What is an “orphan drug"?

It is estimated that over 6,000 rare diseases exist, although some say the actual number is closer to 8,000. Since the prevalence of such diseases is low and the development cost of medicines is high, industry is reluctant to invest in the absence of a foreseeable return.

The medicines for these diseases or conditions are therefore indicated as ‘orphan medicines’, having no ‘sponsor’ or ‘parent’ to develop them.

The European Union has defined an orphan medicine as one that is intended for a life-threatening or seriously debilitating condition that has no satisfactory method of treatment and also:

  • Affects 5 people or less for every 10,000 citizens (known as the “prevalence criterion”);
    or 
  • Where the treatment – without incentives – would not justify the investment in itsdevelopment (known as the “insufficient profit criterion”).

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