Abstract Rationale The introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators marked a shift from symptomatic management to disease-modifying therapy for individuals with cystic fibrosis (CF). Eligibility for CFTR modulators depends on specific genetic mutations, and substantial racial and ethnic disparities exist in research investigating mutation prevalence and subsequently in treatment. Most investigations have focused on genetic eligibility disparities, rather than prescribing patterns in clinical practice. The study objective was to explore racial and ethnic differences in CFTR modulator prescription patterns. Methods This was a retrospective cross-sectional analysis comparing the proportion of CF patients prescribed a CFTR modulator by race and ethnicity. Data were obtained from the TriNetX US Collaborative Network, which provided access to de-identified electronic medical records of over 124 million patients. Patients younger than 90 years of age with a diagnosis of CF were identified from 01/01/2012 until 12/31/2024. Patients were categorized by race and ethnicity into mutually exclusive groups: non-Hispanic White, non-Hispanic Black, non-Hispanic Asian, and Hispanic or Latino. The primary outcome was CFTR modulator prescription for ivacaftor, lumacaftor, elexacaftor, or tezacaftor at any time following diagnosis. Propensity score matching was performed for multiple characteristics including age at index, biological sex, tobacco exposure, comorbidities, BMI, and FEV1 % predicted. Statistical analyses were performed on the TriNetX platform and significance was assessed using Z-test to compare Black, Asian, and Hispanic or Latino cohorts using the White cohort as a reference group. Results Among 3,142 matched Black and White patients with CF, Black patients were significantly less likely to have received ivacaftor (7.8% vs. 22.2%, p 0.0001), lumacaftor (1.1% vs. 7.3%, p 0.0001), elexacaftor (7.1% vs 18.7%, p 0.0001), and tezacaftor (7.0% vs. 19.6%, p 0.0001), as illustrated in Figure 1. Hispanic or Latino patients (n = 3,538) had significantly lower prescription proportions than matched White patients (n = 3,538): ivacaftor (17.4% vs. 33.3%, p 0.0001), lumacaftor (3.4% vs. 11.3%, p 0.0001), elexacaftor (13.7% vs. 28.1%, p 0.0001), and tezacaftor (14.6% vs. 29.4%, p 0.0001). Asian patients (n = 456) were also less likely than White patients (n = 456) to receive ivacaftor (9.2% vs. 23.5%, p 0.0001), elexacaftor (7.9% vs. 19.7%, p 0.0001), and tezacaftor (7.9% vs. 20.8%, p 0.0001). Conclusion Racial and ethnic disparities persist in CFTR modulator prescribing across the United States. The population-level patterns confirmed by the findings of our study reflects a broader phenomenon of stratified access in precision medicine, in which innovations intended to individualize care inadvertently become differentially available along lines of race, class, and geography. This abstract is funded by: None
Hong et al. (Fri,) studied this question.