'I'll be planning my baby's funeral': Norwich mum's fears if cystic fibrosis drug is withdrawn

• ITV News Anglia's Matt Hudson has been speaking with those who benefit from the drug.

The mother of a baby with cystic fibrosis fears she will be "burying my daughter" if plans to axe a specialist drug go ahead.

Kya Howard's nine-month-old daughter Harlow was diagnosed with cystic fibrosis when she was four weeks old.

Ms Howard, from Norwich, said she was "devastated" by the news that specialist drugs, which can prolong the life of people with cystic-fibrosis, may not be available on the NHS in the future due to funding issues.

The medicines regulator the National Institute for Health and Care Excellence (NICE) is concerned the NHS is paying too much for the American-produced medicines Trikafta/Kaftrio, Symkevi and Orkambi.

If the drug is pulled, it will mean that it would not be available in the future for new patients, but those who are already on it will still be able to access it.

Baby Harlow was due to start on the drug in February 2024, and Ms Howard is now concerned her daughter's life will be cut short if she is not able to get hold of the drug.

"Instantly it would give her an additional 30 or 40 years, and I know that she will have a long life and be able to enjoy that life," said Ms Howard.

"Without this medication, she can live a normal life, but she is going to suffer through a lot of it, she's going to probably have multiple illnesses and would be in hospital a lot of the time.

"These modulators prevent children from getting so ill from these infections. So even though they are saying this isn't cost effective on the NHS, it's going to be worse [without the drugs].

"If this drug is pulled a lot of the children are now going to be without this drug and they are going to be in hospital most of the time," Ms Howard told ITV News.

Nice said it was evaluating the "cost-effectiveness" of the medicines so that "taxpayers continue to get value".

However Ms Howard said she was told Harlow would have access to the drug and now is worried about her future.

"We were reassured she would have a healthy long life, she would outlive us and now that chance is being robbed and it is a possibility if they are pulled we are going to be planning our daughter's funeral and burying her one day," Ms Howard told ITV News Anglia.

"No parent should have to bury her child and a price should never be put on a child's head.

"This could change so many people's lives and it's absolutely disgusting that people could change this."

What is cystic fibrosis?

Cystic fibrosis is a progressive, genetic disease that affects the lungs, pancreas, and other organs. 

It's caused by a faulty gene that affects the movement of salt and water in and out of cells.

This, along with recurrent infections, can result in a build-up of thick, sticky mucus in the body's tubes and passageways – particularly the lungs and digestive system.

The condition tends to get worse over time and can be fatal if it leads to a serious infection or the lungs stop working properly.

But people with cystic fibrosis are now living for longer because of advancements in treatment.

Currently, about half of people with cystic fibrosis will live past the age of 40. Children born with the condition nowadays are likely to live longer than this.

Annemarie West's son Declan has been taking Kaftrio for three years, and she calls it a "miracle drug".

"Declan has not had a single admission to hospital since 2019 - so since he started this drug. He's had nearly four years on this drug and I can honestly say with a hand on my heart that it is a miracle drug.

"I can say that the drug has improved his lung capacity, it has increased it," said Ms West.

Ms West said without the drug her son would not have been able to live "a normal life" and any parents should be have access to it.

"I just can't believe we had 15 years of a very very poorly Declan, trying to give him his best life, trying to get him to live a normal life, and he's got that now," she said.

NICE said it was working through consultation on "important aspects" of its conclusions.

Helen Knight, director of medicines evaluation at Nice said: "We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money after interim access where further data was collected."

She said a consultation was taking place, adding: "Existing patients and new patients who are started on treatment while the Nice evaluation is ongoing will continue to have access to the treatments after Nice has issued its final recommendations irrespective of the outcome."

Vertex, which manufactures the drugs, said it was "disappointed with the draft Nice appraisal guidance", and that its data showed "the clinical value and benefit to patients, caregivers and the healthcare systems".

"We believe that the committee has not fully considered all of this real-world evidence and we will be providing additional information and analysis to be taken into account.

"We also disagree with some of the key evidence that Nice has selected to use which has a significant impact on the way that our medicines are valued, particularly underestimating the costs of caring for people with CF and the impact that CF has on a person’s quality of life."