1. Although there is no formal, or internationally agreed, definition of an ultra-orphan disease, the National Institute for Health and Clinical Excellence (NICE) uses the term for conditions occurring in less than 1000 people in the UK (equating to less than 100 people in Scotland).
2. Novel medicines in this context mean a new chemical entity where the mode of action is different to that of existing medicines.
5. Patient Access Schemes represent a discount arrangement to facilitate the entry of new medicines and managed by National Services Scotland (NSS) through assessment of the scheme via the Patient Access Scheme Assessment Group (PASAG).
Email: VERONICA MOFFAT