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Scotland To Improve Early Patient Access to ‘Ultra-Orphan’ Drugs

Scotland is changing that way that medicines for very rare diseases are assessed by the health technology assessment body, the SMC. The changes include a new definition of “ultra-orphan medicine” and a new system for provisionally funding such products while more evidence is gathered on their effectiveness

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Scotland is to have a new appraisal pathway for ultra-orphan drugs

Patients in Scotland with very rare diseases should get faster access to new treatments as a result of changes being made to the pathway used by the health technology assessment (HTA) body, the Scottish Medicines Consortium, to evaluate “ultra-orphan medicines.”

From Oct. 1 this year, if a medicine meets the definition of “ultra-orphan”, and the SMC considers it to be...

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