Vertex Pharmaceuticals has announced two of its cystic fibrosis (CF) drugs, Orkambi (lumacaftor-ivacaftor) and Symkevi (tezacaftor/ivacaftor) will now be available to eligible patients in Scotland through the National Health Service (NHS).
This is a result of the company signing a five-year access agreement with Scotland’s drug pricing regulator the Scottish Medicines Consortium (SMC). The price being paid by the SMC for the two drugs has not been disclosed, but Vertex did reveal it is required to collect real-world data for Orkambi and Symkevi to support future submissions to the regulator.
Vertex senior vice-president Ludovic Fenaux commented: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.
“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi or Symkevi.”
Vertex estimates there are a total 900 patients with CF in Scotland, but Orkmabi is only effective in CF patients with two copies of the F508del mutation.
Similarly to NHS England and the National Institute for Health and Care Excellence (NICE), the SMC had previously rejected Vertex’s submissions for Orkambi and Symkevi on the basis of price.
The latest SMC rejection of the two drugs was 12 August this year on the basis that the “company’s justification of the treatment’s cost in relation to its health benefits was not sufficient” and Vertex did not present “a sufficiently robust clinical and economic analysis”.
Just Treatment, a charity campaigning for UK-wide NHS access to Vertex’s CF drugs, has welcomed the announcement. With campaigner Emily Birchall, who is involved in a CF buyers’ club attempting to gain access to a generic version of Orkambi for use in the UK, saying the announcement is “fantastic… [and] overdue news and sadly comes too late for many, but is reward for all those who have been campaigning extremely hard to obtain access to these life changing drugs, and we are grateful to all parties who have made it happen.”
She continued: “With details of the Scottish deal soon to be shared with counterparts in NHS England, Wales and Northern Ireland, it will be abundantly clear whether or not a deal with Vertex is achievable for the other nations.”
“The rest of the UK must now find a way to treat patients, as the current situation represents one of the starkest examples we have ever seen of health inequality between the Home Nations.”