Representation of Minoritized People with Cystic Fibrosis in CF Therapeutics Development Network Clinical Trials

RATIONALE: Black and Hispanic people with CF (PwCF) experience worse health outcomes related to pulmonary disease compared to White, non-Hispanic PwCF. Improving outcomes in minoritized PwCF is a crucial need in the CF community, but prior evidence suggests that these communities are underrepresented in CF clinical research. OBJECTIVE: To determine whether CF clinical studies are representative of the overall CF population. METHODS: The study cohort included participants from recent cystic fibrosis transmembrane conductance regulator modulator studies (CHEC-SC, PROMISE, PROSPECT, and GOAL). Persons seen at the same centers who were eligible for these studies based on inclusion and exclusion criteria, but who did not enroll in these studies were used as a comparator cohort from the CFF Patient Registry (CFFPR). Rates of study enrollment were assessed separately for Black race and Hispanic ethnicity versus non-Hispanic White PwCF. Propensity weighted Poisson regression models were used to account for potential confounders in the relationship between race and study participation. RESULTS: Demographic characteristics were compared between 3,594 PwCF in the four modulator studies with 14,888 individuals from the CFFPR who were eligible, but did not enroll. Enrollees were younger (median age 19 years), more likely to have had at least three clinic visits per year, and live within 30 miles of the study site. Black and Hispanic PwCF were less likely to have private insurance and less likely to live far from study centers. Multivariable analysis revealed that Black individuals were significantly less likely than non-Hispanic White individuals to enroll in CHEC-SC and PROMISE, with a 17% and 32% lower likelihood, respectively. Hispanic individuals had a 34% lower enrollment rate than non-Hispanic White individuals in PROMISE but higher enrollment in PROSPECT. Differences in enrollment rates were greater at sites with fewer minority participants. CONCLUSION: These results suggest that Black and Hispanic PwCF are, overall, significantly less likely to be included in CF clinical research than non-Hispanic White PwCF. Future research is needed to better understand if these differences are upheld in studies with varying inclusion/exclusion criteria and clinical sites.