New Cystic Fibrosis drug was 'like winning Lotto' for Jillian

A cystic fibrosis (CF) sufferer whose life has been transformed by a breakthrough drug known as Orkambi is calling on the Government and the HSE to fund it.

Jillian McNulty, 39, from Longford Town, had access to the drug on a two-year trial basis and said that she went from having severely reduced lung function, no energy, and a need for regular daily three-to-four-hour naps, to living a normal and full life.

“I have not been in hospital for 11 months now. Prior to Orkambi, my average hospital stay was six to seven weeks, with one stay lasting 13-and-a-half weeks. Between the ages of 31 and 37, I really started to go downhill and was in and out of hospital all the time. Things were getting pretty bleak and I was getting closer and closer to transplant.

“Then I got on the trial at St Vincent’s University Hospital and it was like winning the Lotto. I never thought Orkambi could have transformed my life to the extent that it has. I can plan months in advance now, I feel normal, I don’t need naps. The thought that the drug might be withdrawn is utterly devastating.”

She has written to the National Pharmacoeconomic Centre (NPCE), which assesses both the effectiveness and cost-effectiveness of a drug before making a recommendation on whether the State should fund it.

“I wrote to them asking why my life and the life of other CF patients should have a price tag,” she said.

She was motivated by a change in her own condition as recently as last week after her involvement in the Orkambi trial ended.

Jillian McNulty

Under an “open access” programme, Vertex, the manufacturer of Orkambi, will continue to supply her with the drug until the Government makes its decision on whether to fund it.

However there was a gap between finishing the trial and receiving more drugs through “open access” and her condition deteriorated.

Jillian said: “I know I will have it within a few more days so I will be OK. But for those who haven’t even had the opportunity to give this drug a try, the wait for them is agonising while the battle for recommendation/reimbursement continues.”

Yesterday, junior health minister Kathleen Lynch said she could not say if the drug will be funded — it is estimated to cost €160,000 per patient per annum and approximately 600 patients could benefit. The HSE has already said there is not enough money to pay for it.

Ms Lynch said Orkambi was undergoing a process with the NPCE and “has to jump several hurdles”. She said she would not offer hope to CF sufferers that it would be made available.

“I don’t make promises that I cannot fulfill myself,” she said. “It’s going through the process in the same way as the first drug [Kalydeco, another CF game-changer that the HSE funded, despite a NPCE recommendation to the contrary] went through it, and when that process is finished, both myself and Health Minister Leo Varadkar will get a report on it.”

Ms Lynch was at the official opening at Cork University Hospital of a 20-bed adult respiratory and CF ward paid for at a cost of €2.3m by the charity Build4Life.