Mum wants cystic fibrosis drug available on the NHS
ARUNCORN mother has backed calls for the approval of a precision drug for treating cystic fibrosis (CF) that could help half of all patients with the condition.
Danielle Cartwright, 29, of Grangeway, said that since her daughter Talia started to take the medicine Kalydeco just over a year ago, the tot’s health has improved so much that she appears a perfectly healthy child despite having CF.
She is now eager for another drug, Orkambi, to be approved for prescription on the NHS so that other patients and parents can enjoy a similar turnaround in their quality of life, and has joined thousands of other protestors in calling on the Government to increase access.
Whereas Kalydeco works with 5% of CF patients who fit the right genetic profile to be responsive to that drug, Orkambi is believed to work with 50%, and campaigners said that the addition of a third medicine could boost coverage to treating 90% of patients.
At present, Orkambi is only prescribed in rare cases on compassionate grounds by NHS because the cost of £104,000 per patient per year is prohibitive to wider use.
But campaigners say the drug’s effectiveness would go a huge way to offsetting the price, by reducing hospital visits – that can be as often as two weeks four times a year, by reducing use of other expensive drugs and by enabling patients to spend less time managing their condition and more of their energy in activities such as work. ● A round table event discussion took place in Westminster to discuss the prescription status of Orkambi.
It is also believed the drug could have already saved four young lives had it been prescribed.
All eyes are now turning to a parliamentary debate on Monday, March 19, which is taking place after a petition calling for Orkambi’s approval passed the 100,000-signature threshold for a mandatory House Of Commons discussion.
This followed a round-table event attended by MPs and pharmaceutical company and Cystic Fibrosis Trust representatives in parliament last Wednesday, as protestors endured freezing temperatures to hold a demonstration outside Downing Street.
The Cystic Fibrosis Trust has estimated that there are 3,296 patients in the UK who could benefit from the drug.
Cystic fibrosis is a genetic condition affecting more than 10,400 UK residents.
Patients are born with it and only half make it to their 40th birthday.
Danielle described how her own experience with the benefits of a precision drug for Talia had made her keen for others to enjoy similar benefits. She said: “She’s a happy normal child. “Looking at her you wouldn’t know any different, it’s what’s going on inside.
“Her disability’s invisible, if no-one knew her they’d think nothing’s wrong.
“Since she’s been on Kalydeco, the difference is amazing – she’s got more energy.
“Prior to that she would really struggle with being poorly.
“Since then it’s reversed itself. She’s come off one of her medications, she’s constantly got rosy cheeks whereas before then she was always pasty.
“Since being on Kalydeco, she hasn’t grown any bacteria or infections on her swabs so they removed her nebuliser (oxygen breathing apparatus).”
She added: “I’m on a lot of CF forums and a lot of the people’s children need that drug. It would be so nice if the other children had the same benefit.
“The more people speak about it, the more it’s going to be heard.” ●