Orphan Drug Regulation in the European Union
Publication date: January 22, 2026
Prepared by Kamil Żbik, KIELTYKA GLADKOWSKI KG LEGAL
One of the characteristics of diseases and conditions is that different illnesses often affect people to completely different degrees and with completely different frequencies. With the advancement of modern medicine, organizations have begun to take a more serious look at the problem of rare diseases. However, the diagnosis and treatment of such diseases are still highly problematic. One of the problems associated with this is the low revenue from such drugs. Due to the fact that some diseases are very rare, the cost of the process of developing and introducing them to the market is disproportionate to the expected sales of such drugs. This happens because of a very narrow group of people affected by such diseases. Drugs for these rare diseases are called orphan drugs or orphan medicinal products.