Outcome measures research
The correct choice of outcome measures for a clinical trial is critical to its ability to generate meaningful data that enables the therapy being trialled to move towards regulatory approval. In an activity led by Professor Eugenio Mercuri (Rome), TREAT-NMD is involved in research to define and validate appropriate outcome measures (OMs) for different conditions and is engaging in open dialogue with the regulatory authorities over measures for use in pivotal trials. In related initiatives, promising techniques requiring further development and standardization such as quantitative MRI are being moved forward and standard operating procedures (SOPs) generated.
The TREAT-NMD work in this area is founded on the recognition that it is not possible to find "the" single scale that could be used for all studies and all cohorts, irrespective of disease, age and many other variables, but that the choice of measure should be driven by the trial design. More specifically, the outcome measures should be selected according to the population included in the trial, the functional status of patients at recruitment, the duration of the study, the possible effect of the treatment. This should facilitate the choice of the appropriate measure for that specific cohort and in that specific trial.