On the 21st of June, Diana Ribeiro, Head of Research, Action Duchenne was involved in a meeting hosted by TREAT-NMD and co-sponsored by various MD organisations entitled Ways to Measure Clinical Effectiveness in the Investigation of Medicinal Products for BMD/DMD. This was a document drafted by the European Medicines Agency earlier this year (link below) and has gone out to consultation to all. They have invited further evidence-based comments and feedback in a coherent and structured manner by the 31st of August.
The meeting led by the experts and family members pointed out areas they felt had not been clear with regards to current progress in assessing DMD clinical trials and based on current knowledge can be addressed further. A few representatives from the regulatory agencies were present and shared their personal views. Much of the discussion involved the robustness and validity of current and future outcomes for studies.
There is more evidence to emerge for clinical trial criteria, which is currently unpublished to address some of the main limitations as stipulated by the respective agencies and regulators in the draft guideline document. These need to be assimilated by the experts into a formal response and the information from this meeting and consultation will be shared with all as soon as these become available.
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