Will the new ultra-orphan definition improve access to medicines in Scotland?
Patients in Scotland with very rare conditions should get earlier access to new medicines due to changes in funding rules.
The Scottish Government is introducing a new definition for ‘ultra-orphan’ medicines, which are used in treating people with very rare conditions affecting fewer than one in 50,000. Across Scotland, the definition applies to around 100 people.
Where a medicine meets the definition and the Scottish Medicines Consortium (SMC) considers it clinically effective, the Scottish Government will make it available on the NHS for at least three years to allow its effectiveness to be measured in practice.
After three years, the SMC will review the evidence and decide whether the medicine should be available routinely in Scotland.
Health secretary Shona Robison called on pharmaceutical companies to make the medicines available at a fair price.
Scottish Government: Treatments for rare conditions