As the World Orphan Drug Congress is only a few weeks around the corner on 12-14 November, and our judging panel is now making their decisions about the winners of the ROAR Awards, I felt inclined to reflect on how the year is shaping up so far (does the photo make sense now?) .
Well, this week sees the launch of the EURORDIS campaign to make 2019 the European Year for Rare Diseases, as that year will mark 20 years since the adoption of the EU Regulation on Orphan Medicinal Products and the 10 year anniversary of the Commission Communication and Council Recommendation on rare diseases. Some great things have been achieved in this time and using 2019 in this way would be fantastic to engage the public, politicians and industry alike in getting more involved and working harder on treatments for rare and ultra-rare conditions. I expect that Yan Le Camm will mention this in his session on day one of the congress.
Of course, 2014 has been the year of the ALS Ice Bucket challenge, which has seen a great deal of money raised for that particular cause and lots of awareness raised around rare conditions, which is great. We have a representative from the Belgian branch of the ALS association speaking on their experience around this which will be really interesting.
Then I turned to orphan designations of this year so far, and there have been 80 so far covering 60 different conditions. The highest numbers through the door so far this year go to Cystic Fibrosis (5) and glioma (4). Let’s hope we see many more throughout the rest of the year and beyond!
So, that’s just some of the things that 2014 has brought us so far!
I can’t wait for the World Orphan Drug Congress in just a few weeks, which promises to be a great place to find out where people are now and what their plans are in the future to get the right drugs, to the right people in the best time. Click here to see the details.