International Approach to SPMS/PPMS


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

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