August 03, 2022

2 min read

Cameron reports no relevant financial disclosures. Please see the study for all other authors’ relevant financial disclosures.

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Findings published in Chest highlighted the importance of routinely capturing treatment burden among individuals with cystic fibrosis in clinical trials.

“The recent introduction of CFTR modulator therapies is transforming the outcomes and prognosis for many people with cystic fibrosis, with evidence emerging that their introduction is associated with reduced use of other treatments,” Rory A. Cameron, PhD, MScPH, research scientist at Norwich Medical School at the University of East Anglia, England, and colleagues wrote. “To date, however, these innovations have been designed to be additive to existing regimens, so reduction of burden of treatment remains a priority.”

Cystic Fibrosis

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Researchers conducted an online discrete choice experiment among 103 adults with cystic fibrosis (mean age, 35 years; 52% women) who attended a specialist cystic fibrosis center. The experiment required all participants to evaluate hypothetical cystic fibrosis treatment profiles, defined by lung function impact, pulmonary exacerbations, abdominal symptoms, life expectancy, quality of life, use of inhaled medications and physiotherapy requirement.

The response rate for all participants was 37%. Overall, researchers observed that an average life expectancy improvement of 10 years or more resulted in the greatest impact on treatment preference, followed by a lung function increase of 15%. Individuals reported wanting to trade substantial reductions in such key outcomes to reduce their treatment time or burden.

The largest trade-offs were for quality of life improvement, with participants willing to accept a 8.2% predicted FEV1 reduction or 4.2 years of additional life expectancy.

Across this sample of individuals with cystic fibrosis, researchers identified three distinct subgroups that each placed substantially different importance on life expectancy and lung function compared with other attributes. In addition, participants were also willing to accept lung function or additional life expectancy reductions to reduce their treatment burden with:

  • 6.1% predicted FEV1 reduction or 3.2 years of additional life expectancy to fully stop physiotherapy;
  • 5.3% predicted FEV1 reduction or 2.7 years of additional life expectancy if abdominal symptoms improved with reductions in pancreatic enzyme replacement therapy; and
  • 4.4% predicted FEV1 reduction or 2.3 years of additional life expectancy to cut time spent on inhaled medications in half.

“The study provides important evidence on the relative importance of outcomes from a patient perspective that could be used alongside other scientific evidence in health technology appraisals to support decision-making for either the regulation or funding of cystic fibrosis treatments,” the researchers wrote. “Moreover, the comparative importance of treatment burden for patients suggests it should be considered as an important secondary outcome in cystic fibrosis when designing future prospective trails of novel therapies.”

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