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Key takeaways
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Elexacaftor/tezacaftor/ivacaftor (ETI) therapy improves health outcomes for many people with cystic fibrosis (CF), but experts are raising concerns about disparities in prescribing practices.
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When compared to non-Hispanic white people with CF, people with CF from racial and ethnic minoritized groups are less likely to be eligible for ETI due to having CF gene mutations that aren’t known to be responsive to ETI.
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In addition, when compared to non-Hispanic white people, those eligible for ETI from racial and ethnic minoritized groups were less likely to be prescribed the medication and initiation of therapy took longer.
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The research team speculated these disparities are due to many reasons and called for further investigation to promote equitable prescribing practices.
Research study background
Most people in the U.S. with cystic fibrosis (CF) receive standard-of-care treatment at accredited CF centers, yet compared to non-Hispanic white individuals, health disparities — including delayed diagnosis and increased morbidity and mortality — persist among people with CF of minoritized backgrounds.
CFTR modulators have transformed CF treatment, and the approval of the combination therapy elexacaftor/tezacaftor/ivacaftor (ETI), and its brand name Trikafta, expanded highly effective modulator therapy access to 90% of eligible people with CF ages 6 and older. ETI significantly improves patient outcomes, and early initiation may prevent against and even reverse complications of CF. However, people with CF of minoritized racial and ethnic groups have a greater prevalence of gene mutations that are known not to be responsive to ETI treatment and are less likely to be eligible for ETI treatment compared to non-Hispanic white people, which raises concerns about further exacerbating health disparities.
As it turns out, these concerns are valid. This study, led by experts in the Mike McMorris Cystic Fibrosis Research and Care Center at Children’s Hospital Colorado, is the first to analyze disparities in prescription rates and time to initiation among racial and ethnic groups eligible for ETI.
“We already knew that Black and Hispanic people with CF were less likely to be eligible for ETI because of more commonly having CF gene mutations not responsive to this treatment. To our surprise and concern, they were also less likely to be prescribed ETI, even when eligible. This is an important health disparity that we did not know about.”
- KAMYRON JORDAN, MD
Children’s Colorado investigators analyzed data from the U.S. CF Foundation Patient Registry, the largest worldwide CF dataset. Researchers identified 26,539 people with CF in the U.S. among all racial and ethnic groups in the Cystic Fibrosis Foundation’s care center network from 2020 to 2022. Compared to non‐Hispanic white people with CF, all minoritized racial and ethnic groups had lower percentages eligible for ETI treatment, and the percentage of people prescribed ETI in each group was significantly lower. Further, the time it took for ETI treatment to be initiated was also longer for every other racial and ethnic minority group compared to non-Hispanic white people, even when adjusted for age, lung function and insurance status.
Relevance to practice
These differences did not appear related to access to CF center care; clinic visits within the study period were similar across all racial and ethnic groups and averaged 4 to 4.6 visits per year. Instead, study authors suspect the cause is multifactorial. The team’s findings, published in a letter to the editor of Pediatric Pulmonology, included an urgent call to improve understanding of these disparities in prescribing ETI to actively address systemic barriers and potential biases.
Featured researchers
Kamyron Jordan, MD
Pediatric Pulmonary Fellow
The Breathing Institute
Children's Hospital Colorado
Scott Sagel, MD, PhD
Pulmonologist
Children's Hospital Colorado
Professor
Pulmonary Medicine - Department of Pediatrics
University of Colorado
Timothy Vigers, MS
Research instructor
Pediatrics-Endocrinology
University of Colorado School of Medicine
