Limerick parents say access to cystic fibrosis drug is 'like winning the lottery'

58 adults and children in Limerick to get access to 'life-changing' medication next month

Fintan Walsh

Reporter:

Fintan Walsh

Limerick parents say access to cystic fibrosis drug is 'like winning the lottery'

Delight: Joy O’Donoghue and Katie Sheehan Picture: Adrian Butler

MORE THAN 50 adults and children in Limerick suffering with cystic fibrosis [CF] will have access to “life-changing” medication next month.

This follows Minister for Health Simon Harris’ announcement that vital drugs Orkambi and Kalydeco will be made available to patients in May, pending further discussions with pharmaceutical company Vertex.

Figures received by the Limerick Leader show that 30 adults and 28 children with CF will have access to the drugs, following a vigorous 10-month campaign by the CF patient community, locally and nationally.

According to a source, a representative of Vertex was in Limerick this Wednesday to meet with members of the Cystic Fibrosis Unit at University Hospital Limerick.

Mothers Joy O’Donoghue and Katie Sheehan, whose young children will soon have access to the drugs, welcomed the news on Wednesday morning.

Katie, from Cappamore, said that the positive news for her three-year-old son Michael was “like winning the lottery”.

“I am absolutely elated. When it came through yesterday, I went live on Facebook like a blubbering mess. Most of the CF community are in shock that this has actually happened.

“It was looking like that it wasn’t going to happen, but now the whole community is absolutely over the moon.”

In December, the Government was proposing a price of €30,000 per patient per year for the use of Orkambi. However, it is understood that the company had proposed a price of around €160,000 per patient per year. It not known, yet, what the agreed price is.

“You do have some people saying: ‘I (will) believe it when it is signed.’ I lived in the hope that my son was going to have a brighter future, and that we are going to get this drug. And now I know that my son is going to have a normal life. It is life-changing, we are all working on a high at the moment,” she said.

Though Joy, of Garryowen, is “slightly apprehensive” about the announcement, she said that she is delighted for the children who will be able to avail of Kalydeco.

Orkambi will be made available to patients, aged 12 and older, and Kalydeco will be available for children, aged two to five years, while other age groups will avail of other treatments following market authorisation in Europe.

“I would love to have it for his birthday in October,” Joy said, whose eight-year-old Jason received a late diagnosis when he was four-and-a-half, in April 2013.

“With Orkambi, it doesn’t work for everyone. But we deserve the right to try it, and that is what we have been fighting for. If it works, that’s fantastic. But if it doesn’t, we do know that there are other drugs in the pipeline that are coming along,” she said. 

Both Jason and Michael require constant care from their parents and medical staff.

They each have to take a cocktail of daily medication and both also have to receive regular IVs. Jason receives regular IVs, for a two-week period every three months.

The boys must also take artificial enzymes every time they eat to help them properly digest fat and they are also restricted in the kinds of activities they can participate in, such as PE and playing in play centres. In an interview with the Leader in February, Joy said that while Jason currently has 64% lung function, there is no guarantee he will be alive this time next year given the nature of the disease.

“I was told one day that I was very blasé when talking about Jason’s illness. And I said to the person: ‘Well what do you want me to do?’ Do I crawl into a corner and cry? Who is going to mind my kids? Life is for living. You don’t know what tomorrow is going to bring. And when you do have an illness, there are less guarantees about what is going to happen in your future.”

Erin Sugrue, of TLC4CF, said that the group was “ecstatic” with the news. 

“We are also very relieved because it has been a tense 10 months, particularly for people who were at an advanced stage of the disease, where on a daily basis it was becoming difficult to breathe," she said.

"And if you are in that position, of if your parent of that child, watching them suffer through that, that is heartbreaking.”