Scotland Launches New ‘Ultra-Orphan’ Assessment Pathway
Executive Summary
The Scottish government is launching its new pathway for the assessment of “ultra-orphan” medicines by the HTA body, the Scottish Medicines Consortium, in an effort to speed up access to medicines for patients with very rare diseases
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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