More than 20 Irish patients with severe lung disease are to lose access to a “life-changing” drug within weeks after the manufacturer decided to end a long-running free trial.
The patients, who have genetic emphysema, are due to see their use of Respreeza - provided through a compassionate use programme - finish at the end of this month, the deadline set by the manufacturer, CSL Behring.
An application to have Respreeza, which costs more than €100,000 per patient each year, provided for free in the Irish health service was rejected by the HSE last month.
The drug is the only treatment that slows the progression of the condition by decreasing the frequency and severity of chest infections and associated hospital admissions.
The patients on Respreeza have alpha 1 antitrypsin deficiency - also known as alpha-1 - a hereditary condition that is unusually common in Ireland.
Many were put on clinical trials from 2006 and, when these ended in 2010, a compassionate use programme.
The company has repeatedly threatened to end the programme but has delayed the execution of this threat up to now. However, patients were informed on Friday at a meeting with doctors in Beaumont Hospital that the company will stop providing access to the drug free of charge.
Many of the patients involved have been on the therapy for more than ten years and have no other therapy option, according to the Alpha One Foundation, which runs a HSE-funded screening programme for the condition in the hospital. An additional 40 patients who were diagnosed after the trial ended are waiting to be put on what the foundation says is a "life-changing" therapy.
"Last month we learned of the decision of the HSE not to fund the therapy, which was a devastating blow to the patients affected," said Geraldine Kelly, chief executive of Alpha One. "Today, we see the fallout of that position with the decision by CSL Behring to stop the therapy for the 21 people who have been accessing it to date."
"Both decisions are unconscionable. We have a therapy that works and it is wrong of the HSE and the Department of Health not to fund it. 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."
“Patients are distraught. They have been through the most anxious time over past months, where they have been living under a constant shadow of the withdrawal of this therapy. Now, it would seem, their worst nightmare is coming true. While structures and supports are necessarily being put in place by medical staff at Beaumont Hospital to monitor patients’ health when they come off this therapy, the Alpha One Foundation still hopes these steps will not be necessary.
She urged Minister for health Simon Harris to intervene and for both the HSE and the company to "pull back from the brink".