Date lodged: 3 October 2018
To ask the Scottish Government whether a decision has been made regarding the cystic fibrosis drug, Orkambi, which was being considered for assessment under the new ultra-orphan medicine pathway that took effect on 1 October 2018.
Answered by: Jeane Freeman 29 October 2018
Yes. The Scottish Medicines Consortium (SMC) has determined, further to a request from the Chief Pharmaceutical Officer, that Orkambi ® is not suitable for assessment under the new ultra-orphan pathway. The SMC has come to this view having regard to the approximately 900 people in Scotland who suffer from cystic fibrosis. The ultra-orphan pathway is intended for conditions with a prevalence of 1 in 50,000 or less, equivalent to around 100 people in Scotland. The SMC takes decisions independently of Ministers.
The Scottish Government continues to call upon the manufacturer of Orkambi ® , Vertex Pharmaceuticals, to make a new submission to the SMC, at a fair price that allows general access on the NHS. Clinicians may also apply for individual patients to have access to the medicine, through the Peer Approved Clinical System (PACS) Tier Two, which has been in place since June 2018. The Scottish Government is collecting information from Health Boards about the use of PACS Tier Two and plans to publish statistics as part of the previously announced reviews of the new process six and twelve months after its commencement.