Monday 22 January 2018

Parents fight for 'miracle CF drug' to transform their four-year-old daughter Chloe's life

Four-year-old Chloe Ryan with her mum Angela at their home in Dingle, Co Kerry. Chloe’s parents are desperate for her to have a drug currently only available for children aged six and over. Photo: Don MacMonagle
Four-year-old Chloe Ryan with her mum Angela at their home in Dingle, Co Kerry. Chloe’s parents are desperate for her to have a drug currently only available for children aged six and over. Photo: Don MacMonagle

Jane O'Faherty and Alan O'Keeffe

The parents of a four-year-old girl with cystic fibrosis are appealing to the HSE to buy a drug that could drastically improve their daughter's life.

Angela and Darren Ryan from Dingle, Co Kerry, hoped they could secure the drug Kalydeco for their daughter Chloe.

But negotiations are continuing between the HSE and the manufacturers of the medication, after the suggested price of the drug was deemed too expensive by health authorities.

Chloe has the gene mutation G551D, which makes her eligible for the drug. But Kalydeco is only available for children over the age of six.

Without the drug, Chloe faces the next two years suffering lung infections that can scar and damage her lungs, said her mother.

Speaking to the Irish Independent, Mrs Ryan said securing the drug for children like Chloe is a matter of urgency.

"We were under the assumption that it would be allowed, as there are only 18 children in the country who have this condition and would be eligible," she said.

"Why can't they go with the price for those over six, if that has already been negotiated?"

Kalydeco, also known as Ivacaftor, has proven effective in treating cystic fibrosis and can be used by children aged two to five in America, Scotland and other European countries.

In 2013, the HSE agreed a price with manufacturer Vertex to secure Kalydeco for children aged six and over.

In that age group, it has been shown to significantly increase lung function, while slowing the progression of lung disease and significantly reducing hospital admissions.

Research suggests eligible pre-school children could also benefit from improved pancreatic function, weight gain and movement of salt between cells.

While the drug may not prevent Chloe's risk of contracting infections, Angela says it will help her "live a more normal life".

It has been hailed a "miracle drug" which has transformed the lives of patients with cystic fibrosis.

"There are people with cystic fibrosis who were on transplant waiting lists and couldn't get out of bed in the morning," she said.

"These people have started running marathons and have been taken off transplant lists after taking the drug.

"It treats the cause of the disease, not the symptoms. People get a lot fewer chest infections with the drug. Chest infections cause scars to form on the lungs. When scars get too bad, people need lung transplants.

"Chloe could suffer scars for the next two years if she has to wait until she is six. She shouldn't have to face the risk of another two years of scarring on her lungs."

Chloe has started in primary school, and Angela says it's been challenging to make sure usual school bugs don't aggravate her respiratory issues.

"When she was here at home you could manage all that, but in school it's more difficult," she said.

"If there is a birthday party on, we have to check if any of the other children are ill.

"We can't keep her in a bubble."

At present, Chloe is looked after by doctors at the Butterfly Unit at University Hospital Limerick.

But her family will not give up hope of getting the drug, and will continue to petition Health Minister Simon Harris and his department.

Little Chloe uses a nebuliser twice daily and swallows a wide array of tablets every day.

Some days, she has to take more than two dozen to try to prevent her from getting sick.

A Health Service Executive spokesman said that the HSE and Vertex met on January 5 as part of ongoing discussions about access to the cystic fibrosis drugs Orkambi and Kalydeco.

"Work is ongoing and both parties are exploring all possible options," the spokesman said.

"Following completion of this process the HSE's Drugs Committee will make a recommendation on the cost-effectiveness of Orkambi to the HSE Directorate for a decision taking into consideration the manufacturer's latest price offer."

Irish Independent

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