THIS TIME LAST year, things looked a lot different for cystic fibrosis sufferers in Ireland.
Orkambi, a “life-changing” drug, was not available to patients through the HSE. Around 57% of the 1,300 CF patients in Ireland would benefit from the medicine, which helps to boost lung function and increase overall quality of life for CF sufferers.
Ireland is the country with the highest per-capita rate of cystic fibrosis, or CF, a fatal, genetic disease that causes severe problems with the lungs and digestive system.
The huge cost of Orkambi – put at €158,000 per year per patient, at a cost to the State of €390 million over the course of five years – led to protracted negotiations between the government and the makers of the drug Vertex Pharmaceuticals, and there were fears that no deal would be reached.
Candlelit vigils were held. Protests were organised. Impassioned arguments were made calling for this drug to be made available.
Fast forward a year, and we are already over eight months into Orkambi being available to patients through the HSE and more good news came yesterday when European authorities decided that the drug could be licensed to patients between the ages of six and 11.
Breakthrough
Cystic Fibrosis Ireland CEO Philip Watt told TheJournal.ie that yesterday’s announcement was tremendous news for the young cohort of patients in Ireland who have cystic fibrosis.
“Things have turned around so much for us in the past 12 months,” he said. “We’ve come so far, it’s fantastic.”
He said that around 50 children between the ages of six and 11 would benefit immediately when the drug became available, and stressed how beneficial it can be for CF patients to get this drug as early as possible.
Watt said: “Going on this drug at an early age stops the disease from progressing.
For people who start on it later, it won’t reverse the progression of the disease. It’ll slow it and stop it. The earlier people are on it the better.
He said that Orkambi can be used to treat most of the people who have cystic fibrosis, and that yesterday’s European approval meant that it was “a really positive time for CF” in Ireland.
Rapid access
Previously, it took years between the European approval for Orkambi in patients aged 12 and over and then patients in Ireland being able to access it.
For a drug to be licensed and sold in Ireland, it must first get European approval. But even with this approval, the maker of the medicine must then reach an arrangement with the government and HSE on how much the drug will cost.
In the case of Orkambi, its high price made the negotiations drag out for a long time.
This time around, however, the process will be made much simpler.
As part of the agreement made between Vertex and the government, a condition was made that the deal would cover access to the drug for children between the ages of six and 11 once European authorities gave the go ahead.
In a statement, Vertex said its existing arrangement in Ireland “will enable rapid access to Orkambi for these children”.
Minister for Health Simon Harris sent a tweet welcoming the news and said that children would be able to access the drug “very soon”.
In a statement to TheJournal.ie, the HSE said it would be “putting together the necessary reimbursement arrangements over the coming weeks”.
It is understood that the children who will benefit from Orkambi will begin to receive from as early as the start of February.
Watt added: “The HSE moved very quickly to reassure that the deal would be honoured.
There was massive anxiety even a year ago. This is great news. I’d like to acknowledge and thank the government. So many children and families will benefit from this.
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