people with MS who had been enrolled in trials, with an additional 11,000 in process. Ideally, the existing data may be sufficient to achieve the goals of the project, but some additional research may also be needed.
An early achievement of the project has been the development of new MS data standards – called CDISC standards. This standard permits the harmonization of data collected across different platforms or databases, and may be used by the FDA to provide guidelines for future clinical trials. CDISC is a global effort to improve medical research, and an important bonus of developing MS CDISC data standards is that they will help speed the evaluation of results from future MS clinical trials.
The Consortium is continuing the process of collecting data from completed clinical trials. Progress will continue toward compiling this data and analyzing it to evaluate potential components of a new outcome measurement tool. Regular teleconferences are helping to keep this project moving. Ultimately, the proposed tool will be submitted to regulatory authorities for qualification as a primary outcome measure in MS trials. Upon qualification, the tool will be freely available, and details describing how to use the tool will be published.
Read more about MSOAC
Read details of the first annual MSOAC meeting