How your expertise of living with CF is defining what is measured in future research

For a long time, people with CF and their families have clearly said ‘it’s not just lungs’ as they juggle every aspect of CF on a daily basis. From the point of diagnosis, the aim is to maintain a balance that provides stability of health, prevent decline and support enjoyment and fulfillment – having confidence in their health to be able to make plans for social lives, holidays, education, career and family aspirations or ambitions. To truly live with CF. 

The treatments and medications people with CF take have each been carefully and rigorously tested in development, but each in isolation. Each treatment has its own benefits – one to thin mucus, one to open airways, one to treat reflux, one to help with digestion of foods, one to control blood sugars, one to fight infections. However, each has the potential to tip the balance of another aspect of CF being carefully managed, re-introducing some instability. 

For instance, introducing a new antibiotic to treat a lung infection can disrupt the gut, leading to symptoms such as bloating, diarrhea or discomfort – symptoms that people are already trying to avoid through carefully managing their enzyme intake. Similarly, so many factors can disrupt blood sugar levels making the management of CF diabetes even more challenging than it already is. 

Taking this multi-system nature of CF into account, the community has told us they want a holistic approach. When research seeks to address one factor of CF, these other factors need to be considered at the same time by including some additional measures during clinical trials to understand them. 

Following the roll-out of modulators like Kaftrio, people with CF are talking about symptoms of their condition beyond their lung function. As FEV₁ has stabilised for many people, this has become a lower priority for monitoring health. It is still an important marker, but is not always the key defining factor of someone’s overall health and how they are feeling. Many people with CF are relearning their condition and focusing more on other measures or markers of health. These learnings also have the potential to help those who haven’t been able to benefit from a modulator medicines, ensuring the multi-system nature of the condition is observed. 

These insights from the lived experience of people with CF resonated with the clinical community and it was recognised that outcome measures (the specific things researchers look at to see if their study or treatment is working) used in future clinical trials need to take this into account. Ideally a set of a few core outcome measures would be collected in every CF trial, no matter what the focus of the trial was, to give a more complete picture of the impact any new treatment could have on overall health. 

Another advantage of this approach means the findings of several clinical trials can be compared or combined together, making results even more powerful. 

In June 2021, the Trust hosted an engagement event to discuss the idea of developing a Core Outcome Set to influence the future of CF research. There was a great deal of interest and agreement to start a project to develop the Core Outcome Set for CF (COS-CF). Funding was granted and global engagement started so we could set out an agreement on what measures should form this core set of outcomes to be used in CF research.