Weesgeneesmiddelen: beschikbaarheid, betrouwbaarheid en bekostiging

Orphan drugs are drugs used in the treatment of life-threatening or chronic diseases that affect fewer than 1 out of 2000 persons in the European Union. Since the implementation of the European Regulation on Orphan Medicinal Products in 2000, 61 orphan drugs have been brought to market. One-third of these were granted their marketing authorisations based on non-comparative clinical research. Certain orphan drugs for extramural use will be transferred to the performance-based hospital financing system within the next few years. Unapproved orphan drugs are generally not reimbursed. In so-called compassionate use programmes, unauthorised orphan drugs can still become available to patients who do not participate in clinical trials. Compassionate use drugs are made available by the manufacturer.