Abstract
Rare diseases are any diseases that affected the relatively small number of patients, and generally chronically debilitating, life threatening. The fact that 7000 different rare diseases and disorders affecting more than 300 million people worldwide is reported. Many of these are considered as genetic disorders and affected patients in early life. Rare disease is definitely in the space of unmet medical needs, and orphan drugs, which are the drugs for rare diseases, should be a key in drug development for resolving unmet medical needs. However, it is difficult to obtain adequate numbers of patients in clinical trials due to the rarity. Currently, The number of orphan drugs in development has been growing with the rise of science. I overview the designation and supporting systems for development of orphan drugs in Japan and foreign country, and introduce our experience of promoting the orphan drug in neuromuscular fields.
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