Cystic fibrosis patients in England have health drastically improved by ‘miracle’ NHS treatment

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Thousands of cystic fibrosis patients in England have had their health drastically improved by a “miracle” treatment, allowing one to give birth to a baby, since the NHS made it available exactly one year ago today.

In the past 12 months, NHS patients in England benefited from one of the fastest rollouts in the world of the cystic fibrosis triple-therapy, Kaftrio, alongside treating hundreds of thousands of seriously unwell patients with COVID.

Nine in 10 people with cystic fibrosis in England now have access to therapies which patients describe as a “miracle”, thanks to a series of medicines deals secured by the NHS.

There are around 8,000 people living with cystic fibrosis in England – the second highest proportion in the world.

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.

Kaftrio described by patient groups as a “revolutionary drug” has benefitted people like Laura whose parents were told that she would be lucky if she reached the age of 30.

Laura, now 30, started the treatment a year ago at Royal Papworth Hospital, allowing her health to drastically improve so that she could give birth to a baby boy called Louis.

Laura said:

“My Kaftrio journey has been incredible, not just for me and my health but to start a family as well – to be given this one amazing child that I have wanted for so long.

“Within the space of a few hours of starting the treatment it started to work – it is the miracle drug.”

The NHS Long Term Plan committed to using cutting edge treatments and therapies to save and improve patients’ lives.

Professor Stephen Powis, NHS National Medical Director for England, said: 

“Despite the pandemic and the incredible efforts to treat more than 420,000 with coronavirus in hospitals, NHS staff have also delivered one of the fastest rollouts of Kaftrio in the world, transforming the lives of patients with cystic fibrosis.

“Stories of people like Laura, who have gone on to have miracle babies, is yet another example of how the NHS is offering the latest life-changing treatments for patients and their families.

“Kaftrio is one of many cutting-edge treatments that the NHS has been able to secure at a price that is also fair to taxpayers.”

The deal is one of a package made possible by the NHS’ advanced commercial capabilities, which have delivered an innovative gene-therapy to cure blindness and the world’s most expensive drug, Zolgensma, which can cure paralysis in babies.

Dr Keith Brownlee, Director of Medical Affairs at the Cystic Fibrosis Trust, said:

“Kaftrio is truly a revolutionary drug. It doesn’t just treat the symptoms of cystic fibrosis, it treats the underlying causes of the condition which in turn helps to boost lung function and which will extend lives.”

The drug according to patients like Jack significantly improves lung function, helping them to breathe more easily.

Jack Harris, from Nottinghamshire, 32, has cystic fibrosis and began using Kaftrio in October last year. His lung function has increased 61% to 93%, and he has gained 12 kilograms and is now at a healthy weight.

He said: 

“To hear that Kaftrio had been approved for use on the NHS was nothing short of amazing. It was like all my birthdays and Christmases had come at once.

“The change in quality of life has been miraculous. I can enjoy things more knowing that I won’t get out of breath so quickly. I can laugh without breaking into a coughing fit. I can play golf for hours and not get out of breath. This year, I did a 25 mile walk in nine hours – something I’d never have been able to do without Kaftrio.”

Health and Social Care Secretary, Sajid Javid, said: 

“I’m delighted to see so many patients benefitting from this life-changing cystic fibrosis drug. Laura’s incredible story shows how these treatments can truly transform people’s lives, giving them a better quality of life for longer.

“I want to thank NHS staff for making this one of the fastest rollouts of Kaftrio in the world – a remarkable accomplishment. I continue to be inspired by their spirit and determination, especially in the midst of the pandemic, to improve as many lives as possible.”

Andrew Wilfin, UK and Ireland Medical Director, Vertex said: 

“The innovative agreement that was reached between Vertex and NHS England in advance of the marketing authorisation of Kaftrio meant immediate access for eligible patients as soon as the licence was granted in August 2020.

“Since then, our data shows that the uptake of the medicine in England amongst eligible patients is one of the fastest we have seen globally.  We are delighted that so many people with CF have been able to benefit and we continue to collect real world data which further demonstrates the value that the medicine brings to patients”.

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