The Scottish government has agreed a deal with the manufacturer of Orkambi, the cystic fibrosis drug that NHS England has said is unaffordable for its patients.
Scotland has not disclosed the price it has agreed to pay to treat 350 patients who are eligible for the drug. In England, there are more than 10,000 children and adults with the disease, nearly half of whom could benefit, making the potential bill south of the border very high. NHS England offered £500m over five years for Orkambi and other upcoming cystic fibrosis drugs, but the manufacturer, Vertex, turned that down.
As part of the deal with the Scottish government for access to Orkambi and a sister drug, Symkevi, over five years, Vertex has agreed to data being collected to show how well the drugs work in real life as opposed to clinical trials.
The Scottish Medicines Consortium ruled last month that the two drugs were not value for money, as did the National Institute for Health and Care Excellence in England. Orkambi has a list price of more than £100,000 per patient per year. Vertex will have agreed a discount with the Scottish government.
Campaigners called for the rest of the UK to come to an agreement with the company. David Ramsden, the chief executive of the Cystic Fibrosis Trust, called it “a landmark moment” for families in Scotland who have campaigned for years for the drugs, which can stabilise patients’ lung function and reduce admissions to hospital.
“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,” he said. “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.”
A Department of Health and Social Care spokesperson said: “We always follow clinical advice on what medicines should be made available to patients, ensuring they provide value for money for the NHS and taxpayers.
“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.
“The health secretary will be bringing in Vertex representatives to explore what the barriers are to reaching an agreement.”
