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 aims 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 will help generalise these findings to the broader, UK CF population, and will investigate how preferences vary across sub-strata of the population.
The study will address three questions:
- 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)?
- Can attitudinal measures predict differences in preferences?
- What level of decline in lung function are people with CF willing to accept in order to reduce treatment burden?
We are currently in the project start-up phase, with fieldwork due to commence in summer 2022.
Outputs will encourage the NHS to deliver the interventions that are most valued by CF patients. Patients will benefit because a better understanding and valuation of quality-of-life and treatment burden will enable better informed decisions around the funding of interventions. Decision-makers will benefit from a more rigorous evidence base on which to make funding decisions. Society more broadly will benefit from increased health system efficiency (that is, more of the health outcomes most important to patients will be provided for the same cost).
In the longer term, this research will support patient access to CF treatments and help ensure the sustainability of our NHS. Moreover, the focus on outcomes that are important for patients will be publicised and promoted to clinicians, leading to an anticipated uptake of their routine measurement in CF clinical care. This project has the potential to directly and rapidly benefit patients through promoting a focus on patient-reported outcomes in clinical practice.
Who is involved?
Dr Siobhan Carr (email@example.com), Royal Brompton and Harefield NHS Foundation Trust and Imperial College London
Health economics lead:
Rory Cameron (firstname.lastname@example.org), NIHR Applied Research Collaboration East of England Health Economics and Prioritisation in Health and Social Care Theme
Dr Rory Cameron, email@example.com