Two “life-changing” cystic fibrosis drugs have been rejected for routine use by the NHS in Scotland. The Scottish Medicines Consortium (SMC) said there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs. Manufacturer Vertex and the Scottish government are now working “as a matter of urgency” to find a way to allow broad access for eligible patients. Campaigners described the decision as disappointing and unfair.
Orkambi and Symkevi help to improve lung health in patients with cystic fibrosis, but cost about £100,000 per year per patient. The SMC said it heard “strong evidence” from patient groups and recognised that the decision would be “disappointing”. But chairman Dr Alan MacDonald said: “There remained significant uncertainty around their overall health benefits in the long-term, in relation to their costs. “In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that.” The SNP MP for Motherwell and Wishaw, Marion Fellows – whose three-year-old granddaughter Saoirse suffers from cystic fibrosis – said she was “disappointed” about the decision.
Saoirse was diagnosed with the condition when she was...