Orphan drugs benefit from fast-track approval – but are they getting to patients?

Rare diseases are just that – rare. We may not have heard of them, and we probably don’t know anyone who suffers from them. But rare diseases can have a huge impact on a country’s health care spending as patients can struggle to be treated.

Orphan drug designation, awarded by the US Food and Drug Administration and European Union regulators, can provide drug makers with a period of market exclusivity and other support while they bring these much-needed drugs to the market. Orphan drugs are those developed for rare diseases and conditions that affect fewer than 200,000 people in the USA, or five per 10,000 or fewer people in the European Union.

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