The Scottish government has announced the introduction of a new pathway for ultra-orphan medicines, designed to improve access to treatments for patients with conditions affecting 1 in 50,000 people, or around 100 or less people in Scotland.
The change comes in response to the Montgomery Review, a report into the impact of changes introduced by the Scottish Medicines Consortium (SMC) in 2014 to improve patient access to end-of-life, orphan and ultra-orphan medicines. The review found that, whilst the acceptance of end-of-life, orphan and ultra-orphan drugs combined had increased from 48% between November 2011 and October 2013 to 75% between May 2014 and March 2016, in the same period only 14% of ultra-orphan drugs were approved. As a result, the report recommended that a separate assessment and decision-making process for ultra-orphan medicines be adopted.
The new ultra-orphan pathway will still involve the SMC undertaking an appraisal of the clinical and cost effectiveness of an ultra-orphan medicine. If clinically effective, including where there is uncertainty, the drug will still be made generally available on the NHS for a minimum three-year period, after which the SMC will then review the evidence and make a final decision. This new pathway will also be available for ultra-orphan medicines recently reviewed by the SMC but not recommended for routine use.
The new arrangements will come into effect from the 1st October.