Author
Peter Hübner, as appeared in Alpha1 Journal 1/2024.
On June 5th, an expert panel on the topic of innovations for people with rare diseases took place at the Tagesspiegel in Berlin, to which I had registered as a representative of our patient organization.
Even though the focus was on a rare kidney disease, I was able to draw many parallels to alpha-1 antitrypsin deficiency. It's important that the topic of rare diseases continues to attract public interest. Equally important and interesting is the exchange of ideas after such an event, even if there are no concrete results, because one goal is always achieved: our disease becomes more widely known.
An interesting presentation was the calculation of the socioeconomic burden of disease. This factor indicates how much time is lost to those affected – and also to society – due to treatment times, etc. The infusion and the associated costs immediately came to mind. The socioeconomic burden of disease is an important factor for policymakers and society to pay greater attention to rare diseases, as approximately 4.5 million people in Germany alone are affected by rare diseases.
In my opinion, the topic of self-help groups and patient support, alongside medical care, was somewhat neglected after the often lengthy diagnostic process. I wasn't even able to ask the two members of parliament present my questions on this subject during the Q&A session. However, I will follow up with a written inquiry. MP E. Irlstorfer is currently writing a white paper on rare diseases, which is yet another reason to contact him. A recording of this expert panel discussion can be accessed here.
