Decision pending on lung drug

The availability to Irish patients of the only known drug to treat the underlying cause, and not just the symptoms, of genetic emphysema currently hangs in the balance.

The National Centre for Pharmacoeconomics (NCPE), which decides whether a drug is cost- effective, is in the “advanced stages” of evaluating whether or not to recommend that the State fund Respreeza, a drug shown to slow the progression of emphysema and to reduce severity of chest infections in patients and associated hospital admissions.

Respreeza, available to 21 Irish patients who took part in a clinical trial between 2006 and 2014, is for the treatment of alpha-1 antitrypsin deficiency (AATD) or Alpha-1. AAT is a protein whose main function is to protect the lungs from infection and a deficiency can cause symptoms ranging from breathlessness and wheezing to severe lung, liver and skin problems. Respreeza works by replacing the protein that is lacking.

Dr Tomás Carroll, chief scientist with the Alpha-1 Foundation, said patients were anxiously awaiting a decision from the NCPE and that they had been told in a recent communication that the drug was “in the advanced stages of evaluation”.

“We are hopeful the 21 people on the drug will stay on it and that another 40 will fit the criteria,” Dr Carroll said.

A survey of the 21 patients showed a significant reduction, 68%, in chest infections in the 12 months after, compared to the 12 months prior to taking the drug.

Dr Carroll said the cost of the drug in other countries was roughly €60,000 per annum per patient and that they anticipated the cost here would probably be up to €80,000 a year per patient.

However it was life-changing for patients, he said. Alpha-1 is the most common genetic lung disorder in Ireland and there are around 350 people here who have been diagnosed with severe Alpha-1. Dr Carroll said the decision will be critical for patients currently on Respreeza, and also for those missing out on its benefits.