Lung disease sufferers to lose vital drug access after pricing talks fail
Twenty-one patients with severe lung disease have been told they will lose access to a vital drug at the end of the month after health chiefs failed to strike a price deal with the manufacturer.
One of the Alpha 1 antitrypsin deficiency (AATD) sufferers, father-of-two Gerry Finnerty, who has spent years on the specialist intravenous treatment Respreeza, asked the Health Service Executive (HSE) where their compassion was.
Respreeza is the only therapy in a clinical study to slow the progression of lung disease caused by the genetic condition.
"I don't know if I'd actually be alive without it," Mr Finnerty said.
"I probably would have had to go for a lung transplant and then the problem is whether it would work. That's my honest opinion."
Most of the group have been using Respreeza therapy, made by German pharmaceutical CSL Behring, on a "compassionate" basis for several years after going through a clinical trial.
Their doctor in Beaumont Hospital in Dublin, Gerry McElvaney, Professor of Medicine at Royal College of Surgeons in Ireland (RCSI), told them the drug would be stopped on September 30 after the HSE and manufacturer failed to agree a deal on price.
It is estimated to cost about 85,000 euro per patient per year.
CSL Behring said it wanted a different outcome and was deeply disappointed.
"While we had hoped for a different conclusion, we remain committed to AATD research to improve the quality of life of people living with the condition," the company said.
It said it was looking at a new clinical trial across Europe, including Ireland, where severe Alpha 1 patients would be given the drug.
"We worked tirelessly to identify and agree on a solution that would work for both patients and for the HSE and give Respreeza the best chance of gaining reimbursement," CSL Behring said.
Mr Finnerty, 69, from Kinsealy, Co Dublin, said he had one question for those who determine whether he gets the drug: "Where is the compassion?"
The HSE decided last month it would not fund the drug.
On Thursday, the agency also revealed its budget overruns for this year were soaring to more than 300 million euro.
Mr Finnerty added: "My wife Helen feels very sorry for me, very upset.
"She just feels: 'How can you put a price on my husband's life?' And that's what it is coming down to."
The Alpha One Foundation said 60 patients could benefit from the drug if the HSE could secure a price deal with CSL Behring.
Thousands of other people in Ireland are thought to be living with the condition with varying degrees of severity.
Geraldine Kelly, the foundation's chief executive, appealed for health chiefs to make the necessary money available and for drug maker CSL Behring to cut its price.
"We have a therapy that works and that has been proven effective, and it is wrong of the HSE and the Department of Health not to fund it," she said.
"Equally, it is wrong for a company to discontinue a therapy that works to patients who have provided it with a rich resource of research information that has undoubtedly been of huge commercial benefit."
The HSE said: "The HSE is very much aware that this decision is upsetting to patients who are affected by this condition.
"It will also be a disappointment to their families and the treating clinicians who support these patients."
But it added that its research found Respreeza did not result in a significant improvement in quality of life.
The HSE said it has an onus to ensure that any new drug is cost effective.
It said: "The HSE has to have regard to its wider obligations to the 4.7 million population it serves and needs to maintain the full range of health services to all of the other patient groups within the finite resources at its disposal."