Staffordshire girl, 4, living with cystic fibrosis given green light for "miracle drug"
A mother who feared her daughter would not get potentially life-saving drugs has been given good news over access and funding.
Lainey McDaid, aged four, has cystic fibrosis which is an inherited disease affecting lung functions and the digestive system, causing breathing difficulties and many other issues.
Her mother Louise started a petition to get access to modulator drug Katfrio which was previously only available for six year olds and up. It is proven to increase lung function, growth and life expectancy by up to 40 years – currently a typical age of death for someone with cystic fibrosis without treatment is 50. On Friday, the National Institute for Health and Care Excellence (NICE) released a statement on Kaftrio and other modulator drugs, Orkambi and Symkev, confirming they are highly effective for people with cystic fibrosis but are judged too expensive for the NHS.
But Louise, of Burntwood, has now been told that children between two and six will be eligible for the drug and funding is likely to be available for them. It is a bittersweet news for the mother of two, who is delighted for Lainey but is planning to continue the petition to try and help youngsters who are under four to be able to access the drugs.
She said: “Lainey is only four years old and already takes 26 tablets a day – the gene of her cystic fibrosis means she can only have Katfrio which is the closest cure to a miracle drug and could save or at least sustain her life. The news she will be able to receive it and that it will hopefully be funded is a lifesaver – she often talks of having a baby and getting married and this could mean she will live to fulfil those dreams.
“She has a little brother Alfie aged only one who doesn’t have cystic fibrosis and I was terrified they wouldn’t grow up together and of the impact it could have on him seeing his sister suffer or potentially die. So it is a win for her and others aged two to four but my heart goes out to the parents of sufferers below that age as me and Lainey know from experience what cistic fibrosis can mean and for that reason I will continue with the petition.”
For more information on the petition, go to petition.parliament.uk/petitions?q=Fund+continued+access+to+modulated+drugs+for+cystic+fibrosis+patient.