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Cystic Fibrosis

'Orkambi saved my life, other people should get access to it'

The drug, which costs about €160,000 per patient per year, is giving some people with cystic fibrosis a new lease of life.

JILLIAN MCNULTY HAS cystic fibrosis (CF). She has been taking Orkambi for over three years as part of a trial.

Earlier this year, the National Centre for Pharmacoeconomics (NCPE) assessed the drug and didn’t deem it cost-effective. It currently costs about €160,000 per patient per year.

The HSE is in talks with the drug’s manufacturer, Vertex Pharmaceuticals, in a bid to reduce the cost.

CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1,200 children and adults in Ireland and some 70,000 people worldwide. A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that:

  • clogs the lungs and leads to life-threatening lung infections;
  • obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.

CF Ireland has said that around 500 people with CF in this country have the potential to benefit from Orkambi.

Earlier this month, Philip Watt CEO of CF Ireland, said the Orkambi price negotiations are “still very active and have entered into a crucial third round of talks focussed around price”.

We understand these talks are at a delicate stage. As with all important negotiations the two parties should be given the space to get a successful outcome, but they should also be concluded as soon as possible to ease the anxiety of people with CF and their families.

“This negotiating space is important as a fair price for these drugs also increases the chances of future funding for other promising CF drugs that are currently in the pipeline.”

Jillian’s story 

Jillian knows the benefits of Orkambi first-hand, saying her health has improved dramatically since she began taking the medication in August 2013.

Prior to beginning the trial, Jillian says she would be in hospital “every four or five weeks religiously”, sometimes for up to 12 or 13 weeks at a time.

It wasn’t uncommon for her to spend eight or nine months of the year in hospital.

Jillian, who lives in Longford town, says her lung function had dropped to 39% prior to the trial. She was in hospital for several months and the possibility of her having a lung transplant was raised.

It was very, very scary … I was very upset. I don’t like the talk of that, there are so many complications and such a lack of donors.

Once she started taking Orkambi, Jillian says there was an initial improvement before she became quite ill and had to return to hospital.

“I believe now that was the drug working. When people start [taking it] the body purges massive amounts of mucus at an extra rate,” she says, describing her “constant infections” as “all the badness leaving my body”.

After a difficult few months, Jillian says “started to get better and better” and her visits to hospital became less frequent.

In 2015, she didn’t have to stay in hospital at all, something she describes as “absolutely amazing”.

IMG_8912 (1) Jillian climbed Croagh Patrick five weeks after major surgery on her stomach for reflux issues in 2014. She credits her quick recovery to Orkambi.

This year has been a more difficult one, with Jillian contracting swine flu and influenza A in March.

I truly believe if I wasn’t on Orkambi I would have died, it saved my life … I would be dead without it.

“On two occasions I know Orkambi has saved my life – when I had swine flu and when I was sick before starting the trial. I wouldn’t have lasted another year without it.”

This year doctors didn’t tell Jillian her lung function until after she improved. It had dropped to just 12% when she had swine flu.

“I would have been very distressed if I knew it was at 12%. I couldn’t walk or talk, I was on oxygen. They decided not to tell me.”

Jillian says doctors warned her not to expect her lung capacity to return to 40% for a year.

“Six weeks later it was at 48%, its highest in very long time. That in itself speaks for how well Orkambi has done for me.

“It has completely changed my life. I no longer live my life in a hospital. Before Orkambi I could never plan a week or two in advance, I knew I would get sick,” Jillian says, describing the difference the drug has made to her life as “just incredible”.

I used to dread every morning because I would have a coughing attack, as would a lot of people with CF. I’d lie in bed for 10 to 15 minutes to let it pass before I’d use the nebuliser. Now can get out of bed every day like a normal person. For someone with CF that is massive.

“Before, I was constantly sick – in hospital for weeks at a time, as soon as I got out of hospital a countdown to going back in started.”

Jillian says she has only been in hospital three times this year and the length of her stays have shortened too. She has to take two extra tablets in the morning and evening, with 50g of fat to absorb them.


Jillian doesn’t agree with the stance the drug is not cost effective. She recalls the time she was on a two-week course of intravenous antibiotics at home – at a cost of over €20,000.

She notes that many other CF patients have also had to avail of this at some stage, which quickly adds up, as does the money spent on hospital beds, scans and surgeries for people with CF.

Jillian says it is difficult for her to see friends with CF struggling because they can’t access Orkambi, while she is reaping its benefits, noting: “It’s so frustrating.”

I’ve seen the difference it has made to me. One person has said to me she was preparing herself mentally for my death I was doing so badly. When she said that to me I thought ‘Oh my god’, I didn’t realise how sick I was.

The woman, who has a daughter with CF, told Jillian she thought to herself, “When is Jill going to die? She’s going to die soon.”

Jillian, at the age of 40, jokingly refers to herself as “a geriatric CF patient”.

“To deny people with CF access to Orkambi is mind-boggling … Patients and parents contact me. They know that this drug has the potential to change their life. It is in front of them, but they can’t access it. It’s like they’re saying you’re not worth it … How much is a life worth?
We understand that it is important to get drugs at the best price possible and that there are other drugs they need to buy and we can’t just spend all the funds on Orkambi, but they have been negotiating this for 20 weeks. That is too long, lives have been lost.

Jillian says she hopes to stay on Orkambi indefinitely and avoid going down the route of having a transplant due to the complications this entails.

Jillian and other CF representatives met with Health Minister Simon Harris earlier this year to discuss Orkambi.


Speaking in Dáil about the situation recently, Harris said:

“The HSE has statutory responsibility for decisions on pricing and reimbursement of medicines under the community drugs schemes, in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. Prior to deciding whether to reimburse a medicine, the HSE considers a range of statutory criteria, including clinical need, cost-effectiveness and the resources available.

“The decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds by the HSE, on the advice of the National Centre for Pharmacoeconomics (NCPE)…

“Following a request from the HSE, the NCPE carried out an assessment of the manufacturer’s economic dossier submitted in March 2016 on the cost effectiveness of lumacaftor-ivacaftor (Orkambi). This dossier included details on all relevant costs and relevant cost offsets including hospitalisation, disease management costs, intravenous antibiotics, adverse events and any additional costs arising in patients not taking Orkambi.

The NCPE determined, following an evaluation of the economic dossier, that the manufacturer failed to demonstrate cost-effectiveness or value for money from using the drug.

“The HSE has since had further engagements with the manufacturer in an effort to secure significant price reductions for Orkambi. The HSE will consider the outcome of these engagements and any other, together with the NCPE recommendation, in making a final decision on reimbursement.”

Harris noted that decisions on pricing and reimbursement of pharmaceutical products within public health systems are “a national competence for EU member states”.

“However, the issue of pricing and reimbursement of medicines and patient access to affordable medicines has been addressed at European level. In June of this year, the EU Council adopted conclusions on strengthening the balance in the pharmaceutical systems in the EU and its member states.

Those conclusions invited member states to explore opportunities for cooperation on pricing and reimbursement of medicines and to identify areas for cooperation which could contribute to higher affordability and better access to medicines.

“I have indicated my support for these measures and I welcome the opportunity for member states to cooperate by sharing information so that we can achieve affordable and sustainable access to medicines.”

A demonstration at the Dáil is being planned for Monday 5 December at 1.30pm if negotiations on the cost of the drug have not concluded by then. More information can be found here.

Read: ‘I want to be a reliable friend. A better family member. I want to be present. I want Orkambi’

Read: “A travesty” – Cystic fibrosis sufferers angry over lack of funding for potentially life-changing drug

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