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

Black and Hispanic people with CF (PwCF) experience a greater burden of worse health outcomes related to pulmonary disease compared to White, non-Hispanic PwCF. Prior evidence suggests that these communities are underrepresented in clinical research in CF. We used clinical study data from four recent modulator studies (CHEC-SC, PROMISE, PROSPECT, and GOAL) as well as CF Foundation Patient Registry data on persons seen at the same centers while these studies were enrolling. We selected potential registry controls who had not enrolled into studies but were otherwise eligible based on inclusion and exclusion criteria. We assessed rates of study enrollment separately for Black race and Hispanic ethnicity versus non-Hispanic White PwCF as a reference. We used propensity weighted Poisson regression models to account for potential confounders in the relationship between race and study participation. This study analyzed the enrollment characteristics of 3,594 persons in modulator studies and compared them with 14,888 eligible individuals based on CFF Patient Registry data. Enrollees were younger (median age 19 years) and more likely to have at least three baseline visits and live within 30 miles of the study site. Multivariable analysis revealed racial/ethnic disparities in enrollment. 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 lower enrollment in PROMISE but higher enrollment in PROSPECT. The differences in enrollment rates were stronger at sites with fewer minority participants. These results suggest that Black and Hispanic PwCF are significantly less likely to be included in CF clinical research than White PwCF. Future research will include analysis of multiple CF clinical studies to evaluate if this relationship is upheld in studies with varying inclusion/ exclusion criteria and clinical sites.