Beyond Survival: Measuring What Matters to People with CF

Project Duration – 1 years

What is the research about?

CFTR modulators have transformed care for many people with CF, improving lung function and helping people live longer. But as people with CF live longer new health challenges are becoming more important, including mental health, side effects and other health conditions.

Traditional measures, such as lung function and weight, don’t tell the whole story. They don’t always capture how people feel, function or manage everyday life.

Understanding these experiences is essential to ensuring that advances in CF treatment translate into better lives, not simply better clinical results. To do this, health professionals and researchers need ways to capture what matters most to people living with CF, including aspects of health and wellbeing that may otherwise go unrecognised.

This project aims to develop a new questionnaire, known as a patient-reported outcome measure (PROM), designed specifically for people with CF by people with CF that can be used in this new era of CF treatment and care. It will help capture the aspects of health and wellbeing that matter most to people with CF, in their own words and from their own perspective.

Why does this matter for people with CF?

Everyone’s experience of CF is different. Even when clinical measures look good, people may still be managing significant treatment demands, mental health concerns or side effects.

A tool that captures these experiences could help health teams better understand each person’s needs, support more personalised care and identify concerns that might otherwise be missed. It could also help researchers and health teams better understand how treatments affect people’s everyday lives, not just their clinical results.

The questionnaire could also provide researchers with a better understanding of how treatments affect people’s everyday lives. This is particularly important as new therapies emerge and the goals of CF care continue to evolve. Looking beyond traditional clinical outcomes will help ensure that the experiences and priorities of people with CF remain central to evaluating treatment success.

What will the research do?

The research team will work with children and adults with CF, caregivers and health teams to:

• Identify the symptoms, concerns and health outcomes that matter most to people with CF today.
• Develop and test a questionnaire that captures areas not adequately covered by traditional clinical measures, including mental health, treatment burden, cognitive effects and treatment side effects.

People with CF and their families will help shape the questionnaire throughout the project to ensure it is relevant, accessible and meaningful across different ages and experiences.

What are the next steps?

The team will test the questionnaire to ensure it is reliable and useful across different groups of people with CF. If successful, it could be incorporated into routine clinical care and the Australian CF Data Registry, helping health teams identify unmet needs and make more informed decisions with their patients.

The goal: to look beyond survival and better understand what it takes for people with CF to live well.