Opinion:Imagine for a moment you're being dunked under water and that water is flooding into your lungs. Every area of your insides ache. Your lungs ache, your wasted muscles ache, your congested digestive system aches. Then it's calm again. You're back on the shore and beach goers are playing ball, sipping cocktails in the sand and carrying on as usual.
This analogy is a frequent, graphic description of cystic fibrosis (CF). As I get older, at times this tired analogy can be pretty accurate. There can be daily pain, exhaustion and discomfort. There’s the reality that one minute you’re standing calm and then you’re dragged back into a drowning feeling that constricts your lungs. You get tossed in and out of either scenario whenever the illness chooses. Your lungs get inflamed, they get infected, they bleed, they collapse, you get better.
After a while it starts again.
Now imagine, in your lifetime of this, someone tells you they have found a tiny tablet that can change this pattern. That it works for 4 per cent of the 600,000 people worldwide who are affected by CF. That trials are ongoing to sort out the rest of the population. That this, the closest thing to a cure since the CF gene was found in 1989, has been discovered.
Trial results come in and, watching from the outside, the narrative rolls like a movie. Except it’s real.
A friend in the US says after several days popping this tablet she can take a deep breath. A deep breath. On long cycles she doesn’t sweat profusely like before, as people with CF can do due to the problem with salt regulation in the body.
Then there are the stories that bring stinging tears. There are people in Ireland, considered extreme cases, who have received this drug under compassionate terms.
They’ve taken this little blue pill twice a day and some have been taken off transplant lists. This means they were sitting on a list waiting for someone else to die so they could live. This means they now are too well to be in this position. Lung function improves, weight improves, quality of life improves.
The cost of Kalydeco is estimated at $294,000 for one year’s supply per person. Within hours of Kalydeco’s positive results being released shares in the drug company skyrocketed.
This drug is the first to treat the genetic cause of a certain mutation of CF; it is the treatment my father always told me was coming. The type of treatment I smiled at him about when he mentioned it; but I didn’t hold out any great hope for such revolutionary medicine in my lifetime when we couldn’t even get basic facilities straight in this country.
Now Kalydeco has opened the door for a new trial combination of it and another new drug. If the final stage of trials on the new combination drug is successful, 90 per cent of the CF population – including me – would be suitable to try a treatment that could radically change the course of our lives.
In Ireland, Kalydeco’s cost effectiveness is being assessed by the National Centre for Pharmaeconomics. A decision is expected in the next 10 days. This recommendation would then lead to negotiations between the drug company and the HSE surrounding cost. The Government will want best value for money; the drug company will know that the need is great. There is an option of co-shared risk where the company and HSE share the cost – the drug is given and if it doesn’t work the HSE doesn’t pay out.
There had been hope the drug company would view Ireland as a special case given it has the highest prevalence of CF in the world and the gene that Kalydeco treats known as “the Celtic gene”. In fact 10 per cent of those who can benefit from Kalydeco live in Ireland.
Just before Christmas, activists in the United Kingdom celebrated Kalydeco’s accessibility in England. Negotiations for Northern Ireland, Wales and Scotland are ongoing. In the US, the little blue pill is widely available.
In the UK negotiation details are kept secret, but it has been speculated that up to 20 per cent has been knocked off the original price.
But what price can you put on a drug that has moved people from the margins of death back to life? The unmistakable reality of its effects are worth more than any money. We need access.
* Orla Tinsley is a freelance journalist and campaigner for better cystic fibrosis treatment and care facilities