South West patients to benefit from “Life-changing” Cystic Fibrosis Drug

Patients in the South West have welcomed news that hundreds of people with cystic fibrosis across the region might benefit from a ‘transformative’ treatment.

NHS patients will be among the first in Europe to be prescribed Kaftrio, which significantly improves lung function, helping people with cystic fibrosis to breathe more easily and enhancing their overall quality of life.

The treatment – known as the ‘triple combination therapy’ – has been given the green light by European regulators, setting live a deal struck by NHS England to get the drug onto the frontline of patient care as soon as it was licensed.

The immediate availability of Kaftrio, alongside existing treatment options previously negotiated between NHS England and Vertex Pharmaceuticals, means that most patients with CF – more than 7,000 people in England, the equivalent of 600 patients across the South West – can benefit from a therapy which tackles the underlying causes of the condition.

Zara Teare, a patient at University Hospitals Plymouth NHS Trust, welcomed the news about the new drug and said:  “Being given the chance to have Trikafta [the marketing name used for Kaftrio in the USA] is like being given a second chance in life. A potential 10% increase seems like the impossible, I’ve forgotten what it feels like to have a lung function like that… or to even take in a big deep breath. It’s really true when people say this drug can be life changing, there’s no words to describe that feeling of being given that chance.”

Cystic fibrosis accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year, as each patient stays in hospital for approximately 10 days. Although the amount of people who die from cystic fibrosis is reducing, unfortunately 111 people died in 2012.

Today’s watershed moment builds on previous NHS support given to people with cystic fibrosis that saw thousands of people with the condition receive devices allowing medics to monitor their condition remotely, as part of the NHS drive to give more people connected, supported and personalised care in their own homes, saving them from having to make trips to hospital.

Welcoming the news that the NHS can start prescribing the new treatment, Dr Michael Marsh, Medical Director NHS England and NHS Improvement in South West said:  “At the same time as staff came together to put in a herculean effort to treat patients for coronavirus in hospitals, they also continued providing round the clock care for patients with long term conditions such as cystic fibrosis, and this landmark deal has put NHS patients at the front of the queue for transformational treatment.”

The deal for the triple combination therapy was made possible by the NHS working in partnership with the National Institute for Health & Care Excellence (NICE), the internationally renowned body for assessing the clinical and cost effectiveness of new treatments and technologies.

The deal will last for four years to allow for further data to be collected to better understand the impact for patients of this game-changing treatment.

In clinical trials Kaftrio has been shown to significantly improve the lung function of cystic fibrosis patients with two copies of the F508del mutation or one copy of the F508del mutation with a Minimal Function mutation, enabling them to breathe more freely and enhancing their overall quality of life.