Vertex has revealed that it will be submitting its cystic fibrosis medicines Orkambi and Symkevi to the Scottish Medicines Consortium for appraisal, following “constructive discussions” with the Scottish government.
If accepted by the SMC for use on the NHS in Scotland, eligible patients with the condition in Scotland could have access to these precision medicines in 2019, the firm noted.
“Our recent conversations with the Scottish Government, and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators,” noted Ludovic Fenaux, senior vice president of Vertex International.
“Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF,” he said, adding that the firm is “hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon.”
Access to Vertex’ cystic fibrosis medicines via the NHS in England and Wales is still very uncertain, as discussions with NHS England and the National Institute for Health and Care Excellence over the cost-effectiveness of these medicines are ongoing.
The parties have been in talks over provision of Vertex’ cystic fibrosis medicines since NICE first rejected Orkambi back in 2016, deeming it too expensive.
Negotiations remain confidential, but earlier this year Cystic Fibrosis Trust chief executive David Ramsden said the latest offer would see NHS England pay Vertex around £500 million over the next five years and over £1 billion over the next 10 years for its existing and future CF therapies, though there have been further discussions since then.
As part of an inquiry into the situation, MPs recently requested relevant documents from all parties, including full details of all formal offers made to NHS England and evidence in support of the firm’s public statement that it has offered the NHS in England the lowest price for its cystic fibrosis drugs of any country in the world.