Mum welcomes 'life-changing' drug access for daughter

A Londonderry mother has said the continued access of new cystic fibrosis (CF) drugs in Northern Ireland will be "life-changing" for her one-year-old daughter.

Laya Barr’s daughter, Robyn, was diagnosed with CF when she was just two weeks old.

Kaftrio, Symkevi and Orkambi are modular drugs that work to treat the root cause by bypassing the genetic errors responsible for the disease.

Other campaigners said the news was a "big breakthrough".

In December, the National Institute for Health and Care Excellence (NICE) published draft guidance deeming the drugs too expensive to be recommended for use on the NHS.

However, on Tuesday, Vertex, which manufactures the medication, confirmed it would be made available to NHS patients in NI.

The company said it has finalised a reimbursement agreement to allow for "extended long-term access" to the medication for all eligible existing and future patients.

Last week, a similar deal was announced with the NICE to provide NHS patients in England with access to the drugs.

The initial guidance from NICE stated patients already taking Kaftrio, Symkevi and Orkambi would have been kept on them, but the drug would not be prescribed for new CF patients, including children.

Laya Barr’s daughter, Robyn, was diagnosed with CF when she was just two weeks old.

Since her diagnosis, Robyn has been on numerous medications, attended multiple physiotherapy appointments and has been in and out of hospital throughout her young life.

Robyn’s mother said CF affects Robyn’s lungs, digestive system and pancreas.

She told BBC News NI that she is hopeful the drugs will help her daughter have fewer admissions to hospital, and "live a better and longer life".

"When we found out initially that these drugs weren't available here, it was like someone dangling a carrot in front of you and then taking it away - it was like a kick in the stomach for us, as parents, and that's why I campaigned for it.

"It is just so reassuring that she can now access this - it's life changing."

Ms Barr said it was not just about her daughter, but "all other children with CF, who can now potentially benefit from this being available Northern Ireland".

Liam McHugh's daughter was diagnosed in 1992.

He said that he, and other parents of children with the disease, are "absolutely ecstatic" about the latest news.

"The CF community in Northern Ireland are over the moon about the whole thing," he told BBC News NI.

"This is a big, big announcement and we're all very pleased about it."

Mr McHugh said for younger CF patients, Tuesday's announcement means they can access drugs at an earlier stage.

"It means that newly-born cystic fibrosis patients are going to have so much of a better chance of life for their future and the years ahead," he added.

"They’re going to get an early start on these life-saving drugs.

"It’s just going to mean that they’re going to live a nearly normal life - it’s a big breakthrough."

In a statement, Stormont's Health Minister Mike Nesbitt welcomed the announcement.

"The newly-published final draft Nice guidance will underpin continued access to these life-changing treatments for new and existing patients in Northern Ireland, where treatment is considered clinically appropriate by their treating clinician," he said.

Mr Nesbitt said there are still "necessary formal processes" to be completed, including the publication of the final NICE guidance.

However, he added that "very important progress has undoubtedly been made" which will be welcomed by patients and families in Northern Ireland.

Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system.

It can cause lung infections and problems with digesting food.

Symptoms usually start in early childhood and vary for each individual, but the condition gradually gets worse over time with the lungs and digestive system becoming increasingly damaged.

It can cause patients to die before reaching their 40s.

It is thought about 11,000 people have the condition in the UK.

There is no cure for CF but a range of treatments can help control symptoms and reduce complications.

Kaftrio is a triple treatment drug and has been available on the NHS in Northern Ireland since 30 July 2020.

In 2022, the former Health Minister Robin Swann approved the drug for use by suitable children over six in Northern Ireland.

Orkambi and Symkevi have been available on the NHS in Northern Ireland since 2019.

Orkambi is used to improve lung function and reduce breathing difficulties, and Symkevi is also used to treat symptoms of CF.