The challenges facing people with cystic fibrosis

29 March 2023

Ellie Davies from Cystic Fibrosis Trust discusses the current challenges facing people with cystic fibrosis, and how the Trust are supporting them.

What is cystic fibrosis?

Cystic fibrosis is a rare, genetic condition affecting over 10,800 people in the UK. You are born with cystic fibrosis and cannot catch it later in life as it is caused by a faulty gene inherited from both parents. The gene affected by cystic fibrosis controls the movement of salt and water in and out of cells.

People with cystic fibrosis experience a build-up of thick sticky mucus in the lungs, digestive system, and other organs, causing a wide range of challenging symptoms affecting the entire body. Large amounts of treatment are required every day to stay well including antibiotics, steroids, medicines which break down mucus, pancreatic enzymes to help them break down food more effectively, and physiotherapy.

Living with cystic fibrosis

After a sustained campaign by the Trust and our community, some people with cystic fibrosis can now access transformative modulator therapies, which target the underlying cause of the condition. Part of the NHS deals to grant access included the agreement that data would be collected on their effectiveness and the National Institute of Health and Care Excellence (NICE) is soon to embark on an appraisal of the modulator therapies.  We have provided feedback to NICE on the different types of medicines people with cystic fibrosis need to take and whether NICE has included the appropriate outcome measures to capture all the benefits of these treatments. We have also shared the incredible impact these medicines have had on people with cystic fibrosis who are eligible to take them.

Not all people with cystic fibrosis are eligible for modulator therapies, and some cannot tolerate them. For those that cannot benefit, the future can feel uncertain. The Trust is actively supporting all of our community, as well as investing in research such as better ways to treat and manage cystic fibrosis related infections. We are also working hard to ensure that we make cystic fibrosis research an attractive area to invest in.

Improved healthcare outcomes for some has led to a need for psychological and social support as some of our community come to terms with a different, longer, and sometimes more uncertain future from the one they thought they may have. Some of those who are accessing modulator therapies may now need support accessing training or further education opportunities, or they might become anxious about the impact the treatment has on their body weight. People with cystic fibrosis require a high-calorie diet, often needing up to twice the number of daily calories. One benefit of the transformative modulator therapies is an increased ability to gain and sustain weight and we know this adjustment can be challenging for some.  Our patient experience surveys and staffing reports show that many people with cystic fibrosis can’t access the adequate support they need. 1 in 5 participating cystic fibrosis centres reported not having a clinical psychologist as part of their multidisciplinary team (MDT) and more than 1 in 2 centres reported not having a social worker as part of their MDT. Therefore, we have been calling for improved access to specialist cystic fibrosis psychologists and social workers to address and prevent mental health and wellbeing issues in our community.

We have always known that having cystic fibrosis means extra costs and lost income. Recent research from the University of Bristol in partnership with the Trust provides evidence of this. The findings show a typical family living with cystic fibrosis is now spending almost £6,800 more every year than someone without the condition. Food and energy bills were the biggest concern for people with cystic fibrosis and their families – both are essential to the health and quality of life for people with cystic fibrosis. Families with cystic fibrosis are more likely to be struggling with their finances than the public – 24% of adults with cystic fibrosis and 35% of parents of children with cystic fibrosis described meeting their bills each month as a ‘constant struggle’, compared to 17% of UK households overall. This reinforces our calls for urgent action from politicians and policymakers, including a review of the prescription charges exemption list and a commitment to continue to raise benefits in line with the cost of living.

Who is the Cystic Fibrosis Trust?

Cystic Fibrosis Trust is the charity uniting people to stop cystic fibrosis. We are a supportive community who improve care, speak out and race towards effective treatments for all.

The Trust’s work includes various projects to help us achieve our vision of a life unlimited for everyone with cystic fibrosis. From innovative research programmes that accelerate breakthrough science and therapeutics to greater support to clinical teams in the NHS and improvements in the way care is delivered. We work with our community to provide accurate and relevant information, support and advice based on what they tell us.