Scotland Launches New ‘Ultra-Orphan’ Assessment Pathway

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

Fast Track
Scotland is introducing a faster HTA process for ultra-orphan drugs.

Efforts to speed up patients’ access to drugs for very rare diseases are taking shape after the Scottish government launched its new pathway for assessing "ultra-orphan" medicines for severely disabling conditions. 

Under the pathway, drugs that meet the new definition of “ultra-orphan medicine” and are initially deemed to be clinically effective,...

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