.

Currently, there are about 7000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% all diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process repurposing drugs for new indications, compared with development novel orphan drugs, is a time-saving and cost-efficient method resulting higher succes...

Economic problems related to the prevention, diagnosis and treatment of rare diseases are presented paying particular attention to the costs of financing treatment, including the issue of its refund, which is a fundamental and difficult to solve economic problem of the health care system. Rare diseases, despite the low frequency of occurrence, together cover a large group of diseases being a se...