Delilah was born with cystic fibrosis, but may not have access to ‘life-changing’ medicine when she turns six
One mother said she would have to watch her baby daughter die if certain cystic fibrosis medications were stopped.
The National Institute for Health and Care Excellence (NICE) has withdrawn recommendations for a number of cystic fibrosis (CF) drugs due to cost.
Charlotte Perkins, 38, whose 17-month-old daughter Delilah has had CF since birth, said she was “devastated”.
NICE said it was assessing the “cost-effectiveness” of medicines to ensure “taxpayers continue to receive value”.
“We are forced into a situation where we have to watch our daughter get worse every year until she dies at a young age. We can’t afford that,” said Charlotte, from Lareston, Bridgend.
The lungs of people with this condition can become damaged over the years and eventually stop working.
NICE’s draft guidance, which is open for consultation until 24 November, states that Kaftrio, Shinkevy and Orkambi are not recommended for use in new patients with cystic fibrosis.
However, existing patients will continue to receive the drug.
image source, family photo
Charlotte said Delilah was told she could get a “life-changing” drug when she was six years old, but that’s now in doubt.
Delilah’s six-year-old sister has written to NICE to make sure her baby sibling has access to life-saving medicine.
She writes, “Even though she is still young, she should also receive medications that older children and adults can take.”
She added that she was the one who wrote the letter, saying, “Delilah is still unable to write or speak properly, so as her older sister, it is my job to support her.”
image source, family photo
image source, family photo
Delilah’s six-year-old sister told NICE: “Please do the right thing for my little sister.”
Charlotte said: “I can’t imagine them taking the lives of children when there are medicines that can stop this disease and give them full, healthy lives.
“Our children deserve to live, they don’t want to be born with this disease… She is an innocent baby.”
Charlotte said she was told Delilah would have access to Kaftrio when she turned six and that it would “change her life”.
“We were told to ignore the life expectancies we read online and that children would no longer die from this disease,” she added.
image source, family photo
Charlotte said her family “broke apart” when they heard about NICE’s new guidance.
She said that when she first heard the news that her medication might be discontinued, she “couldn’t do anything for two days.”
“I tried to do some things, but we just completely fell apart as a family,” she said.
Katherine Mayer’s five-year-old daughter Charli was scheduled to start medication for CF before Christmas, but she was told that might not happen.
image source, family photo
Five-year-old Charlie was due to start before Christmas ahead of new NICE guidance.
Catherine, who lives in Porthcawl, Bridgend, said: ‘I know this drug is out there and is meant for her, but because of the cost she might not be able to get it. I just don’t like it. .It’s surprising.”
“It’s really shocking to be told that everything is going to be fine, that your daughter is going to be healthy and live a long life…Now you’re saying, ‘Oh, actually, these drugs are really, really expensive and we can’t afford them. It’s really shocking to be told, ‘I might not be able to do it because I can afford it.’
“This drug has the potential to extend the lives of children currently born with cystic fibrosis by more than 30 years, potentially making a huge difference in their quality of life.
“It is imperative that we give these children a chance at life.”
image source, family photo
NHS nurse Catherine said the only comparison was children being turned away in hospital to save money.
Catherine, who is an NHS nurse herself, said it was “indescribable” how she felt when she was told her daughter’s medication was being stopped because of cost.
“The only thing I can compare it to is taking your child to a hospital where treatments are developed and treatments are given according to her age, but hospitals do it because of cost. I can’t do that.”
The Cystic Fibrosis Charitable Trust said it was important to remember that the draft guidance from NICE was “not a final decision” and would not affect anyone currently taking the drug.
A spokesperson for the charity said: “We hope that this uncertainty will be resolved in the coming weeks and have written to the Secretary of State to urge Vertex, NICE and the NHS to resolve the issue. We are asking everyone to work together quickly to find him.”
“We would urge people that the best thing they can do now is to submit their evidence to the Attorney General. NICE consultation Encourage others to do so by 17:00 on November 24th. ”
“Evaluation of cost effectiveness”
The Welsh Government said it was relying on “the advice of independent reviewers, including NICE, to determine how NHS resources are best used”.
Helen Knight, director of drug evaluation at NICE, said: “We are assessing the cost-effectiveness of these cystic fibrosis drugs to ensure taxpayers continue to get value for money.” Stated.
“The Commission would like to hear from stakeholders through consultation on key aspects of the draft conclusions.
“Existing patients and new patients who started treatment while their NICE assessment was in progress will continue to be able to receive treatment.”
