Rare Disease Day takes place every year on the last day of February. This day was
established in Europe in 2008, and is now observed in more than 80 nations. To put it in …

It is estimated that over 350 million individuals worldwide are affected by one of over 7000-
8000 rare diseases. International definitions of rare diseases vary; the definition in the …

Rare diseases are a large market for pharmaceutical and biotech companies because there
are over 6,500 rare diseases with unmet medical needs. The US and most EU governments …

Conclusion The combination of scientific advances and orphan drug legislation policies
have made it possible for orphan drug development to grow steadily in the last few decades …

Only 10–15 first‐in‐class new medicines are approved each year by the global
pharmaceutical industry for all diseases, of which less than a third is for rare (orphan) …

Rare diseases are serious, chronically and/or progressively disabling, and can be life-
limiting and life-threatening. According to the US National Institutes of Health (NIH), there …

A rare disease is characterized as (a) a serious or life-threatening disease and (b) has a
significant impact on the lifespan as well as quality of life of the individual, and (c) may …

In 2023, more than 50%(28 of 55) of the novel drugs reviewed by the US Food and Drug
Administration (FDA) received orphan drug designation for the prevention, diagnosis, or …

Rare diseases are a large market for pharmaceutical and biotech companies because there
are over 6,500 rare diseases with unmet medical needs. The US and most EU governments …

The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for
Rare Diseases: Common Issues in Drug Development (referred to as “Rare Diseases …