Powys mum calls for cystic fibrosis drug to be given on NHS

Kate Wozencraft from Rhayader wants Kaftrio to be made available on the NHS as it could help her son Bertie, one, who has Cystic Fibrosis live 30 years longer.

CF is genetic condition that causes sticky mucus to build up in the lungs and digestive system – leading to lung infections and problems with digesting food.

“It is horrific,” said Kate.

“He has very poor sleep because he doesn’t like to lie down because he’s coughing so much. He can’t get to the end of his cough and ends up choking himself and will make himself sick, you can’t leave him on his own.

“He doesn’t like to lie flat, he has to be with you. He gets very little undisturbed sleep, gets very high temperatures. He is unable to go to nursery. We can’t go to work if he is ill which is a massive cost and looking forward to the future it will really affect his education.

“They get frequent infections and need hospitalisations and are in hospital for at least seven days on IV antibiotics. They need a side room, they are at risk of infection and picking bugs up more frequently.”

Bertie suffers from Cystic Fibrosis which means he is more prone to infections. (Image: Kate Wozencraft)

Bertie already has to take regular vitamins, supplements for digestion, steroids and prophylactic antibiotics. As time goes on her could have to take 60 tablets a day.

But the breakthrough with Kaftrio can boost life expectancy from around 30 to around 60 and massively reduces the need for medication.

“The drug is known as the miracle drug in the CF community,” said Kate.

“The people who have gone on it have noticed a difference especially in their lung function overnight. You wouldn’t believe how good the drug is.

“Kaftrio is a tablet only taken three times a day. The benefits are immense but so are the price tags.”

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The National Institute for Health and Care Excellence (NICE), which decides what medications are available on the NHS, says a decision is yet to be made about whether Kaftrio will be prescribed to future patients – despite it being recently licensed for children aged two to five.

American biopharmaceutical company, Vertex, charges £180,000 per year for each patient – despite the list price of the drug being under £9,000.

Bertie may not be able to access the miracle drug due to it's high cost for the NHS (Image: Kate Wozencraft)

A spokesperson for Vertex said: “The price of our medicines reflects their clinical value and benefits to patients, caregivers and healthcare systems.

“Looking at production costs alone is not enough to determine the price of a medicine. It has taken over 20 years and billions of dollars to fund our research and development. This investment has enabled us to do what was once thought impossible - developing medicines that treat the underlying cause of CF which have fundamentally changed the way the disease is treated. 

“It is also important to remember that many of the prices that you see quoted publicly are list prices which do not reflect the final reimbursed price agreed with the NHS.  Reimbursed prices are not set unilaterally by the manufacturer but agreed with the health authorities in each country.”

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.

“The committee want to hear from stakeholders through consultation on important aspects of its draft conclusions. This is so that we have all the relevant information to accurately capture the value of these effective medicines when the committee makes its final decision.

“We are continuing to work collaboratively with the company, NHS England and other stakeholders including the Cystic Fibrosis Trust to deliver the best outcome both for people with cystic fibrosis and for the wider NHS.”

However, Kate argues that NICE should make the drug available and scolded Vertex's pricing.

She said: “Cost shouldn’t be put on a child’s life. The drug is available and has effective benefits. Why aren’t we letting them have it to live a long, health and happy life?”

The decision has led to a petition being put up for the future of the drug to be debated in Parliament and has already gained thousands of signatures.

“We’ve already contacted MPs and the petition has already gone to the Senedd. Mark Drakeford is already aware of it,” said Kate.

“We are hoping for it to get to 100,000 to get it to Parliament to make more of a noise about it – so more people are aware of it and the condition so they can see how outrageous it is.”

For those wanting to give their support to the petition you can sign it here.