Evidence-based valuation of patient-centred outcomes in Cystic Fibrosis II

Background

Cystic fibrosis (CF) is a genetic condition that affects more than 10,000 people in the UK. People with CF accumulate thick, sticky mucus in their lungs, gut and other organs. They need to follow a time-consuming treatment routine every day to maintain their health. For example, most adults with CF dedicate between one and four hours every day to their treatments. This treatment burden can impact everyday life significantly for people with CF and their families, interfering with their work, school, university or home life. It often makes routine activities more difficult, and limits social activities. In addition to their usual treatments, a lot of extra treatment – such as antibiotic injections, hospital visits and stays in hospital – is necessary when they are unwell. Feedback from the CF community has indicated that reducing treatment burden is considered to be their top research priority.

Health-related quality-of-life and treatment burden are particularly important to patients and caregivers but are poorly considered in assessments of new treatments. This research aimed to address this issue and ensure more accurate valuation of patient-centred outcomes within the decision-making process for access to new treatments and clinical practice. In the longer term, this research will bring a more precise understanding of what it means to say that a CF treatment “works”.

HEP02 was a single-centre study, which found that while people with cystic fibrosis prioritised improving their life expectancy, but were willing to accept reductions in this outcome in order to reduce their treatment burden. This study helped generalise these findings to the broader, UK CF population, and investigated how preferences vary across sub-strata of the population.

Project aims

The study addressed three questions:

1. What are the treatment outcome priorities amongst people with CF, and what differences are there in priorities in different CF populations (for example those on CFTR modulators vs those ineligible for these treatments)?
2. Can attitudinal measures predict differences in preferences?
3. What level of decline in lung function are people with CF willing to accept in order to reduce treatment burden?

Project activity

Data collection for this study has now completed and analysis is currently underway.

Next steps

Study findings will be presented at the European Cystic Fibrosis Conference in June 2026 and subsequently published in an open-access peer-reviewed journal.

The results will provide new evidence on the treatment outcomes most valued by people with cystic fibrosis and how these preferences vary across different patient groups. In particular, the study will explore whether the introduction of highly effective CFTR modulator therapies has changed how people with cystic fibrosis perceive their condition and the trade-offs they are willing to make between treatment burden and health outcomes.

This work will help ensure that outcomes important to patients, such as quality of life and treatment burden, are better reflected in the evaluation of new treatments and in decision-making around access to therapies.

Papers and resources

This project can be viewed in a case study format for easy accessibility

Who was involved?

Co-Principal investigator

Dr Siobhan Carr (s.carr@rbht.nhs.uk), Royal Brompton and Harefield NHS Foundation Trust and Imperial College London

Health economics lead:

Rory Cameron (rory.cameron@uea.ac.uk), NIHR Applied Research Collaboration East of England Measurement in Health and Social Care Theme

Contact us

Dr Rory Cameron, rory.cameron@uea.ac.uk