Abstract:

Rare diseases are health problems that have been ignored for many years by both the health and pharmaceutical sector as well as by scientific and political communities due to their low prevalence. However, patients with these diseases deserve access to quality, safe and effective medications and treatments as well as patients with more common diseases. With the recent developments, especially in the field of medicine, technology and human rights, a growing interest has begun in the world in rare diseases and orphan drugs used in the treatment of these diseases. This study aims to raise awareness of this issue by presenting an assessment of the current situation in the world and in Turkey regarding rare diseases and orphan drugs that bring serious burdens to individuals and communities. In this context, rare diseases and orphan drugs have been studied in a conceptual way and the developments and steps taken in this area have been sought by statistics, research results and projections.