Medical bosses reject offering cystic fibrosis drugs on the NHS in Scotland

Campaigners say the news will come as a shock to families

Author: John CallanPublished 12th Aug 2019

Two drugs for treating cystic fibrosis will not be offered on the NHS in Scotland, despite calls from campaigners.

The Scottish Medicines Consortium has ruled against offering Orkambi and Symkevi because of concerns about its cost.

Orkambi can cost up to £100,000 a year per patient and has already been rejected by the NHS in England on similar grounds.

It's thought it could help improve the lives of around 330 people in Scotland who have the debilitating lung condition.

SMC Chairman Dr Alan MacDonald said: “We recognise that the decisions on lumacaftor-ivacaftor Orkambi and tezacaftor-ivacaftor Symkevi will be disappointing .

"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.

"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 decision making, 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.''

"The Government hopes that all parties continue to work together to achieve a positive outcome for all the children and adults with cystic fibrosis in Scotland.''

Manufacturer Vertex said it hoped to reach a solution with the Scottish Government on making the drug more broadly available.

Cystic Fibrosis Trust chief executive David Ramsden said: "The SMC's advice will come as a shock to people with cystic fibrosis and their families in Scotland.

"While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these life-saving drugs now.

"The tragic wait has to end and now is the time for the Scottish Government to remain committed and Vertex to do all they can to ensure that this chance does not slip away again."