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Five-year-old Eliza Turner has cystic fibrosis, a genetic condition affecting more than 10,500 people across the UK, which means she has to be hooked up to breathing apparatus evening morning, must take a cocktail of tablets with every meal and is particularly prone to potentially fatal infections.
Her parents Phil and Rachel, who live in Wadsley Bridge, believe a new drug called Orkambi could dramatically improve her day-to-day existence and extend her life by some 20 years.
But the expensive treatment, which is made by a company called Vertex and has been shown to significantly slow the decline in lung function experienced by people with the disorder, is currently only available through the NHS in Scotland and not the rest of the UK.
Health chiefs in England are in talks with the manufacturer about providing it on the NHS here but Eliza’s dad says ‘time is against us’ and they cannot afford to wait for the outcome when Eliza so urgently needs access to the drug.
“If someone with cystic fibrosis like Eliza is lucky and avoids all the coughs, colds and bad chest infections that could kill her they might live to their mid 30s or 40s, which is half a life as far as I’m concerned,” said Phil, a 40-year-old HR manager for Royal Mail.
“Clinical trials show this drug could increase Eliza’s life expectancy by up to 20 years but time’s against us because she’s turning six in December and she needs to start her treatment as soon as possible.
“Scotland’s done the right thing for its cystic fibrosis community but in England they’re telling us a deal can’t be done.
“They’re putting a price on our daughter’s future and that of thousands of other people in England, for whom this is a death sentence. It just feels like we’re invisible.”
Phil has already been for a job interview in Scotland and is in talks with his employer about moving north of the border, despite the upheaval it would cause for Eliza and her four-year-old brother Henry.
“We’ve lived in South Yorkshire all our lives, we’ve got a good community here, decent schools and our family and friends around us, so we don’t want to uproot,” he said.
“But as parents you’d do anything for your children, and we don’t feel we have any other choice.”
The Cystic Fibrosis Trust, which is campaigning for access to Orkambi and other life-changing drugs, says Orkambi could benefit around half of those with the condition in the UK and has been shown to improve lung health and reduce hospital admissions.
Last month, the Scottish government announced a five-year deal giving access to Orkambi and another drug called Symkevi for everyone there who could benefit, yet the drugs are not yet recommended for use on the NHS in England, Wales or Northern Ireland, where they are only available rarely on compassionate grounds or through limited clinical trials.
Gill Furniss, MP for Sheffield Brightside and Hillsborough, who last week highlighted Eliza’s case in parliament, claimed no family should have to move hundreds of miles just to access the drugs their children need.
“If it’s good enough for Scotland I just feel we have to do the same for people in the rest of the UK,” she said.
“Even more shocking is that the company revealed it destroyed 8,000 packets of Orkambi, which could have transformed the lives of many people living with cystic fibrosis, after they expired while negotiations were ongoing with the NHS.”
A spokeswoman for the organisation said: “The vast majority of companies behave responsibly and work well with the NHS. Sadly, however, Vertex is an extreme outlier in its pricing.”
The Department of Health and Social Care urged Vertex to accept NHS England’s ‘very generous offer’ and said health secretary Matt Hancock would be bringing in representatives from the US firm to ‘explore what the barriers are to reaching an agreement’.
A spokesman for Vertex said it was ‘committed’ to reaching a long-term agreement for access to its medicines for all eligible patients in the UK and continues to meet regularly with NHS England.
“In the absence of an access agreement, Vertex will continue to provide free medicines to patients who are the most seriously ill, based on objective clinical criteria. Since its inception, these medicines have supported more than 800 of the sickest CF patients in England and over 1,000 patients across the UK,” he added.
The company strongly refuted the $21bn figure quoted by Ms Furniss, which it said was inaccurate.
It said that while it recorded a profit in 2018, this was only the third year it had done so, having incurred $3bn cumulative losses throughout its history yet continued to invest heavily in research and development.
The firm added that it does everything it can to avoid having to withdraw medicine stock from use but had to adhere to regulations regarding quality.
Cystic fibrosis causes a build-up of mucus in the lungs and other parts of the body, which leads to a range of symptoms including susceptibility to lung infections and difficulty digesting food.
It can be a particularly lonely condition for people like Eliza because the risk of cross infection means they cannot meet others with the disorder, and things other children take for granted like splashing in puddles or playing with sand can be fraught with danger due to the bugs which could be lurking.
Despite this, Phil says Eliza is a happy girl who is a popular pupil at Parson Cross Church of England Primary School, has a great sense of humour and loves dancing.
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