Press Release: Lend Your Voice to the UK’s Cystic Fibrosis Community

Press Release: Lend Your Voice to the Fight to Breathe Faced by the UK’s Cystic Fibrosis Community

Cystic fibrosis families from around the UK have joined forces to release a video featuring their own children who are all living with the genetic disease.

The video is a bid to raise awareness about the fight for access to Orkambi, a ground-breaking precision drug for cystic fibrosis (CF) that is currently not available on the NHS in the UK. Orkambi treats the F508del mutation of the disease – the most common CF mutation that around 50% of people with cystic fibrosis in the UK have.

Although Orkambi was approved for use in the UK in 2015, both NICE (National Institute for Health and Care Excellence) and SMC (Scottish Medicines Consortium) were unable to recommend Orkambi for general use within the NHS/HSCNI on grounds of cost-effectiveness and a lack of long-term data.

There are however numerous anecdotal accounts of people whose life has been transformed by the drug – people who have been lucky enough to take the drug during clinical trials or who have access to Orkambi in other countries.

The awareness video shows images and video clips of the many hospitalizations, painful treatments, constant medication and daily physiotherapy that people living with cystic fibrosis have to endure. It can be viewed on YouTube.

Cystic fibrosis is a life-shortening genetic condition – only half live to celebrate their 40th birthday. It causes the internal organs, especially the lungs and digestive system, to become clogged with thick sticky mucus, resulting in chronic infections and inflammation of the lungs.

Having a child with cystic fibrosis means a change of lifestyle for any family as they adapt to the regime of antibiotics, vitamins, physiotherapy, clinic visits and hospitalisations required to keep a person with cystic fibrosis healthy.

Lorraine Barnes, the mother of two sons who both have CF said “This video shows the harsh reality of daily life with cystic fibrosis. The CF community desperately need hope and the chance of a brighter future. We all fundraise – many of us for years, in the hope of a breakthrough drug. Then when one comes along – in this case Orkambi – it is deemed too expensive to provide on the NHS. Our children are suffering and we feel helpless in the face of this terrible progressive disease.”

“Knowing that a drug that has transformed the lives of people in other countries is sitting on a shelf just out of reach is heart-breaking.  Orkambi isn’t suitable for everyone with cystic fibrosis, but we hope to reach a deal similar to the one achieved in Ireland recently, where swift access to other CF drugs currently in the pipeline would be included as and when they become available.”

In June Orkambi protests were held in England, Scotland, Wales and Northern Ireland. A recent event was also held at Westminster on November 29, to inform MP’s about Orkambi and encourage them to help find a way forward in negotiations between Vertex, the company who manufacture Orkambi and the NHS.

Anyone wishing to support the Orkambi campaign can do so by writing to their MP or signing this petition. Although the petition is for the Welsh Assembly, anyone in the UK or abroad can sign it – once one nation grants access, pressure will be put on the others in the UK to follow suit.

Read more about the Orkambi campaign on the following links:

To connect with another cystic fibrosis community, check out Breath With Me.

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