This follows its recommendation that the drug not be funded by the HSE at its current annual cost of €159,000 per patient.
However, Jillian McNulty, a CF campaigner who has been taking the drug for three years as part of a trial, says the health service would actually save money by funding the drug, even at the current cost.
“They are saying it is too expensive but last year, while on Orkambi, I did not have a single hospital admission whereas before being put on it my life revolved around hospitals,” said Ms McNulty. “At one stage, I was on an intravenous antibiotic at home for two weeks at a cost of €24,000. Without Orkambi, I could be in hospital for a minimum of six weeks every few months and taking three or four IV antbiotics. It does not take a genius to work out that putting me on Orkambi actually saved the HSE money.”
Michael Barry, head of the NCP, said yesterday he is hopeful that the HSE will be successful in its negotiations with Vertex Pharmaceuticals, the company that manufactures the drug, to significantly cut its cost.
Prof Barry said the NCPE has a good track record in talks with drugs companies.
“We don’t put a price on life, but we believe the manufacturers got the price wrong here,” he said. “The price is too high; we need a five-fold reduction.”
The NCPE said the drug would cost around €400,000 over five years, at the current price. As a result, it advised that the HSE should not cover the drug under State schemes and that the price would have be lower than €30,000 a year, per patient, to be cost-effective.
Philip Watt, CEO of Cystic Fibrosis Ireland, said CF patients in Ireland were dismayed by the ruling from the NCPE and he urged the Government to clarify that it will enter negotiations with Vertex to bring about a major price reduction.
Cystic Fibrosis Ireland welcomed the fact that the positive health impact of Orkambi is recognised by the NCPE.
“We support a fairer deal for this drug but it will be a travesty if this drug is not provided to our patients or if there is a significant delay in providing this drug,” said Mr Watt.
Ms McNulty said that Orkambi, which targets the cause of CF, had transformed her life and reduced greatly her hospital admissions.
For the past three years, Ms McNulty, from Longford, has been attending the research centre at St Vincent’s Hospital in Dublin where the drug trial is conducted.
“It allows me to live as normal a life is possible,” she said.
However, with just five weeks’ supply of the Orkambi in her possession, she is worried she may no longer have access to it.
“Even the research centre doesn’t seem to know if I and others on the trial will still be able to get Orkambi. We are in a limbo situation.”
‘It allows me to live a more normal life’
Life for Jillian McNulty, a cystic fibrosis (CF) survivor, has revolved around hospitals for the past nine years when she became very ill as a result of the hereditary condition.
Her brother Derek died from CF at the age of five, before she was born, but even though she also had the disease, it did not affect her until well into adulthood:
“I only started to get really ill at the age of 31,” she says. “Up to then, I was pretty healthy which is unusual for someone with CF.”
2005 was Jillian’s annus horribilis. She was made redundant from her job at a radio station in Longford, having worked there for 12 years.
Within weeks, CF struck with a vengeance.
“Imagine living your life knowing that, no matter how well you look after yourself, no matter how compliant you are, that the disease that lives inside your body is slowly but surely killing you,” she says.
“Imagine waking up at night almost drowning in your own blood that’s spouting from your lungs or imagine waking up choking and coughing so hard you almost black out.”
From then until starting a trial of the groundbreaking drug Okambi, she would spend at least six weeks in hospital every few months, on intravenous antibiotics. Overall, she spent up to nine months in hospital every year.
Okambi transformed her life and reduced her hospital admissions.
“My lung functions had dropped from 59% to 39% the day that I started taking Okambi,” says Jillian. “It doesn’t work for everyone but it did for me and it allows me to live a more normal life.”
Jillian, from Longford, has been taking the drug for three years as part of a trial at St Vincent’s Hospital research centre and she has no doubt that Okambi can be a lifesaver for some people with CF.
“I started on Okambi in July 2013,” she says. “Within a few days, I could sense small things changing. My body temperature started to drop. There were other differences, too. Before taking it, on hot days my body would produce salt on my hands and face. It was awful, like sand. But that has all gone now, even when I go out running, which I love.”
The big changes came after about six months when Jillian began to feel well again and last year, for the first time in eight years, she didn’t have a single hospital admission: “That was incredible.”
But she has not been so lucky of late as she contracted a double dose of swine flu and influenza A.
Her 40th birthday, on May 3 of this year, was spent in St Vincent’s Hospital and she recalls the bitter-sweet celebration.
“I woke up physically drained, exhausted and weak but emotionally I was extremely happy as I had defied the odds,” she says. “I had shown CF what I thought of it by reaching 40 years old. The staff here in St Vincent’s were amazing, they decorated my room in birthday banners, balloons and my favourite colour — pink. The nurses and staff barged in singing happy birthday to me with a fabulous birthday cake.”
She has since recovered but is worried she may no longer have access to Okambi. The decision not to fund it was a bitter blow.
“I am devastated that my life and the lives of up to 60% of CF patients in Ireland means so little,” says Jillian.