Humanity is not the perfect species, there is no promise of healthy perfect babies, but few people become pregnant with the expectation of having a child with a life altering disease. Cystic Fibrosis is a genetic disease, and Ireland has the highest instance of CF sufferers in the world, and most couples don’t know a thing until they are presented with a ‘Salty Baby’.
CF affects the cells that produce mucus, digestive enzymes and sweat, hence ‘Salty Baby’. Instead of acting as a lubricant as in a non CF person, the mucus clogs pathways, predominantly in the lungs and pancreas. Nebulizers can help lungs to breathe more easily, and drugs, physio and enzymes do their part, but it is never enough. Some children are fed through a tube; others may have CF related diabetes, or collapsed veins from constant intravenous drips.
There is hope; it sits mired beneath a cloud of financial negotiations which ultimately determine; how to save a life. That hope is Orkambi.
Orkambi is a twofold drug, a combination of a corrector which fixes the broken protein and transports it to the cell and the chaperone which opens it at the cell wall to allow sodium and chloride to pass through. This means that the mucus produced by the mucus membrane organs (lungs, pancreas, intestines, etc.) is thinner and can be cleared from the lungs.
This drug was approved by The European Medicines Agency (EMA) for Irish users, however, the department of health has not cleared it, due to cost. #YesOrkambi has entered its 18th week; yet Minister for Health Simon Harris has still not divulged how price negotiations are going.
While the campaign has remained strong, a lack of ministerial response was too much for one mother, who this week penned an open letter to minister Harris, while waiting for her 6 year old daughter to have a port inserted in her chest.
“You’re the Minister for Health so I know I don’t have to explain to you that the reason for this procedure is to allow doctors gain direct access for IV antibiotics, instead of struggling to find lines in her little hands all the time.” She wrote.
Marisa Reidy’s daughter Hanna is only 6, but her life could alter exponentially once the EMA approve Orkambi for the under 12s. In her letter, Marissa explained how her daughter was extremely sore after the operation and asked her mum, through her tears, why she had to undergo this procedure.
“I said I would write and ask.” Marissa said, also telling the minister: “I appreciate you’re currently extremely busy trying to arrange automatic medical cards for all our prisoners – which I’ve no doubt is a time-consuming job – but when you’ve finished securing the future health care needs of our murderers, rapists, pedophiles and drug dealers, I wonder if you might get a chance to concentrate on the hundreds of Cystic Fibrosis patients who are desperately waiting for approval of life-saving drug, Orkambi.”
Orkambi in its current approval could directly impact approximately 500 patients in Ireland, as it has been approved in Europe for over 12s only, however, the Federal Drug Authority in America has now approved Orkambi for over 6s. The process to have it approved by the EMA should take less than 6 months.
So how much does it cost to save a life?
Currently the cost per patient for treatment with Orkambi would be in the region of €160,000 per year, a monumental amount, but considering Ireland has 500 CF sufferers who this drug can treat, and more under the age of 12, the drug company Vertex have little to worry about in relation to an investment return, almost regardless of what the final agreed price is.
Quantifying the costs to the Irish health system in the 18 weeks of negotiations on Orkambi would be difficult.
Eighteen weeks is a very long time in the life of a CF patient, Eighteen weeks can reduce lung function dramatically, and eighteen weeks can be an actual death sentence.
Hanna’s mammy says we must personalize the reality of life with CF so those in power can see that it can’t be about money: “These are real people, adults and children, whose health and future they are playing with.”
And as for what Hanna thinks? “She’s totally oblivious. She’s too young to have to worry about all that yet. She knows she has CF and needs meds etc., and that’s enough for now.”
Hanna was a Salty baby, and Orkambi could one day save her life. #YesOrkambi
Orla O Driscoll
Photo credit: Simon Burch