Initial assessment of ultra-orphan medicines

The Scottish Medicines Consortium (SMC) has been conducting initial assessments of ultra-orphan medicines for over 4 years. Submissions for medicines that are validated as ultra-orphan according to the definition are assessed by the SMC and are then available to prescribers while further clinical effectiveness data are gathered.

No decision on access is made at this stage in the ultra-orphan process. Following stakeholder feedback, some minor improvements have been made to the initial assessment process. Going forward, initial submissions for ultra-orphan medicines will continue to be reviewed by New Drugs Committee, however, there will now be no requirement for SMC committee discussion at this stage in the process. There will continue to be patient group and clinical expert involvement. This will not affect the timelines for publication of the ultra-orphan medicines assessment report on the SMC website.