Twenty-one patients have been getting Respreeza from its manufacturers on compassionate grounds — some for as long as 10 years — after they participated in clinical trials of it before it was formally approved for use.
However, health chiefs here failed to agree a price for the drug with the US manufacturer, CSL Behring, and the company is to stop providing it for free from the end of this month.
Johnny Hannan, from Mallow, Co Cork, who has the Alpha-1 condition that Respreeza keeps in check, is adamant he would not be alive today without it.
“I started a trial of Respreeza nearly 12 years ago and I’m here today because of it,” he said. “None of us know what the effect will be when we come off it. Will we deteriorate slowly or will we go down hill rapidly? We just don’t know. It’s like waiting on death row.”
Mr Hannan and the other patients have the inherited condition Alpha-1 which usually results in emphysema, leaving people with severe breathing difficulties and very vulnerable to serious chest infections.
Respreeza keeps the condition in check and is being prescribed by health authorities in 12 other EU countries. The HSE and Department of Health declared the asking price — €84,000 per patient per year — too high after a study last year.
CSL Behring was given time to make representations in response but the HSE said yesterday it had considered CSL’s submissions and reached the conclusion that the drug was not sufficiently effective to warrant the expense.
In a statement, it said: “We are very much aware that this decision is upsetting to the Alpha One Foundation and patients who are affected by this condition. It will also be a disappointment to their families and the treating clinicians who support affected patients.
“We have to consider our wider obligations to the 4.7m population we serve and need to maintain a full range of health services within the finite resources at our disposal.”
Mr Hannan said patients deserved better.
“It’s been torture for the last year, getting the drug month to month and not knowing what happens next,” he said. “We are absolutely devastated that, after all we’ve been through, they’re saying no more. We feel let down by the HSE and by the country as a whole.”
Geraldine Kelly, CEO of the Alpha One Foundation, said the group would not give up.
“Greece, which has no money, provides this drug to its patients,” she said. “Spain and Portugal too, despite their financial problems. We want the HSE to explore an EU-wide deal with CSL. We’ve only a small number of patients here but the numbers combined across Europe would surely bring the price down.”
CSL Behring said it was "deeply disappointed" with the decision. In a statement the company said: "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 in the Republic of Ireland.
"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. Based on this commitment to patients, we are evaluating the opportunity of having patients participate in future trials."