Drug row could cost lives

A COUPLE from Castleknock is calling on the Government and a pharmaceutical company to reach an agreement on the cost of a new drug that could extend the life of their young child.

Marie and Niall Duffy’s five-year-old daughter Grace suffers from Cystic Fibrosis and they believe she could benefit from the drug, Orkambi.

The drug can increase lung function and weight in sufferers as well as slowing down the disease progression, meaning less hospital admissions.

However, treatment is costly and the HSE and drug manufacturer Vertex have failed to reach an agreement on its price, despite months of negotiations.

Grace was born in November 2011 at the Rotunda and before being discharged underwent a routine check called a heel prick test. 

Her parents thought nothing of it but two weeks later, while Marie was registering Grace’s birth, a phone call changed their lives forever.

“I was at the birth registration office, registering Grace’s birth, when the children’s hospital number appeared on my phone,” she said.

“Grace’s heel prick results had come back and they showed that Grace may have a lung disease called Cystic Fibrosis.”

Grace had showed no symptoms or signs that anything was wrong, but the following day she tested positive for Cystic Fibrosis.

“We were told her life expectancy will be around mid-20s,” Marie recalled. 

“Can you imagine hearing that news while holding your precious newborn baby in your arms?”

Marie and Niall were taught how to give Grace medications, treatment and physio, which has to be done twice a day for the rest of her life. 

“The support we received from the hospital staff was amazing,” Marie said.

“We learned more about the disease and the importance of keeping her healthy, and away from people with coughs and colds.”

Brave Grace is now doing relatively well, despite the regime needed to keep her in the best shape possible, which includes taking up to 20 tablets a day.

“She inhales medication to keep her lungs healthy, vitamins to keep her from getting malnutrition and a very high calorie diet to keep her weight in check,” Marie said.

 “And she does all of this with a smile on her face as she knows no different.

“We attend the hospital regularly to keep her in check but we potentially face a long, difficult and unknown road with our daughter.

“We have learned that CF by nature is a very unpredictable illness and you have no idea what tomorrow will bring so we just have to live in the moment.”

When Grace was diagnosed in 2011, her parents were told there were huge efforts being made to find a cure and make CF more manageable.

They were hopeful that by the time she reaches her teens, Grace would have a better outcome.

“I hung onto this hope with everything I had,” said Marie.

“So now this drug Orkambi has come along and seeing people benefit from this drug and preventing damage to the lungs is amazing.”

However, the Duffys and hundreds of other Irish families with CF sufferers have had their hopes dashed. 

The HSE say the cost of Orkambi is considered “unjustifiably expensive” and “not sufficiently cost effective” and has spent months trying to negotiate a price reduction.

“The HSE has undertaken considerable engagement and negotiation with the manufacturer Vertex over a protracted period in an effort to secure a significant price reduction for Orkambi with a view to having it approved for reimbursement for CF sufferers in Ireland,” a HSE spokesperson said.

However, the HSE has expressed disappointment with Vertex’s “level of meaningful engagement” in lowering what it called the “exorbitant” cost of the drug during the talks, and has yet to make an official final decision on the matter.

In a letter to Cystic Fibrosis Ireland last week, Vertex said it had “continually engaged in good faith with the HSE” and denied it had failed to meaningfully engage in negotiations.

Company Vice President Simon Lem
said Vertex was committed to finding a solution and called on Minister for Health, Simon Harris, to intervene ahead of the HSE making a decison on the drug “in the mutual interests of patients, clinicians and industry.”

“Vertex stands ready to meet the Minister at short notice in order to resolve this vitally important issue for the CF community of Ireland as soon as possible,” he wrote.

The Vertext letter was in response to an open letter from Cystic Fibrosis Ireland.

This Wednesday (December 7) the Duffys will join other families of CF sufferers in a protest outside the Dáil to vent their frustration over the situation.