Mum slams baby's 'death sentence' as miracle cystic fibrosis drug costs too much

23 January 2024 , 15:50

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Baby Layla is five months old (Image: Rula Samara-Sellers / SWNS)

A furious mum says her five-month-old daughter faces a "death sentence" as she is being denied a life-changing drug on the NHS.

Rula Samara-Sellers has pleaded for her baby Layla to receive the wonder medicine Kaftrio, costing up to £160,000 a year per patient, to treat her cystic fibrosis. The mum-of-three said she and her husband Connor, 31, endured the “worst days of our lives” when her daughter was given the devastating diagnosis.

Layla has the rare genetic condition and struggles with infections from mucus build-up in her lungs and digestive system. Those with the condition often have tragically shortened lifespans. Rula, 37, said her family were originally told Layla could qualify for Kaftrio at the age of two, which NHS England previously hailed as a ‘miracle treatment’.

But she now fears Layla may never get it after the National Institute for Health and Care Excellence (NICE) put the drug's cost-effectiveness under review. Rula from Leeds, West Yorkshire, said: “I think it’s absolutely disgusting. If they did say that she was not entitled to it when she was two, they are literally giving her a death sentence. She is sick, and now you’re saying that it’s too expensive - and her life is too expensive?

"So she’ll continue having a terrible quality of life for the sake of saving money for the NHS. They’ll be providing everyone in the community with a death sentence and saying their lives are not worth it… This is inhumane, they cannot decline it.”

Man diagnosed with life-changing disease forced to take 40 tablets a day to live

Mum slams baby's 'death sentence' as miracle cystic fibrosis drug costs too much

Little Layla has cystic fibrosis (Rula Samara-Sellers / SWNS)

Layla and her mum Rula (Rula Samara-Sellers / SWNS)

Rula, who is head of customer experience at a financial company, said Layla was diagnosed with cystic fibrosis at three and half weeks old following a heel prick test. Rula said her daughter, who is susceptible to bacterial infections, now requires constant care and has made numerous trips to the hospital.

She says she felt the promise of the 'miracle drug' Kaftrio by specialists had kept them going through some “extremely difficult” situations. “It was the worst day of our lives because we didn’t know much about cystic fibrosis. Everything that you look at on Google isn’t that great. But what is factual is that it does lead to a shortened life expectancy and a life that is difficult.

“Typically, children born with cystic fibrosis, until they get to the age of one, shouldn’t experience many issues. But Layla has currently had multiple rounds of IVs in hospital, she’s had surgery, and she’s had multiple rounds of antibiotics. The only thing that was keeping us going was that she’ll be entitled to Kaftrio.”

Dad Connor with Layla (Rula Samara-Sellers / SWNS)

She has been in and out of hospital in her young life (Rula Samara-Sellers / SWNS)

Rula said Layla currently requires 12 scoops of micro 'Creon' tablets per day so she can digest her milk as her pancreas doesn’t produce enough enzymes. But she might be forced to take up to 60 of the adult-sized pills daily just to process certain kinds of foods when she's older.

In November last year, NICE published new 'draft' guidance that suggested Kaftrio, in their view, was likely too expensive for use on the NHS. Their report concluded: "The cost-effectiveness estimates were substantially above the range NICE considers an acceptable use of NHS resources."

This meant those who had already begun a course of the drug or been prescribed it could keep taking it, but new patients, like Layla, might be barred in the future. Rula said about hearing the news: “The whole of the community, us included, couldn’t sleep, we couldn’t stop crying. It was genuinely the worst experience in the whole entire world.

“What they are saying is anybody who is born after 2023, essentially, will no longer be entitled to it, so they are discriminating by age. Because you’ve missed the cut-off, you can just have a shortened life expectancy.”

Rula is now campaigning to ensure the voices of cystic fibrosis sufferers are heard ahead of an update from NICE, expected this week, and a final decision in March. She added: “I will fight tooth and nail to make sure it's approved in the UK. I will work with the Cystic Fibrosis Trust and they will support me all the way.”

A NICE spokeswoman aid: “NICE is liaising with key stakeholders to determine the most appropriate next steps for NICE’s assessment of these treatments. This will include exploring potential commercial solutions The NHS commercial framework for new medicines outlines how NHS England can negotiate complex confidential commercial agreements with companies.

“The feasibility of implementing a commercial access agreement is assessed directly by NHS England, and NHS England has indicated that it is willing to enter formal commercial negotiations with Vertex once the NICE committee outputs are clear for all parties. Negotiations would be entered into with the aim of ensuring the full portfolio of licensed cystic fibrosis medicines are available to everyone who may benefit, now and in the future, in a way that is fair to patients and fair to taxpayers.