Cohen JA et al. for the International Advisory Committee on Clinical Trials in Multiple Sclerosis.Disability outcome measures in multiple sclerosis clinical trials: current status and future prospects.Lancet Neurol. 2012 May;11(5):467-476.Cohen JA et al.
Many of the available disability outcome measures used in clinical trials of multiple sclerosis are insensitive to change over time, inadequately validated, or insensitive to patient-perceived health status or quality of life. Increasing focus on therapies that slow or reverse disability progression makes it essential to refine existing measures or to develop new tools. Major changes to the expanded disability status scale should be avoided to prevent the loss of acceptance by regulators as a measure for primary outcomes in trials that provide substantial evidence of effectiveness. Rather, we recommend practical refinements. Conversely, although substantial data support the multiple sclerosis functional composite as an alternative measure, changes to its component tests and scoring method are needed. Novel approaches, including the use of composite endpoints, patient-reported outcomes, and measurement of biomarkers, show promise as adjuncts to the current disability measures, but are insufficiently validated to serve as substitutes. A collaborative approach that involves academic experts, regulators, industry representitives, and funding agencies is needed to most effectively develop disability outcome measures.
We have been saying for years that the outcome measures to monitor progressive disability need to be changed as the standard EDSS is too insensitive to detect change quickly. Whilst this is important in RRMS trials it is particularly important for studies on progressive MS. This study agrees with our views but rather than tinker with this EDSS scale, which is part of the assessment of efficacy by the regulators by FDA, EMA, it is the view that new outcomes need to be developed there have been international tasks forces set up by the MS Socieites to try and address this aspect. Some have these have already been working for years on this already. Some outcome measures are being added into trials to help validate them. This is yet another function of the MS Socieites that perhaps goes un-noticed, but is an important part of what they do to try and help MSers in the long run