Q&A Patient Preference and Treatment Priorities in Cystic Fibrosis

Most patients with cystic fibrosis (CF) will require treatment throughout their lifetime, including frequent hospital stays as well as an intensive daily regimen of therapy. The daily time needed for CF treatment has been estimated to take more than 1.5 hours. This can pose a serious impact on a patient’s daily life activities and contributes to the overall burden of disease.

Rory A. Cameron, PhD, MScPH, and colleagues developed a study to explore what treatment outcomes are considered valuable to patients with CF and what trade-offs they may be willing to make to attain these out-comes. Dr. Cameron recently discussed the findings of this study with Physician Weekly as well as its application to practice.

What prompted you to study treatment burden in association with attaining desired outcome in CF?

The study was partly developed in response to the 2017 James Lind Alliance Priority Setting Partnership in CF, which found that simplifying treatment burden was the number one research priority among the CF community. It remains in the top 10 for the refreshed 2022 version.

Despite this high level of priority in the community, treatment burden and HRQOL outcomes are poorly considered in the assessment of new treatments. In this study, one of the first to address the treatment preference of people with CF, we sought to quantify the importance of improvements in patient-centered outcomes relative to more familiar clinical outcomes, such as lung function.

What are the most compelling findings for practicing physicians?

The results must be considered against the limitations of the study design.

It was a single-center study, albeit the largest adult CF center in the UK—so caution should be exercised in generalizing the results to the wider population.

It used a stated preference methodology; respondents described preferences for hypothetical scenarios that may not necessarily reflect their real-world behavior.

It was initiated prior to the introduction of ETI [elexacaftor/tezacaftor/ivacaftor] therapy, and the prognosis for many people with CF is now quite different—so preference patterns and priorities are consequently likely to have shifted (Table).

Those limitations notwithstanding, our findings indicate that the most important outcomes for people with CF relate to life expectancy and lung function—in general agreement with the universal use of the latter as a primary outcome measure in clinical trials in CF. Our research provides further evidence that reducing the treatment burden is important to patients.

Secondly, we found that there was considerable variation in the levels of importance that different people placed on each of these outcomes, and we believe this variation is driven more by attitude than by clinical or demographic characteristics.

Is there anything else you’d like to mention?

I think it is important that clinicians recognize the variation in priorities for people with CF regarding treatment outcomes. Elsewhere, we have found that perceived treatment burden is only moderately correlated with treatment volume. Clearly, there are unobserved factors that influence patient priorities, highlighting the importance of entering a dialogue with the patient as an individual when discussing treatment plans.

Pierre-Régis Burgel, professor of respiratory medicine at Université Paris Cité, wrote a great companion editorial article discussing our study, where he highlights the importance of considering patient preferences and scientific evidence. While the SIMPLIFY study in the United States showed no differences when discontinuing hypertonic saline or dornase alpha for people on ETI over a short 6-week period, and we are involved in a similar 1-year study in the UK (CF-STORM), there is currently limited evidence as to the safety of withdrawing any CF treatments.