Review of Access to New Medicines

An independent review to assess the impact of the new approach introduced in 2014 by Scottish Medicines Consortium (SMC).

Appendix 2: Task & Finish Group Definitions for end-of-life, orphan and ultra-orphan medicines

Task and Finish Group Definitions

End-of-Life Medicine:

"A medicine used to treat a condition at a stage that usually leads to death within 3 years with currently available treatments."

Orphan Medicine:

"A medicine with European Medicines Agency ( EMA) designated orphan status ( i.e. conditions affecting fewer than 2,500 people in a population of 5 million) or a medicine to treat an equivalent size of population irrespective of whether it has designated orphan status."

Ultra-Orphan Medicine:

"A medicine used to treat a condition with a prevalence of 1 in 50,000 or less (or around 100 people in Scotland)".


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