A Ballinakill mother of three is facing an uncertain future, if she does not get life-saving treatment for her rare blood disorder.
Mary Gorman was diagnosed an ultra rare and life threatening blood disorder, Paroxysmal Nocturnal Haemoglobinuria (PNH) in October 2012.
In January 2013, Mary’s consultant prescribed her Ecluzimab - the only drug that can help her condition but to date she has not received the medication.
The cost for the drug is approximately €437,000 per patient per year.
Without treatment, PNH has a life expectancy of just 5-10 years.
Mary and her family claim that the HSE and the Minister for Health have denied her access to treatment for the last 22 months by not funding it for her.
The only way that Mary has of managing her condition is to have regular blood transfusions. As her condition worsens she needs more regular transfusions, she needs them every four to five weeks now.
“She gets two good weeks now before she starts to go down hill again after a transfusion. The blood transfusions are like topping up a sieve,” Mary’s sister, Catherine O’Connor said on her behalf.
“This drug is the only thing that can control the leakage.”
“We have no answer from the HSE, they haven’t said yes or no. We are getting nothing from them.”
Mary has already suffered acute kidney damage since being diagnosed and has been forced into early retirement from her work as a physiotherapist with the HSE.
Catherine said: “The sickness is hard enough. But the trauma of having to fight for treatment is starting to take its toll also.”
“They have chosen to let me become disabled and / or die instead of giving me a chance to live a reasonably normal life,” Mary recently wrote.
“I am slowly but surely dying without treatment. Transfusions are not a treatment for PNH. They are the only means of keeping me alive while they remain effective.
A statement from the HSE said that Eculizumab is one of the most expensive medicines in the world and is not approved for reimbursement in Ireland.
The HSE said they are discussions are ongoing with Alexion, the manufacturer, to try to agree a mutually acceptable successor agreement to the 2010 Pilot ‘Access with Evidence’ Programme, which 10 patients participated in. They all remain on the pilot project.