Scottish government to introduce alternative pathway for ‘Ultra-Orphan Medicines’

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.

Tagged with

Published 20. June 2018 in News, News UK