Hannah Lindley, from Leeds, has slammed health body NICE after it released a draft guidance recommending the drug Orkambi has its funding cut.
The 20-year-old and her younger sister Iona were both diagnosed with the illness as babies, but Iona’s condition deteriorated far more quickly and she passed away in November.
Hannah was horrified in February when NICE advised that as the drug costs £104,000 a year per patient and only provides a ‘modest’ benefit, it is no longer cost-effective.
“When Orkambi was first available, everyone called it a wonder drug and a medical breakthrough. It was making a huge difference to people, so I can’t understand how it’s now in the process of being cut. It’s like being handed a death sentence,” Hannah said.
Hannah’s lung function is at 55 per cent and she says it’s only a matter of time before it gets worse.
As a baby, Hannah experienced jaundice, coughing and wheezing and was unable to gain weight, before finally being diagnosed at 14 months old.
Her younger sister Iona had just been born and was immediately discovered to be a sufferer. Despite the early diagnosis, her condition declined more quickly.
The sisters spent their childhood going to hospital appointments together and having nebuliser treatment each morning before school.
Tragically, Iona continued to rapidly lose lung function and died in November last year.
“Me and Iona were inseparable,” Hannah reflected. “Mum always referred to us as twins, as we were so close in age, our facial features and personalities.
“We had the same friends, wore the same clothes and shared everything. When we were well enough we did ballet, tap and piano lessons together.
“Losing her was very hard, even though we were able to prepare for it. We hoped she would get a lung transplant but in May last year she was at 20 per cent function and couldn’t even stand up. She wasn’t strong enough for surgery. Iona died in hospital two days after our mum’s birthday.
Hannah has started an online campaign in partnership with the Cystic Fibrosis Trust in an attempt to overturn the decision not to prescribe Orkambi.
“I know it costs a heck of a lot of money, but it would prevent other costs. For example in October I had a collapsed bowel and needed an ultrasound, X-ray and MRI scan and I racked up nearly £10,000 then. That would cover over a month of Orkambi,” Hannah said.
“Two of my closest friends with cystic fibrosis died in February, within four days of each other. One was 22 and the other was 23 – people are dying so young and it’s not fair.
“I don’t know how quickly my lungs will decline. I can do everything for myself most of the time, but some days I spend the whole day in my pyjamas as I’m so drained. Lately I’ve not been well, so just getting out of bed is an issue. Making a cup of tea knocks it out of me.”
Hannah has to take so much medication she has a big whiteboard in the utility room to keep track of it.
She needs antibiotics to prevent chest infections, drugs for her liver, more for her stomach and several vitamins and calcium.
Tests have shown that Orkambi benefits patients by reducing instances where they experience a sudden worsening of symptoms that requires hospitalisation.
Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation, said that the decision was taken very carefully, adding: “Orkambi is a new treatment option and it is disappointing that we are not able to recommend it.
“However our independent committee found that when compared to the current standard of care, the benefit it offered was modest and comes at a considerable cost.
“We have to recognise that the NHS is a finite resource and we can only recommend treatments for routine funding that are both clinically effective and represent good value for money.”
Consul-tees, healthcare professionals and members of the public can still comment on the preliminary recommendations, which are up for public consultation until April 15.