Scottish Government Announces New Pathway Opens for Ultra-Orphan Treatments

09 October 2018

To be eligible for this pathway, a company needs to establish that:

  • The condition has a prevalence of 1 in 50,000 or less in Scotland,
  • The medicine has an EMA orphan designation for the condition and this is maintained at time of marketing authorisation,
  • The condition is chronic and severely disabling and
  • The condition requires highly specialised management.

SMA has been designated an ultra-rare condition.

The good and hopeful news is that medicines that fall under the new definition and which have been recently reviewed by SMC but not recommended for routine use, will be admitted to the new pathway. This should include nusinersen. If then the Scottish Medicines Consortium (SMC) consider it clinically effective, it will be made available on the NHS in Scotland for at least three years while further information on its effectiveness is gathered. Following this period, the SMC would then review the evidence and make a final decision on its availability on the NHS in Scotland. You can read more about the process on the SMC website

We will continue to work hard with the other charities, Biogen, and members of the SMC and Scottish Parliament to ensure that nusinersen is re-assessed under the new pathway as soon as possible.