Treatment preference amongst people with cystic fibrosis: the importance of reducing treatment burden

Rory A. Cameron, Daniel Office, Jessie Matthews, Mark Rowley, Janice Abbott, Nicholas J. Simmonds, Jennifer A. Whitty, Siobhán B. Carr

Research output: Contribution to journalArticlepeer-review

Abstract

BACKGROUND
There is a growing consensus that the perspective of the patient should be considered in the evaluation of novel interventions.

RESEARCH QUESTION
What treatment outcomes matter to people with cystic fibrosis (CF), and what trade-offs would they make to realise these outcomes?

STUDY DESIGN AND METHODS
Adults attending a specialist CF centre were invited to complete an online discrete choice experiment (DCE). The DCE required participants to evaluate hypothetical CF treatment profiles, defined by impact on lung function, pulmonary exacerbations, abdominal symptoms, life expectancy, quality of life, inhaled medicines usage, and physiotherapy requirement. Choice data were analysed using multinomial logit and latent class models.

RESULTS
103 people with CF completed the survey (median age 35 years (range 18-76); 52% female; mean ppFEV1 69% (SD 22)). On average, an improvement in life expectancy by 10 years or more had the greatest impact on treatment preference, followed by a 15% increase in lung function. However, it was shown that people would trade substantial reductions in these key outcomes to reduce treatment time or burden. Preference profiles were not uniform across the sample: three distinct subgroups were identified, each placing markedly different importance on the relative importance of both life expectancy and lung function compared to other attributes.

INTERPRETATION
The relative importance of treatment burden to people with CF, compared to life expectancy and lung function suggests it should be routinely captured in clinical trials as an important secondary outcome measure. When considering the patient perspective, it is important that decision makers recognise that the values of people with CF are not homogenous.
Original languageEnglish
JournalChest
Early online date19 Jul 2022
DOIs
Publication statusE-pub ahead of print - 19 Jul 2022

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