Abstract
Drugs with the status of therapeutic substances for rare diseases are meant for diseases which occur in less than 5 people per 10000. The basic legal framework for (not only) the drug policy in the field of rare diseases was laid down by the Regulation of the European Parliament and Council from the year 2000, which had as its goal providing patients with rare diseases with the same standard of treatment that is received by patients with commonly occurring diseases.
Rare tumours represent 20% of all malignancies, but they are responsible for 50% of the mortality of all malignant tumours. Both common and rare tumours have similar symptoms.
The basic problem is that a malignancy is usually not initially thought of as the reason behind the symptoms that the patient exhibits, even when incidence of all tumours is on the rise and it can be expected that tumours will soon surpass even cardiovascular diseases as a leading cause of death. If there is a suspicion for a rare tumour, the patient should be promptly transferred to highly specialised centres (complex oncological centres), which can provide him or her the current standard in multidisciplinary approach, from diagnostics, to treatment, to follow-up care.