The Press and Journal (Inverness, Highlands, and Islands)
‘No’ to cystic fibrosis meds
Health: Life-prolonging drugs ‘not cost effective’
Two medicines that reduce the main cause of death in cystic fibrosis will not be routinely available on the NHS in Scotland.
The Scottish Medicines Consortium (SMC) has ruled them out over doubts about cost-effectiveness.
Cystic fibrosis is a debilitating life-shortening illness that creates a mucus build-up that causes chronic lung infections and progressive lung damage.
Orkambi is used to treat cystic fibrosis in patients aged two and older, while Symkevi treats those aged 12 and up. The SMC heard both are used to reduce hospital admissions.
The Cystic Fibrosis Trust previously said Orkambi could benefit more than 330 people in Scotland.
While not a cure, it slows decline in lung function.
SMC chairman Dr Alan MacDonald said the consortium recognised the decisions would be “disappointing”.
“We understand the profound impact that cystic fibrosis has on patients and their families,” he said.
“Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines.
“(But) there remained significant uncertainty
“Patient groups and clinicians gave powerful testimonies”
around their overall health benefits in the long-term, in relation to their costs.
“In order to be able to accept these medicines, the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that.”
Health Secretary Jeane Freeman said she understood the “great disappointment” that would be felt from the decisions.
She said: “Since the government became aware of the SMC decision last month we have been working with the SMC, the manufacturer and other interested parties to develop the principles of a solution that can make possible the widest availability of these medicines, wherever clinically appropriate.
“This work continues now, as a matter of urgency.”
Manufacturer Vertex said it hoped to reach a solution with the Scottish Government regarding broad access to the drugs for eligible patients over the coming weeks.
In the meantime, the company said it would continue to allow access on an individual basis based on clinical need, which applies to 65 patients across Scotland.
Cystic Fibrosis Trust chief executive David Ramsden said: “While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these lifesaving drugs now.”