The lecture explains the medical and economic aspects of special drugs for the treatment of rare diseases (orphan drugs).

Orphan drugs are drugs for the treatment of rare diseases that are not so rare overall: It is estimated that a good 5% of the population suffer from a rare disease. The mostly congenital and genetic diseases are clinically extremely challenging, both in terms of diagnosis and treatment. The business model for the drugs that have been and are being developed specifically for the individual diseases differs significantly from that of the traditional pharmaceutical industry. The lecture will shed light on the specific medical and economic aspects of orphan drugs and rare diseases and discuss the particular challenges of this therapeutic and industrial sector.