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Twenty-one Irish patients suffering with genetic emphysema, a severe lung disease, have been given a month’s extension on what the Alpha One Foundation, the advocacy and representative body for these patients, have described as a “life-changing” drug treatment.

The patients suffering with Alpha-1 antitrypsin deficiency were placed on a trial between 2006 and 2010. The compassionate use programme, which had been supplying the patients with the Respreeza drug since, was due to end this week. The manufacturer, CSL Behring, have repeatedly claimed that it would put an end to the compassionate use programme but have since changed the deadline to the end of February.

The RAPID study conducted on the drug featured 180 Alpha-1 patients in 13 countries worldwide, supervised by 29 principal investigators.

Published in The Lancet in July 2015, the study conclusively found that Alpha-1 patients treated with Respreeza saw a significant slowing down by 34 per cent in the progression of lung disease (emphysema), as measured by CT scan. As a result of these findings, the therapy was licensed by the European Medicines Agency in July 2015.

By slowing the progress of this lung condition, Respreeza minimises the severity and frequency of chest infections and therefore hospital admissions.

The drug costs over €100,000 for each patient and funding for reimbursement was rejected by the National Centre for Pharmacoeconomics (NCPE) in early December.

The Minister for Health has previously stated that it was his understanding that the NCPE would meet with the Alpha One Foundation in relation to this matter but, although Alpha One representatives have been “at the ready”, a meeting has yet to be arranged with either the NCPE or directly with the New Drugs Group.

The foundation has stated it is critical that they receive an opportunity to respond to the NCPE decision and make a case on behalf of patients. They believe that no final decision should be made by the HSE New Drugs Group before it has the opportunity to gain the patient’s perspective.

CEO of the Foundation, Kitty O’Connor, admitted that it was frustrating to be dealing with closed doors, explaining that it has been “difficult to get information” regarding the HSE New Drugs Group meeting, which they believe took place on Thursday 26th January. Forty more patients who were diagnosed since the end of the trial are waiting to begin taking the drug once it has been approved.

The Alpha One Foundation is calling on the HSE to acknowledge the benefits of this therapy and for both the HSE and CSL Behring to come to an agreement as to a price.