Cystic fibrosis drugs rejected for use by NHS in Scotland

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Two cystic fibrosis drugs which have been described as “life-changing” by campaigners have been rejected for use by the NHS in Scotland.

The Scottish Medicines Consortium (SMC) said there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs.

Both drugs are manufactured by Vertex.

It said it was working with the Scottish government to find a solution to allow broad access for eligible patients as a matter of urgency.

The SMC said it heard “strong evidence” from patient groups while considering the use of Orkambi (lumacaftor-ivacaftor) and Symkevi (tezacaftor-ivacaftor).

Chairman Dr Alan MacDonald said it recognised that the decisions would be disappointing.

But he said: “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.”


‘It’s not a pretty thing to watch’

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SNP MP Marion Fellows with granddaughter Saoirse, who was diagnosed when she was three weeks old

The SNP MP for Motherwell and Wishaw, Marion Fellows – whose three-year-old granddaughter Saoirse suffers from cystic fibrosis – said she was “disappointed” about the decision.

Saoirse was diagnosed with the condition when she was three weeks old and received painful physiotherapy.

Ms Fellows said: “It’s not a pretty thing watching a three-week old baby scream when they’re getting their first lot of physio and you’re told by their parents no, we’re not allowed to comfort her because her body has to get used to this.

“Now my granddaughter is used to it, but we still don’t want that to happen to other people.”

She added: “I will continue in Westminster to deal with the outcome from this result and hope that things in the future pick up and will get better. I’m sure at the end of the day the Scottish government and Vertex can come to some good agreement that will change this decision.”


Scottish patients had previously been able to gain interim access to Orkambi and Symkevi through a system known as Peer Approved Clinical System Tier 2 (PACS Tier 2).

The Scottish government introduced this system in June 2018, whereby doctors applied for access on behalf of their patients.

Earlier this year campaigners appealed to the UK government to use its powers to break a deadlock in making Orkambi available on the NHS in England.

Vertex refused a £500m offer for the drug over five years – the “largest commitment” NHS England had ever made.

Those affected want other drug firms to be asked to make a cheaper version.

The Department of Health said its approach “remains urging Vertex to accept NHS England’s generous offer”.

Scottish Health Secretary Jeane Freeman 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.”

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage, and affects about 10,400 people in the UK.

Only around half of those with the condition live to the age of 40.


What is Orkambi?

Since 2015, the drug Orkambi has been licensed to treat cystic fibrosis in patients from two-year-olds to adults, who have a specific genetic mutation known as F508del.

It was not available on the NHS, except for certain people on compassionate grounds. Symkevi is used to treat the same mutation in patients age 12 and older.

The mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Orkambi has been shown in clinical trials to improve lung function and respiratory symptoms in people with cystic fibrosis.

It is the first of a string of drugs that have been developed, with newer ones expected to be even more effective.

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