Patients 'ecstatic' as cystic fibrosis drugs approved
Patients with cystic fibrosis (CF) in Northern Ireland will have a "much better chance at life" after a new deal to access drugs to treat the life-limiting condition was reached, a campaigner has said.
CF is an inherited disease that causes thickened mucus to build up in the lungs, and can lead to pneumonia and bronchitis.
Kaftrio, Symkevi and Orkambi are modular drugs that work to treat the root cause by bypassing the genetic errors responsible for the disease.
But in December, the National Institute for Health and Care Excellence (NICE) published draft guidance deeming the drugs too expensive to be recommended for use on the NHS.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 Northern Ireland.
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.
Parents 'over the moon'
Liam McHugh's daughter Rachel was diagnosed with CF 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."
The initial guidance from NICE stated patients already taking Kaftrio, Symkevi and Orkambi would have been kept on them, but they would not be prescribed for new CF patients, including children.
Mr McHugh said for younger CF patients, Tuesday's announcement will "transform their lives" as they will be able to 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."
'Important progress'
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.
What is cystic fibrosis?
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.
What are Kaftrio, Orkambi and Symkevi?
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.
