Abstract:
The aim of this study is to conduct the analysis of legislation in the field of orphan drugs in world
practice. The review of literature has shown that significant achievements in research and development of
orphan drugs have really begun only after adopting different laws and regulations followed by development
of therapies for rare diseases. About 7000 rare diseases affect over 350 million people worldwide and
have a negative impact on patients and their families. The research and development in the field of orphan
drugs are difficult processes because of the lack of understanding the mechanism of the disease and due
to the high cost of these processes. Therefore, to encourage the research in this field many countries have
developed and implemented legislations that offer stimuli, support and assistance to drug manufacturers,
starting with the formulation of these drugs up to receiving the marketing authorization. The Orphan
Drug Act was firstly adopted in the United States in 1983. As a result, in the first ten years, 620 drugs became
orphan, and 87 received the marketing authorization. Following the great impact of this act, Japan,
Australia and the European Union later adopted such policies. Currently, 89 orphan drugs covering 12 ATC
groups have been authorized in Europe.