'Nobody told us at all' - CF community devastated HSE won't fund 'game changing' drug
The Cystic Fibrosis (CF) community have expressed their “devastation” following a HSE decision to reject a “game-changing” drug that could dramatically improve and extend their lives.
CF sufferers say the Orkambi drug has the potential to give patients a better quality of life, along with an increased life-expectancy rate.
However, the HSE have recently decided that the Orkambi drug will not deliver sufficient benefits to patients to justify its €159,000 annual price.
A spokesperson told Independent.ie: “To date the drug has been considered unjustifiably expensive and not sufficiently cost effective by both the National Centre for Pharmacoeconomics (NCPE) and the HSE Drugs Committee.”
The decision was met with shock and disappointment within the CF community who have been continuously pressuring the government to introduce the drug in Ireland.
Siobhan Brophy, whose nine-year-old daughter Saoirse suffers from CF, said she is devastated by the news.
“Nobody told us at all. The whole CF community are extremely disappointed that we had to find this out through the media instead of the government delivering the news to us personally.
“With CF you end up with a sticky mucus that clogs up your pancreas and blocks your lungs which causes all kinds of infections.
“What Orkambi does is break down the sticky mucus that clogs up your pancreas and blocks your lungs. It hits the problem head on as opposed to just papering over it like the other drugs.
“That’s why it’s such a game changer. It would mean that people living with CF won’t be in hospital as much or miss as much school. They will also be able to finish their college courses and also hold down a full time job.”
Linda Whitmarsh, whose two-year-old son Finn has CF, told Independent.ie that the Orkambi drug was the only hope she and her family had.
“Every time I go to a funeral I wonder about my son’s fate and every time I go to a wedding I wonder if he’s ever going to get married and have a normal life.
“Orkambi was a silver bullet for the CF community. It wasn’t a cure but it was the only hope we had."
“Finn was diagnosed with CF when he was two weeks old and it was an absolutely devastating time for us all. In the last two and a half years he has attended hospital 38 times - everyday is a battle for him. We know that without Orkambi Finn will be lucky to live until his 30s."
Cystic Fibrosis Ireland has vowed to fight the decision.
In a statement, released on Sunday afternoon, CEO Philip Watt said: "Cystic Fibrosis Ireland is angry at the cynical way the HSE has conveyed this information to our patients, some of whom are very ill.
"The decision was leaked to a Sunday newspaper even before the Minister was informed, never mind patients. It’s a disgraceful and heartless form of communication."
A HSE spokesperson said it 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.
"To date the drug has been considered unjustifiably expensive and not sufficiently cost effective by both the National Centre for Pharmacoeconomics (NCPE) and the HSE Drugs Committee.
"The HSE is disappointed around the level of meaningful engagement by the manufacturer (Vertex) in lowering the exorbitant price of Orkambi for Irish patients (and indeed equally for CF patients in the UK, Canada, Scotland and Australia).
"The HSE has recently completed its most recent round of negotiations with Vertex. On foot of this, the HSE’s Drug’s Committee (together with a NCPE recommendation and other expert advice) is in the process of sending its latest recommendation on the cost-effectiveness of Orkambi to the HSE Directorate.
"The HSE Directorate will then make a decision taking into consideration the manufacturers latest price offer," said the spokesperson.
A number of members from Cystic Fibrosis Ireland have been invited to the AV Room in Leinster House on December 1 to voice their opinions on the Orkambi drug in front of a panel of TDs.