Life-changing cystic fibrosis drug 'bittersweet' as sister denied access
Video report by ITV News Meridian's Charlotte Briere-Edney
A woman from Basingstoke, who is living with cystic fibrosis, says being given a life-changing drug is bittersweet because her sister, who suffers from the same condition, is being denied access to it.
Louise McLean, 28, and her sister Lauren were both born with the same, rare type of cystic fibrosis, but only one of them has been prescribed the medicine Kaftrio.
Louise was diagnosed as a baby, which has meant nebulisers, hospital stays and hours of therapy every day. Then, last year, she tried the new drug.
Louise says the pill has dramatically improved her quality of life.
She said: "Within hours of starting it, I felt the effects. From that day it has just been incredible. My lung function starting was 28%, and I'm now at 43%. I never thought I would get that number again."
Cystic fibrosis is caused by genetic mutations, which lead to a build up of mucus in the lungs. Sufferers are prone to chest infections and breathing difficulties.
Kaftrio was licenced in the UK in summer 2020, but not for everyone. In England, some patients with rare gene mutations cannot get the drug on the NHS.
In fact, that is what happened to Louise. She's only taking Kaftrio because she was so terribly ill, the pharmaceutical company which makes the treatment, gave it to her for free under a 'compassionate use scheme'.
Louise's sister Lauren has the exact same cystic fibrosis mutation, so like her sister, she also cannot get Kaftrio on the NHS, but she is not sick enough to be granted compassionate use.
However, if Lauren lived in Wales or Scotland, she would be able to benefit from the therapy.
Some patients can be given medicines off-label, which means doctors can prescribe a drug outside of the licence it has been granted. But Lauren's clinicians told her that is not possible for her.
Louise is calling on NHS England to make it available for all sufferers.
The scope of the Kaftrio licence is being reviewed, and NHS England says it is "working to support clinicians with the option of prescribing Kaftrio 'off-label' for patients".
There is also hope that a government motion will speed things up and get patients like Lauren the drug that could change their lives.