Newtownabbey mum hits out at U-turn on cystic fibrosis wonder drug that saved her life

A cystic fibrosis campaigner from Northern Ireland has added her voice to calls for life-extending drugs to still be made available on the NHS so that children battling this cruel disease can have a future.

Nicole Adams from Newtownabbey is living proof of how a cystic fibrosis (CF) modulator therapy called Trikafta (also known in the UK and Europe as Kaftrio) has improved her health and given her a future she could never have imagined just a few short years ago.

Nicole, 32, was speaking as hundreds of parents of Northern Ireland children with CF have been left worried and in limbo after a recommendation to remove access to three drugs to treat it.

READ MORE: Brother’s tribute to ‘warrior’ who passed away after lifelong health battle

READ MORE: NI campaigners add their voices to calls for fair global access to CF drug

Many parents are concerned that their children will not have access to Orkambi, Symkevi and Kaftrio medicines because of proposed changes which mean it could be taken off the NHS within weeks.

Draft guidance from the National Institute for Health and Care Excellence (NICE) says the drugs are too expensive to be recommended for use on the NHS.

Vertex’s earlier drugs Orkambi and Symkevi became available on the NHS in 2019 with Kaftrio following later. They were the first effective treatments on the health service for the life-limiting condition affecting 11,000 UK patients.

But now draft guidance from NICE says the drugs are too costly to give to new patients. Kaftrio has a listed price of about £200,000 per patient per year, Symkevi £173,000 and Orkambi £104,000.

CF affects more than 500 children and adults in Northern Ireland and campaigners like Nicole say this is a devastating blow to all those impacted by the condition. It’s caused by a faulty gene which controls the movement of salt and water in and out of our cells. Sticky mucus clogs up the lungs and bowels and makes it hard to breathe and digest food.

Kaftrio has been life-changing for Nicole who has called for global affordable access to the drug that saved her life almost four years ago. Back then Nicole came close to death when her lung function dropped to just 11% but she has experienced an amazing health transformation on Kaftrio – so much so, that she has been able to have a child of her own.

Most women with cystic fibrosis were unable to cope with a healthy pregnancy prior to access to this drug. Nicole and her partner Ciaran McVarnock welcomed their baby son Colby in September 2022. While 14-month-old Colby does not have the condition, he carries the gene.

Pictures of Nicole lying gravely ill and struggling to breathe in intensive care in a Belfast hospital in December 2019 went viral when Ciaran shared them in a desperate bid to get her life-saving treatment and convinced drug company Vertex to give her Kaftrio.

Doctors told her and Ciaran that it was likely that Nicole would not survive and there was little that could be done for her. But Ciaran refused to accept Nicole’s diagnosis and did all that he could to secure Kaftrio for her. A few weeks later they were together at home after the drug helped her return from the brink.

Since then the couple have continued their campaign in order to “spread the good news ” about Kaftrio, which has now been used to treat children as young as six with CF and given them opportunities in life they would have never had before.

Nicole said: “Four years ago my lung function was 11% and I was hours from death, then I received the gift of life – which was Kaftrio. If I hadn’t got that drug when I did, I wouldn’t be here today to be happy and healthy with a baby son. I’ve gone from being on the brink of death to being a busy mum and loving life.”

She added: “I am living proof that these miracle drugs can work but now it feels like there’s been a price tag put on our heads. To be told that we can’t afford them anymore is like dangling candy in front of a baby and then snatching it away so yes it does feel that our lives are being numbered with a price.”

NICE, which advises on new medicines, has said the 8,000 NHS patients currently using Vertex’s drugs can stay on them, regardless of its final decision.

Nicole added: “While adults like me can stay on the medication, it’s demolishing the future for parents of kids with CF. I have friends whose children have CF but they are just too saddened by this news to speak out so I’m doing it on their behalf.

”We didn’t ask to be born with or have children born with this condition but we are grateful for all the drugs to treat it and all the research that has gone into CF so let us live our lives.”

In a statement, Northern Ireland’s Department of Health said that “the draft guidance is not Nice’s final guidance”.

A DoH spokesperson added: “The Department of Health has a formal link with the National Institute for Health and Care Excellence (NICE) under which NICE Technology Appraisals are reviewed locally for their legal and policy applicability in Northern Ireland.

“Where found to be applicable, they are endorsed for implementation within Health and Social Care (HSC).

“NICE has recently issued draft guidance on the use of Kaftrio, Orkambi and Symkevi for consultation. Consultation on the draft guidance closes on Friday 24 November 2023.

“The draft guidance is not NICE’s final guidance and comments received during this consultation will be considered by NICE at a further appraisal meeting before the next draft guidance is issued. NICE expect to publish final guidance around 20 March 2024.

“When final guidance is issued by NICE, the Department will consider it under the usual process for NICE technology appraisals as per the guidance set out in circular HSC (SQSD) 2/13 which is available here.”

For all the latest news, visit the Belfast Live homepage here and sign up to our daily newsletter here.