The CF STORM Study: A Registry-Based Trial Investigating Therapy Rationalisation in People with Cystic Fibrosis

Why was the research needed?

Cystic fibrosis (CF) affects more than 10,000 people in the UK and remains a lifelong, life-limiting condition. Although advances in treatment and care have significantly improved outcomes, people with CF often spend several hours each day managing complex treatment regimens, including nebulised therapies to maintain lung health and prevent infections. This substantial treatment burden can affect daily activities, social participation and quality of life. Feedback from the CF community has consistently identified reducing treatment burden as a top research priority, aligning with the UK Government's Rare Diseases Framework.

What did we do?

CF STORM was designed to investigate whether people with cystic fibrosis who are established on Kaftrio, a medicine that target the underlying cause of CF, can safely stop taking mucoactive nebuliser treatments, including DNase and hypertonic saline, without a significant decline in respiratory function. The study aimed to address a key priority identified by the cystic fibrosis community: reducing treatment burden. Alongside the clinical trial, we led the economic evaluation to assess the cost-effectiveness of treatment simplification. Patient follow-up was completed in September 2025, and analysis of the study data is currently underway.

This project is related to the Evidence-based valuation of patient-centred outcomes in Cystic Fibrosis project.

What did we find? 

We found that some people established on Kaftrio could stop certain nebulised treatments without a meaningful decline in lung function, reducing daily treatment burden, with estimated NHS savings of around £5,000 per patient over a year. The findings have gained international recognition and could support treatment simplification for eligible people with CF. 

How did we work with communities, services and organisations?

CF-STORM was a large collaborative study involving people with cystic fibrosis, clinicians, researchers, NHS organisations, charities and trial specialists from across the UK. Led by Alder Hey Children's NHS Foundation Trust, the study brought together partners from Cardiff University, Imperial College London, Liverpool Clinical Trials Centre, Oxford University Hospitals NHS Foundation Trust, Royal Papworth Hospital NHS Foundation Trust, Queen's University Belfast, Royal Brompton and Harefield NHS Foundation Trust, the Cystic Fibrosis Trust, University College London, the University of East Anglia, the University of Manchester and the University of Nottingham. 

What will the impact and benefits of this research be? 

Our findings have the potential to reduce daily treatment burden for people with cystic fibrosis, giving them more time for work, education, family life and other activities that matter to them. They also support treatment simplification for eligible patients. By improving understanding of the personal and economic value of CF treatments, the research can inform NHS funding decisions and investment in interventions that matter most to people with cystic fibrosis. The robust clinical and economic evidence generated by the study can also support future clinical practice and decision-making in cystic fibrosis care in the UK and internationally.

How will we share and apply the findings? 

The findings have been shared internationally, including at the European Cystic Fibrosis Conference, with clinical and health-economic results published as conference abstracts in the Journal of Cystic Fibrosis. Full peer-reviewed manuscripts are currently under review.

The study's innovative approach to assessing a treatment 'stopping strategy' following the introduction of a highly effective therapy will also be reported and may be relevant to other conditions where transformational treatments are becoming available. A patient-centred knowledge transfer programme will share key findings with people with cystic fibrosis and other stakeholders through the Cystic Fibrosis Trust, including its social media channels, helping to make the findings accessible and inform future care and decision-making.