Leeds mum “devastated” over proposal to withdraw life-saving cystic fibrosis drug from NHS
Leeds woman Rula Samara-Sellers pleas to her Facebook community as a cystic fibrosis medicine her new-born daughter is expected to rely on is suggested to be withdrawn from the NHS.
Samara-Sellers posted online in November to get support for her cystic fibrosis daughter’s future.
“We received devastating news last night that the body that decides which medicines are cost-effective in the NHS has decided this drug is too expensive and these lives are too expensive to save. All we have been doing is counting down the days until Layla could start this medication at age 2.”
Samara-Sellers, November 2023
On November 3, the National Institute for Health and Care Excellence (NICE) published a report on the three cystic fibrosis medications — Orkambi, Symkevi and Kaftrio.
Although they have said that these clinically effective treatments bring important benefits to patients, they are too expensive at the list price for NICE to be able to recommend them for use on the NHS.
“Anybody that’s already on the drug, the NHS has already confirmed they will continue to pay for them. But Layla’s only 3 months, so she’s the start of the generation that will be affected by this.”
-Samara-Sellers, November 2023
“It’s like a death sentence by withdrawing it from people.”
“Kaftrio can prolong Layla’s life from the average of 30 now to around 60, or even 80. It’s wonderful to know that my daughter can live a beautiful life.”, said Samara-Sellers.
While there is no cure for cystic fibrosis, there are treatments to alleviate symptoms, minimise complications, and prolong life expectancy. The medicine in question targets the root cause of the condition.
NICE’s latest appraisal update is not a final decision, and there is currently an ongoing 4-week consultation which will end on November 24. The body will meet again in December to discuss the consultation responses. The conclusion of the evaluation is expected to be published in March 2024.
Samara-Sellers is hoping the feedback collected from the consultation form will change the committee’s verdict. “What I need your help with please is to fill in this form with feedback by placing a comment on the online consultation form. It will make a massive impact on the NHS considering funding this drug”, she writes on Facebook.
Cystic fibrosis is a genetic condition that causes recurring lung infections and problems with digesting food. This is due to a build-up of sticky mucus in the lungs and digestive system.
Patients are often diagnosed at birth, and the condition tends to worsen over time. About receiving her daughter’s diagnosis, Samara-Sellers said, “It really was the worst day of my life. You begin to grieve the life your daughter would’ve had and I literally just felt everything shutting down.”
“The healthiest my daughter will ever be is the day she was born. Every single day brings potential scarring and damage to her organs.”
“My reality is the constant fear of outliving my own baby girl, this haunts us every minute of the day.”, said Samara-Sellers.
John Stewart, National Director for Specialised Commissioning at NHS England, said in a statement: “NHS England remains committed to ensuring that these life-changing cystic fibrosis medicines are available to everyone who may benefit, now and in the future, and will continue to work with Vertex and NICE through the next stages of the appraisal process to make this possible in a way that is fair to patients, and fair to taxpayers.”
David Ramsden, CEO of Cystic Fibrosis Trust, said, “We must never return to a situation where people with cystic fibrosis die far too young, knowing there’s a treatment that could change that.”
Cystic fibrosis affects more than 10,900 people in the UK, with around 4% of the population carrying the faulty gene that causes the condition.
The consultation form for NICE’s appraisal is available at https://www.nice.org.uk/guidance/indevelopment/gid-ta11187.
