Why has my sister got wonder drug while I must wait?
TWO sisters suffering with cystic fibrosis have highlighted the health injustice that means only one can get a wonder drug.
Last week the Daily Express exposed the scandal of sufferers in England being denied Kaftrio while people in Wales, Northern Ireland and Scotland can receive it.
Siblings Louise McLean, 28, and Lauren Cole, 29, were born with the genetic condition and have identically rare CF mutations.
As schoolchildren, every day started with a nebuliser and an hour of physio.
Last September, after falling gravely ill, Louise got Kaftrio for free from drug firm Vertex under its “compassionate use” scheme – with amazing results.
Married Louise, mum to eight-year-old Alyssia, said: “I love updating my family with my good health news – but it’s with a heavy heart.
Tough
“That’s because my sister still cannot access Kaftrio, yet she watches on and sees all my benefits. I know she is happy for me but it is so tough for her.
“She is getting married this year and wants children and is thinking about the future.”
Lauren, of Basingstoke, Hants, is engaged to James Belam, 31, and currently taking Vertex’s oldest drug, Kalydeco. That stabilises her lung function at around 60 per cent.
But she knows Kaftrio almost doubled her sister’s lung function.
Office manager Lauren said: “I’m quite well but have to work hours a day to maintain my health. I was really worried about Louise before she got Kaftrio and relieved when she got it. But it makes me sad we can’t experience the wonderful effects of the drug side by side.”
When Louise became pregnant her lung function plunged from 60 to 24 per cent, which caused serious breathing problems.
She said: “I felt CF robbed me of my early motherhood.”
Last September her doctors applied to Vertex for compassionate use of Kaftrio and her lung function soared to 43 per cent.
She said: “I could feel it working immediately, and now I breathe so easily. I don’t cough when I get up any more, and I feel I’ve a future as a wife and a mother.”
There are 10,509 people in the UK with genetic condition CF, a chronic disease that affects the lungs and digestive system.
Usage in the US shows Kaftrio works for around 90 per cent of those with CF. But NHS England (NHSE) is not allowing all those suitable to use it.
Around 1,000 patients with CF gene mutations like Louise and Lauren can take Kaftrio in the US, Wales, Northern Ireland and Scotland. But overcautious health bosses in England are stalling.
However, there may be hope. Giving patients clinically approved drugs not yet granted a specific licence is called off-label prescribing. After we alerted NHSE to its Kaftrio inequality last week, it suggested it was reviewing possible future off-label prescribing.
It said: “The NHS is working to support clinicians with the option of prescribing Kaftrio off-label for patients that need them.”
Yesterday, David Ramsden, chief executive of the Cystic Fibrosis Trust, said they met with NHSE last week, “and reaffirmed a shared commitment to ensure every eligible patient can benefit from effective treatment”.