Leeds family 'living in turmoil' as they await decision on daughter's life-changing cystic fibrosis medication

Cystic fibrosis, a rare condition that causes sticky mucus to build up in the lungs and digestive system, can mean tragically short lives for sufferers.

But in recent years, the medication Kaftrio has been given hope to families of youngsters with the hereditary disorder.

It significantly improves lung function, helping those with the condition breathe more easily and enhancing their overall quality of life.

Currently, it is available to children over the age of two - and has had breakthrough effects for those who may have been hospitalised previously for minor bugs and colds.

But last year, the National Institute for Health and Care Excellence (NICE) decided that the drug was too expensive to be recommended for use on the NHS, and a consultation was launched over its continued availability.

Those who are currently in receipt of the treatment will continue to be given access to Kaftrio, regardless of an expected NHS decision. But for children younger than two who have not yet had a chance to experience its benefits, the drug may never be available.

It was previously expected that a decision would be made by March 20. However, families said this week that they were still waiting to hear.

Rula Sellers, 36, was among those frustrated about delays. Her seven-month-old daughter Layla, who she described as a "ray of sunshine", was diagnosed with cystic fibrosis just weeks after she was born.

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Rula, from Rothwell, said of the delays: "There has been absolutely no regard to the impact this has had on the families like ours, who are living in turmoil waiting on a decision."

She previously told the YEP that denying the drug would be tantamount to a “death sentence”.

Rula discovered that Layla had cystic fibrosis after a heel prick test, which is given to babies five days after birth and can pick up any one of nine rare health conditions. They were contacted soon after the test by a health visitor, who told them they’d need to be seen by a nurse.

"The NHS has entered negotiations about Kaftrio, but at no point are they providing us or anyone with anything useful or helpful," said Rula.

It is estimated that around 11,000 people in the UK have cystic fibrosis. They include Nellie Watson, six, from Roundhay.

The youngster started on Kaftrio last year and has already seen the benefits, according to mum Kirsty, 34.

She said that it “breaks her heart” that other children could be denied the same treatment – and has been calling for NICE’s recommendation to be rejected.

It is understood that commercial discussions between NHS England and Vertex, the manufacturer of Kaftrio, are ongoing. The outcome of those discussions will be used by NICE in formulating a final recommendation on the use of the drug to treat cystic fibrosis.

On 4 March, NHS England issued a statement from John Stewart, the service's National Director for Specialised Commissioning. He said: “Since 2019, thousands of people with cystic fibrosis have been able to benefit from licensed treatments – firstly Orkambi and Symkevi and then Kaftrio, following its marketing authorisation in 2020.

"Access to these treatments is provided under the terms of an interim commercial agreement reached between Vertex and NHS England, with the full support of NICE.

“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 we are currently negotiating with Vertex with the aim of reaching a long-term commercial agreement that makes this possible in a way that is fair to patients, and fair to taxpayers."

His statement added: “Commercial negotiations between NHS England and Vertex remain on-track and are being undertaken constructively and within a confidential environment to best enable progress, however we remain committed to ensuring patients and the wider community of family, carers and friends, are provided with regular updates as negotiations continue.

“Notwithstanding the ongoing commercial negotiations, the existing agreement contains a flexible commercial mechanism that will ensure continued access for patients, already receiving any of the licensed treatments whatever the outcome of the final NICE evaluation is. Eligible children and adults with cystic fibrosis are continuing to receive ongoing treatment and be initiated onto treatment with these drugs, as clinically appropriate.”

The YEP has asked NHS England when families can expect a decision on the continued availability of Kaftrio.