Norfolk families affected by cystic fibrosis push for life-changing treatment to be made available amid price row

PUBLISHED: 10:27 24 April 2018 | UPDATED: 10:27 24 April 2018

Ellie Loades. Photo: Loades family

Ellie Loades. Photo: Loades family


A drug manufacturer has been urged to lower the price of a life-changing treatment for cystic fibrosis (CF) sufferers so it can be made available on the NHS.

Ellie Loades' parents Nick and Elaine. Picture: Ian BurtEllie Loades' parents Nick and Elaine. Picture: Ian Burt

Health ministers Steve Brine and Lord O’Shaughnessy called on Vertex Pharmaceuticals, which makes the drug Orkambi, to bring negotiations over cost to “an urgent resolution”.

The drug is not currently available on the NHS because it is not considered cost-effective, but has been shown in clinical trials to improve lung function and respiratory symptoms.

And families in Norfolk affected by the condition have thrown their support behind the medication being made available.

Nick Loades’ daughter Ellie, from Colkirk, died aged 16 in November 2013 - she was diagnosed with CF at birth.

CF sufferer Ryan Hughes. Photo: Liz CoatesCF sufferer Ryan Hughes. Photo: Liz Coates

Since, he and wife Elaine started the charity, Ellie’s Love, and have campaigned for improved treatment.

He said: “I think it’s a brilliant idea that the drug is here but it’s ridiculous we are having to battle with the pricing.”

Mr Loades said while Ellie was alive, they had a similar battle over medication which could be given at home instead of at hospital.

As Mrs Loades suffers with multiple sclerosis (MS) when Ellie went into hospital she would have to go to respite care.

But potential treatment for Ellie at home was denied because of the cost.

“It has a horrendous impact,” Mr Loades said.

And the drug’s effect of improving lung function would be beneficial, he added.

“I can just imagine it because when you go into hospital it’s the first thing they measure, it’s so disheartening when it’s bad.

“I just think they’re playing with children’s lives. If Ellie was still here we’d be fighting for it, [the drug company] is holding the NHS to ransom.”

Gina Hughes from Ormesby St Michael, whose 23-year-old son Ryan has CF, hoped the introduction could mean a wider range of drugs being made available.

She said: “Although this drug wouldn’t help Ryan because of his genetics, it could open doors to other drugs being used.”

The family were told an equivalent drug for Ryan’s form of CF was still five years away - Orkambi is for people with the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

But Mrs Hughes said any improvements would save hospital admissions - and therefore money - in the long run.

“It does make me angry when they say it costs this, it costs that,” she said. “I know it does seem a lot of money but when Ryan was last in hospital that admission cost £55,000. It’s not a cure but it will alleviate symptoms.”

NHS England has submitted a counter-offer to a deal proposed by the manufacturer but is awaiting a response.

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage and affects around 10,400 people in the UK.

Only around half of those with cystic fibrosis live to celebrate their 40th birthday.

A Vertex spokesperson said: “We are pleased that the government is in agreement that we urgently need to find a solution to get our medicines to cystic fibrosis patients as soon as possible and are encouraged that we already have a date in the diary in the coming days to start a meaningful dialogue.

“As we previously said, negotiations over email are rarely productive so we are surprised to be asked to respond to a letter via the media that we have not yet received.

“The current counter offer amounts to a refusal to make any additional funding available for ORKAMBI® (lumacaftor/ivacaftor) or future medicines.

“We have responded to all requests for further information and look forward to discussing them with NHS England this week. We hope the NHS truly values the transformative nature of our medicines in a manner consistent with the value applied to KALYDECO® (ivacafator), which is available through the NHS and was last positively reviewed just over a year ago.

“We hope we can agree an approach with NHS England that benefits all eligible patients equally, as soon as possible. We call on the NHS to bring a positive and flexible approach in the best interest of CF patients.”

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