Abstracts by keywords: regulation
“Rare diseases” are not “rare” at all, but extremely common. After all, around four million people in Germany suffer from a chronic “rare disease” (SE). In Europe, a disease is defined as rare if it affects fewer than 5 in 10,000 people. Of the 30,000 currently known diseases, around 7,000 to 8,000 are classified as “rare”. Despite the small number of patients per disease, the impact of SE can be profound not only for patients and their families but also for society. Rare diseases therefore pose particular challenges for all those involved in the healthcare system. A special case among “rare diseases” are those that are treated with biotherapeutics. These include defects in the immune system, autoimmune disorders or chronic inflammatory diseases of the nervous system. Preparations obtained from plasma are vital for the treatment of these diseases. The uniqueness of the raw material plasma as a starting material for the production of vital medicines must therefore be given special consideration. Immunoglobulins (IG) obtained from human plasma are of particular importance in the treatment of immune defects. IG preparations are highly complex biological medicines that cannot be produced synthetically but are obtained exclusively from voluntary blood/plasma donations. Raising awareness of and improving the diagnosis of immune defects as well as an expansion of immunomodulatory indications are causing the need for plasma to continue to rise. At the same time, however, the number of potential donors is decreasing due to demographic developments and also due to a lack of willingness to donate. This development can lead to bottlenecks in the plasma supply in the long term.