In the world of mitochondrial medicine many topics are being discussed and many issues are to be resolved. If these are purely medical, it is obvious that they must be dealt with by the medical (and other) specialists. Yet, there are also issues which should be dealt with in a broader circle, with patient representatives, other scientists, regulatory authorities (EMA, FDA) or with representatives from pharmaceutical industry.
Therefore, IMP has taken the initiative to organise and (partly) fund international workshops to deal with different issues. Main goal is to have all relevant stakeholders together so that different subjects can be discussed, consensus can be achieved, and the path to a therapy/cure will be smoother.
Last year in November in Rome the first workshop took place. This was done with the great support of a select group of academic researchers. Key opinion leaders from the academic world, all involved in mitochondrial disease, and representatives from different patient advocacy groups met. The aim was to achieve consensus on a list of outcome measures for trials on primary mitochondrial myopathy. Consensus on this is essential for the process of having trials and drug development approved by FDA and EMA. This would speed up the whole process. And after a couple of days of hard work and additional work by four KOL'S a consensus list was achieved. The list will serve as guidance for researchers organising trials for primary mitochondrial myopathy.
The academic researchers are now planning another comparable workshop, focussing on another type of mitochondrial disease.
A second workshop took place in March this year in Amsterdam. This time also representatives from (7) pharmaceutical companies participated. The aim was to share knowledge and experiences with organising research studies and trials and with the FDA and EMA. All participants were open in sharing and eager to learn from each other. It is unique that within a disease community all stakeholders meet and discussions take place in such an open atmosphere. The added value of such workshops became clear and the need for more was expressed.
As it is in the interest of mitochondrial patients that such meetings take place with the aims of learning from each other, having in-depth discussions, achieving consensus, and as such speeding up the path to a cure. Therefore, IMP will continue with its efforts to organise these.
