Hope for children suffering from cystic fibrosis

Sarah Drinkwater with daughter Chloe.
Sarah Drinkwater with daughter Chloe.
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PARENTS of children suffering from a debilitating disease have been heartened by news of a potential breakthrough in the availability of a life-changing drug.

For three years campaigners have fought for the drug Orkambi, used to treat cystic fibrosis, to be available on the NHS.  

Health Secretary, Matt Hancock, has been meeting with the pharmaceutical company Vertex over the provision of the cystic fibrosis drug, Orkambi.'PRESS ASSOCIATION Photo. Picture date: Monday March 4, 2019. See PA story POLITICS Grayling. Photo credit should read: PA Wire

Health Secretary, Matt Hancock, has been meeting with the pharmaceutical company Vertex over the provision of the cystic fibrosis drug, Orkambi.'PRESS ASSOCIATION Photo. Picture date: Monday March 4, 2019. See PA story POLITICS Grayling. Photo credit should read: PA Wire

After an eight-month stand-off between National Institute for Health and Care Excellence (NICE), who govern drug provision, and the pharmaceutical company Vertex, news has emerged of a ‘positive’ meeting having taken place.

Gemma Weir, of Paulsgrove, has tirelessly campaigned to get Orkambi freely available to help save lives – including her five-year-old daughter Ivy.

Gemma said: ‘This is a life-changing drug which could make such a difference to Ivy and other sufferers. Very few sufferers live past 30. If this treatment is started early it can lead to a near normal life expectancy.’

The dispute revolves around the cost of the drug with NICE failing to authorise the treatment due to the prohibitive £104,000 per patient per year cost. 

Gemma Weir with her five-year-old daughter Ivy, from Portsmouth, after she delivered hundreds of letters to Downing Street calling for drug Orkambi to be free.

Gemma Weir with her five-year-old daughter Ivy, from Portsmouth, after she delivered hundreds of letters to Downing Street calling for drug Orkambi to be free.

Gemma said: ‘The last we heard was the NHS offered a rate of £10,000 per patient which was dismissed by Vertex with the company saying other companies were paying 90 per cent more. At the time the NHS said it was their final offer. It is good news that all parties have re-entered discussions. As far as I know this is the first time Mr Hancock has been involved.’  

A statement from Mr Hancock has now given parents hope their children could soon benefit from the life-changing treatment.

Mr Hancock said: ‘We had a constructive meeting with Vertex, NHS England and NICE to identify ways of breaking the current impasse so we can get this treatment to those who desperately need it.’

Vertex have also confirmed they hope to make the ‘potentially life-changing medicines available to people living with cystic fibrosis in England as quickly as possible’.

For Havant resident, Sarah Drinkwater, 31, whose daughter Chloe also suffers with cystic fibrosis, it provides hope that an agreement could soon be reached.

Sarah said: ‘This is huge step forward and an indication that all parties are on the same page. Everyone involved needs to show some leeway. We are grateful for companies such as Vertex for their research but it is only useful if people can access treatments. I won’t be reassured until I get that first prescription in my hand.’

Gemma added: ‘I don’t think Mr Hancock would have made this public statement if he wasn’t confident of getting a deal done. However I can’t relax until a deal is signed. This has been a long battle and I just want to get my daughter and other sufferers the treatment they need.’

Mr Hancock and Vertex have both confirmed further discussions will take place next week.