Two cystic fibrosis drugs won’t be made available on NHS

SMC has ruled out medicines due to doubts over cost-effectiveness

Two medicines that reduce the main cause of death for people with cystic fibrosis will not be made routinely available on the NHS in Scotland.

The Scottish Medicines Consortium ( SMC) said it heard “powerful testimonies” from patients and medics about the “potential benefits” of the drugs but ruled them out over doubts on cost-effectiveness.

Cystic fibrosis is a debilitating lifeshortening 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”.

This work continues now, as a matter of urgency. HEALTH SECRETARY JEANE FREEMAN

“We understand the profound impact that cystic fibrosis has on patients and their families and we are aware of the need for effective treatments that target the underlying cause,” he said.

“Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines.

“As these are orphan medicines, our committee members were able to apply additional flexibility in their decisionmaking but there remained significant uncertainty 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 with 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.”

She said a “considerable number” of patients are accessing the medicines through the Scottish Government’s system for individual access.