Rare is frequent and frequent is costly: rare diseases as a challenge for health care systems

From an economic point of view, the health care system should achieve the following goals: efficient production of health care, just distribution of resources and stable financing. The current intensive discussion on rare diseases demonstrates the difficulty in achieving all three goals at the same time. Rare diseases collectively are frequent. That is why they are an extraordinary financial and organizational challenge for payers. The treatment of patients with rare diseases is either very costly and/or extremely difficult. For many of those diseases, no effective therapy exists.