Joy following cystic fibrosis drug deal

A NORTHSIDE mother, whose daughter has cystic fibrosis, said she is delighted a deal for a drug that could improve her quality of life has been agreed between the Health Service Executive and the manufacturers. 

The HSE and pharmaceutical company, Vertex, have come to an agreement that will see Orkambi (for patients aged 12 and over) and Kalydeco (for patients aged 2-5) made available from next month for some cystic fibrosis patients.  Laura Carrick (14), from Raheny, possesses the gene – CF Double Delta F508, the most common form of cystic fibrosis – that makes her eligible to receive Orkambi, a drug that could help treat the effects of her illness. Her mother Bernadette told Northside People: “We were delighted when we heard the news. It is fantastic for us.

“Laura has an appointment with her doctor at Crumlin Hospital next month and we’re hoping for confirmation that she will be able to get the drug. 

“She is due to receive Orkambi as it suits her gene-type but we’ll have to wait and see how exactly it is going to be given out and who will get it first.

“Laura is delighted with the news. They say it’s a wonder drug so we’ll see.

“I could not believe it when the news came on the TV that we finally got an answer and it was a ‘Yes’. 

“I was due to go to another protest on the day the agreement was announced, but didn’t go in to it then.” 

Bernadette, along with other parents, spent many years campaigning on behalf of cystic fibrosis patients.

Their campaign included a candlelit vigil in February at the Dail for all those who lost the battle, and for hope that a deal would be reached for the drugs.

“As a parent of a child with cystic fibrosis I am very grateful for all who have campaigned like Gillian McNulty and so many more like another hard working mam, Bernie Martin, who did amazing videos for cystic fibrosis found under #yes Orkambi,” said Bernadette.   

“Our children are heroes. They fight a battle with cystic fibrosis every day and l for one am most grateful that they now have the drugs, Orkambi and Kalydeco, to help them in this fight.” 

Laura requires the use of a nebuliser several times a day, she must do physio daily and often misses school due to tiredness as a result of her condition.

Every year her lung function is decreasing. 

“They say this drug would only give a 3 per cent increase but 3 per cent for a person with cystic fibrosis is vital,” said Bernadette. 

While it is not certain whether Orkambi would have the same positive effects on Laura as it has on other patients, Bernadette sees it as another chance to make life easier for her daughter. “This drug could at least help her get up and go to school in the morning and then we can look to the future,” Bernadette added.

“Then she can think: maybe I can get to college and get a job and she wouldn’t be leaning on the HSE as much because she’d be earning herself. It’s important to point out that Orkambi is not a cure for cystic fibrosis but it greatly improves a person’s quality of life.”

On April 13, Bernadette, Laura, Laura’s sister Louise and close friends took part in Cystic Fibrosis Ireland’s flagship fundraising appeal, 65 Roses Day.

On the day they, along with many others, sold purple flowers to help raise funds for cystic fibrosis services and awareness of the condition.

Meanwhile, Minister for Health Simon Harris said he wanted to recognise the work the HSE put into the negotiations with the drug manufacturers over a long period.

“I thank them for that,” he said. “I want to also especially acknowledge that this has been an extraordinarily difficult time for CF patients, their families and friends as they have been waiting for this process to conclude.”