Vertex cystic fibrosis drugs become available in Scotland after NHS rejection
pharmafile | September 12, 2019 | News story | Sales and Marketing | SMC, Vertex, cystic fibrosis
Two potentially life-changing cystic fibrosis drugs are to become available for a potential pool of 400 patients in Scotland just a month after they were rejected by the NHS.
Boston-based pharmaceutical company Vertex has announced it has signed a five-year deal with the Scottish Government for Orkambi and Symkevi (lumacaftor/ivacaftor) – drugs which help improve lung health, but were to cost about £100,000 per patient.
The Scottish Medicines Consortium (SMC) had previously rejected the use of the drugs stating that there were uncertainties about its long-term cost effectiveness. It is hoped that the deal will eventually lead the SMC approving the drugs in the future.
Orkambi has been licensed to treat cystic fibrosis since 2015 in patients aged two years and above who have a specific genetic mutation known as F508del. Symkevi is used to treat the same mutation but in patients aged 12 and older.
The mutation itself causes the production of an abnormal protein that interferes with the transportation of water and chloride in the body.
Orkambi is the first drug to show effectiveness in improving lung health and is the first in a string of drugs that have been developed, with newer ones expected to have even greater efficacy.
Scottish Health Secretary, Jeane Freeman, said: “This is fantastic news for those living in Scotland with cystic fibrosis and their families. This new pricing agreement with medicines manufacturer Vertex Pharmaceuticals, secured by the Scottish Government, will allow more people with cystic fibrosis to benefit from medicines Orkambi and Symkevi and live fuller lives for longer.
“The agreement has been reached after extensive discussions between the Scottish Government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount.”
Around 900 people in Scotland live with cystic fibrosis and, of these, 400 may be eligible for treatment with the new medications. However, some Scottish patients have previously had access to the drugs through the Peer Approved Clinical System Tier 2 (PACS Tier 2), which permits doctors to apply for access on behalf of specific patients on compassionate grounds.
Nik Kiran
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