Patients devastated by HSE decision not to fund treatment for rare genetic disease
Patients suffering from a rare genetic disease say they are devastated that the HSE has decided not to fund a new treatment for the condition.
The decision affects 60 patients who suffer from Alpha 1 - a genetic form of Emphysema.
The Alpha One Foundation is urging the Minister for Health Simon Harris to intervene.
They say the drug called Respreeza is the only therapy approved to treat the condition.
Of the 60 patients, 21 have been able to access a "compassionate-use" scheme operated by the drug manufacturer, CSL Behring, due mainly to their participation in a clinical trial.
However, the company has previously said that supplying it on a compassionate basis indefinitely is not an option.
Professor Gerry McElvaney, the Consultant Respiratory Physician involved in the clinical trial of Respreeza in Ireland, and Chair of the Alpha One Foundation, said the drug approvals process is not working.
He said: "The drug Respreeza has been shown to be effective in slowing progression of emphysema. The only question is one of cost. It is absolutely clear that those adjudicating on this matter are not in a position to comment or to make a decision on the efficacy of the drug.
"Respreeza definitely slows the progress of emphysema. All of the 21 Irish patients have experienced an improvement in the quality of their lives. No Irish patient died during the study and its extension, a time period of over 12 years. If the European Medicines Agency and 12 countries in Europe agree the drug works and is effective.
"Why is the HSE saying it isn’t? The longer patients are not receiving this drug the more lung damage they will suffer. This lung damage cannot be reversed."
