Reilly urged to approve costly lung disease drug

Minister for Health James Reilly has been urged to reconsider the decision of the National Centre for Pharmacoeconomics (NCPE) not to make available the drug pirfenidone to sufferers of a rare form of lung disease.

The centre says it cannot recommend approval of the drug to treat idiopathic pulmonary fibrosis (IPF) because of its high cost (€32,700 per patient per year), the significant impact it would have on the health budget and the absence of long-term clinical data on its effectiveness.

However, the Irish Lung Fibrosis Association has called on the Government to approve funding for pirfenidone, which it says is the first and only drug proven to prolong life expectancy in patients with IPF.

The association says that without access to pirfenidone, patients with IPF may not live long enough to receive a lung transplant. The drug also offers a lifeline to IPF patients who are not eligible for a transplant, by interrupting the production of cells and molecules involved in the formation of scar tissue in the lungs and improving progression-free survival, it says.

"It would be regrettable if this therapy was not available to Irish patients, given the serious nature of the condition, and the fact that it is available in other countries including the UK," said Prof Jim Egan, a leading international expert in IPF and a consultant respiratory physician at the Mater hospital.

Threshold cost
The number of patients anticipated to receive the drug is 97 in year one, rising to 207 after five years.The cost of pirfenidone is up to three times the threshold set by the NCPE for approving reimbursement under the medical card scheme.

The centre acknowledged it has been shown to have a “modest but measurable” effect on slowing decline in lung function in mild to moderate IPF.

“Whilst pirfenidone may represent an important treatment option for patients with IPD, there are significant uncertainties, including the absence of long-term health outcome data.”

Fianna Fáil says Dr Reilly should intervene in the same manner as he did with cystic fibrosis drug Kalydeco, rejected by the NCPE on cost-effectiveness grounds. Dr Reilly has ordered his officials to approve Kalydeco, which will cost €220 million over 10 years.

Fibrosis, the development of scar tissue in the lungs, causes the lungs to become stiff, preventing the effective transfer of oxygen from the blood to the lungs. IPF is more common in men than women, between 60-80 years of age. Patients develop extreme breathlessness, fatigue, coughs and ultimately respiratory failure. This requires those living with IPF to rely heavily on oxygen therapy 24 hours a day. There are no drugs to cure IPF.