How Scotland Plans To Improve Access To Drugs For Very Rare Diseases

Scotland is to change the way it appraises new drugs for very rare diseases for use on the National Health Service, with a new “conditional yes” option for the Scottish Medicines Consortium that will require further evidence-gathering and subsequent reassessment, and a new appraisal procedure for “true ultra-orphan drugs” for very rare conditions where a cost-benefit assessment may not be appropriate. There will also be provision for earlier engagement on orphan drug pricing with the pharmaceutical industry and for more patient involvement in the SMC’s processes.