DRUGS that can help extend the life of people with cystic fibrosis (CF) will be made available on the NHS in Scotland after a deal was reached on costs.
Scotland is the first part of the UK to approve Orkambi and Symkevi for routine NHS use. It is estimated that about 400 patients will benefit.
Among those who welcomed the deal was SNP MP Marion Fellows, whose three-year-old granddaughter has CF.
Pharmaceutical firm Vertex International has reached a pricing agreement with the Scottish Government. The drugs will be made available for five years with a “confidential discount”. The move comes just a month after the Scottish Medicines Consortium (SMC) – the body that approves drugs for use by the NHS in Scotland – rejected the treatments, saying the “cost in relation to its health benefits was not sufficient”.
Reports suggested Orkambi would cost £100,000 a year per patient. England’s National Institute for Health and Care Excellence (Nice) has deemed it not cost-effective.
Motherwell and Wishaw MP Fellows said: “As the granny of three-year-old Saoirse, who suffers from cystic fibrosis, I know personally the impact CF can have on families. I’m so glad that so many families across Scotland will benefit from these new drugs. Hundreds will be able to lead longer and fuller lives thanks to this decision.
“The Scottish Government never let up in discussions and is leading the way in CF treatment. But CF knows no borders. The UK Government must follow the Scottish Government’s lead and make it available to sufferers in England also.
“Of the 900 people in Scotland with CF, 400 may benefit from the new drugs. This is such a big moment for these people and their families. They have been relentless and inspiring in their efforts to see these drugs become available in Scotland.”
David Ramsden, chief executive of the Cystic Fibrosis Trust, hailed the access agreement as a “landmark moment for the hundreds of people with cystic fibrosis and their families across Scotland who have tirelessly campaigned for years to access these drugs”.
He said: “This breakthrough is a victory for their perseverance and enduring hope. It means eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.
“We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland.
“Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery. Scotland’s success must now be replicated across the UK without further damaging delay.”
Cystic fibrosis is a debilitating, life-shortening illness which creates a mucus build-up that causes chronic lung infections and progressive lung damage.
Orkambi is used to treat patients aged two and above, while Symkevi treats those aged 12 and up. While the drugs are not a cure, they can slow the decline in lung function, which is the main cause of death.
Scottish Health Secretary Jeane Freeman said of the agreement: “This is fantastic news for those living in Scotland with cystic fibrosis and their families.”
As part of the five-year access agreement, Vertex International will collect data on the effectiveness of the drugs, which will be used in future submissions to the SMC.
Ludovic Fenaux, senior vice-president at Vertex International, said: “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.”
A spokeswoman for the UK Government’s Department of Health and Social Care said: “NHS England has put forward a very generous offer, the largest in its 70-year history, that would make Orkambi available to NHS patients in England and we urge Vertex to accept the deal so patients can benefit from treatment as soon as possible.”
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