‘I want my son to have a full, long life’ - father of two-year-old CF patient
For the 1,200 Irish people living with cystic fibrosis, waiting to hear if a wonder drug is too expensive for the Government to fund means living in a cruel limbo.
Like most other 20-year-olds, Kelsey Nolan enjoys socialising with friends. Yet while her pals make haste to the nearest chipper after a night out, she returns home to face her nightly medication (around 30 in all). “My friends say they don’t know how I do it, but to me it’s life,” she shrugs. “Today, I’m in college, but I need to get up at 6am to take medication and go home during the day to do my nebuliser.”
Having been diagnosed with cystic fibrosis (CF) just before her first birthday, Kelsey has long called Carlow her ‘first’ home, and Crumlin Hospital, and later St Vincent’s Hospital, her ‘second’.
“As I’m getting older, I need to spend up to four weeks at a time in hospital, and I face a hospital stay before Christmas as I’m going downhill at the minute,” she explains. “My mum has also just been in hospital with my little brother (also a CF sufferer) for two weeks.”
Yet Kelsey and about 1,200 other children and adults are currently waiting on tenterhooks for the outcome of negotiations between the HSE and the manufacturer of the drug Orkambi, which has been helping the Irish people taking it through clinical trials.
Living with the condition is a constant struggle by anyone’s yardstick, but hope seemed to appear on the horizon with the advent of this trial by manufacturer Vertex. One participant was Brian McCarroll, 28, from Walkinstown.
“I was an in-patient in St Vincent’s when they told me I was one of eight people there picked for the trial,” Brian recalls. “I just remember tears of joy, and feeling so lucky. Everyone around me was delighted.”
Within six months, life had improved dramatically for McCarroll; his lung capacity, previously at 40pc at the outset of the trial, had increased to 72pc on Orkambi.
What’s more, CF Ireland have estimated that almost half of those living with CF can have the potential to significantly benefit from the drug.
“I reacted really well and everyone could see the changes right away. You still have to look after yourself a lot with diet and exercise as it’s not a cure, but it really does give you an extra boost,” explains Brian.
“It’s the effect it has on your mind, giving you the extra push to do things. When it comes to things like holidays and weddings, I don’t need to worry that in six months’ time I might not be able to go while I’m on the drug.”
Prof Stuart Elborn, a consultant in respiratory medicine, says: “The drugs work really well on people with two copies of the F508del mutation. Some people get a huge benefit, others a modest and some get none, but overall there’s a significant reduction in hospital admissions and some improvement in quality of life.”
Earlier this year, the National Centre for Pharmacoeconomics (NCPE) assessed the drug and didn’t deem it cost-effective. It currently costs about €159,000 per patient per year.
Then this weekend, disarming news broke in a Sunday newspaper: that the HSE ‘look set to reject the drug in the public health system’. The ‘Sunday Business Post’ reported that a formal announcement by the HSE was imminent after its drugs committee recommended against funding Orkambi at a recent meeting, noting that the drug was considered “unjustifiably expensive and not sufficiently cost effective” by both the NCPE and the HSE drugs committee. It was reported that the committee had concluded that the drug didn’t deliver sufficient benefits to patients to justify the hefty price tag.
Then yesterday, the HSE said that after “considerable engagement and negotiation with Vertex” it was “disappointed around the level of meaningful engagement by the manufacturer in lowering the exorbitant price of Orkambi for Irish patients”.
It said the HSE directorate “will then make a decision taking into consideration the manufacturer’s latest price offer”.
Vertex, too, have issued a statement saying they were “currently awaiting a response from the HSE on our latest proposals. We remain optimistic that a solution will be found that will allow all those who could potentially benefit from this medicine to do so and are fully committed to working with all parties to achieve this”.
So far, it seems as if there’s something of a stalemate. But while those receiving the drug will continue to do so until an agreement is reached with the Government, Ireland’s CF sufferers and their families still find themselves mired in an unnerving limbo. More than anything, they’d like to see the drug widely available and affordable to all CF sufferers.
Dubliner Keith McCabe is father to Senan, who turns three in March, and admits that recent developments (or lack thereof) have proved frustrating.
“I’m in a CF Facebook group, and we’ve been discussing this story,” he reveals. “There’s absolute despair, frustration, and anger. People are heartbroken. It’s the drug of hope, yet we find ourselves pawns in a negotiating game.
“There is a lot of frustration around the red tape. (Talks) have taken 25 weeks so far with no outcome… that’s six months a CF patient doesn’t have. And now it will hang over Christmas, and it’ll still be shrouded in uncertainty. There are not emotions that should be associated with the disease.
“Ultimately, I’d love to see it available through the long-term illness scheme,” he adds. “Why not give access to the drug to all patients on a trial basis, and see who sees benefits and improvements?
“(Minister Simon) Harris has talked about leveraging with ministers for health across Europe and negotiate as a euro ‘block’, but how long will that take?
“I just want my son to have a full long life, and a family of his own. I don't want a drug company put a noose around us,” adds Keith.
“When we heard Orkambi was here, it was like a new lease of life for us,” says Kelsey. “People are saying it’s so expensive, but I spend around 160-180 days a year in hospital. That’s about €180,000 a year just for the bed. We have a price tag put on our lives.
“If I had access to it, it could be a real game changer, and it would certainly mean that I wouldn’t have constant thoughts in my head like, ‘will I make it to graduation?’, ‘will I have a family?’ or ‘will I face a bad infection and be gone?’.
“I know people whose lives have been changed, and we all deserve that chance.”
For more information, contact CF Ireland on cfireland.ie or call 01 496 2433.