Scotland launches new ultra-orphan drug designation

The Scottish government has  launched a new “ultra-orphan” designation for medicines in development for certain severe rare diseases for which there is a high level of unmet need.

Medicines designated as an orphan drug by the European Medicines Agency may be considered for inclusion as an ultra-orphan drug by the nation’s medicines authority, the Scottish Medicines Consortium, if they meet certain criteria.

These are that the relevant condition should affect less than 1 in 50,000 people in Scotland, be “chronic and severely disabling” and require “highly specialized management.”