New drugs for cystic fibrosis patients in Scotland

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Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage and affects about 900 people in Scotland

Two “life-changing” cystic fibrosis drugs are to be made available to about 400 patients in Scotland.

It comes a month after the medications were rejected for use by the NHS north of the border.

Orkambi and Symkevi help improve lung health, but cost about £100,000 per patient, per year.

Now Scottish ministers have struck a five year deal with pharmaceutical company Vertex, securing a “confidential discount” on the drugs.

However patients in other parts of the UK are still not eligible for the treatment.

It has led to calls for an end to the “postcode lottery” by campaigners who want the drugs made available in England, Wales and Northern Ireland.

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The drugs can reduce hospital admissions by stabilising lung health

Scotland’s Health Secretary Jeane Freeman described the deal as “fantastic news” which would allow cystic fibrosis patients to “live fuller lives for longer”.

“The agreement has been reached after extensive discussions between the Scottish government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount,” she added.

In August, the Scottish Medicines Consortium (SMC) rejected the use of the drug saying there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs.

As part of the five-year agreement, US-based Vertex has also committed to collecting data on the medicines to support any future submissions to the SMC.

Ludovic Fenaux, Vertex senior vice president, said: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.

“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi and Symkevi.”

‘Landmark moment’

Some Scottish patients have previously had access to Orkambi and Symkevi through the Peer Approved Clinical System Tier 2 (PACS Tier 2), which allows doctors to apply for access on behalf of individual patients.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “This is a landmark moment for the hundreds of people with CF and their families across Scotland who have tirelessly campaigned for years to access these drugs.

“This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.”

He said the trust would continue to campaign for access to the drugs in England, Wales and Northern Ireland to end the “postcode lottery” for thousands of patients.

“Scotland’s success must now be replicated across the UK without further damaging delay.”

What are Orkambi and Symkevi?

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.

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage, and affects about 10,400 people in the UK – around 900 of them in Scotland.

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

NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with cystic fibrosis.

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 – which was described as the “largest commitment” NHS England had ever made.

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

The UK government’s Department of Health said its approach was still to urge Vertex to accept NHS England’s “generous offer”.