Dear Mrs May, Please help me live a longer and happier life. Alex, aged 11
SCHOOLGIRL WITH CYSTIC FIBROSIS PENS PLEA TO PRIME MINISTER TO MAKE LIFELINE DRUG AVAILABLE ON NHS
A LITTLE girl with cystic fibrosis has written to the prime minister asking for a life-lengthening drug to be prescribed on the NHS.
Alex Darkin, 11, was diagnosed with the debilitating condition at birth and knows it will shorten her life. The youngster, from Prestwich, has now written to Theresa May urging her to support a campaign to allow people with cystic fibrosis access to new medicines. One of the drugs is called Orkambi, a medicine that nearly half of people with cystic fibrosis could take, including children aged six and over.
In her letter, Alex wrote: “My name is Alex, I’m 11 years old. I have a condition called cystic fibrosis. It’s very annoying and boring. Cystic fibrosis means I’ve got to go into hospital a lot and have a lot of medication. There’s a drug called Orkambi. If I had it I wouldn’t have to go to hospital as often and I would live a longer and happier life. It makes me sad when I have to go to hospital because I hate needles and I miss my family and friends.”
Alex’s brother Harrison, eight, has also written a letter about her illness. “I miss Alex and my mummy when they have to go to hospital, it makes me feel sad,” he wrote.
The siblings’ letters have now been handed in at 10 Downing Street. Orkambi cannot cure the condition, but it has been found to slow decline in lung function – the most common cause of death for people with cystic fibrosis.
It has also been shown to significantly reduce chest infections requiring hospital treatment.
Orkambi was recognised by the National Institute of Health and Care Excellence (NICE) as an important treatment in 2016.
However it is not available to the majority of cystic fibrosis patients because the drug is not considered cost-effective by NICE.
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “Cystic fibrosis is a terrible condition. Significant advances in medical science mean new precision medicines are available or in development that have the potential to transform the lives of people with cystic fibrosis and we urge the prime minister to ensure that they are available for the thousands of children and adults who have been waiting far too long.”
A NICE spokesman added: “NICE issued guidance in July 2016 which did not recommend Orkambi to treat cystic fibrosis. The decision was based on the evidence and price proposal provided by Vertex at the time of the appraisal. A consideration of a review of the guidance can be brought forward where there is new evidence that is likely to impact on the current recommendations. We remain open to a further approach from the company, for example, with new data or a revised price.”