(355) Investigating Disparities in Cystic Fibrosis Clinical Trial Participation: One Cystic Fibrosis Center’s Self-Study

Background: Patients with cystic fibrosis (CF) identifying as part of a racial or ethnic minority group comprise an appreciable and increasing proportion of patients with CF in the United States. While there is limited data surrounding the racial and ethnic backgrounds of patients with CF in clinical trials, the published literature suggests that less than 20% of CF pharmaceutical clinical trials report the race or ethnicity of their study participants. When reported, patients with CF from racial or ethnic minority groups are underrepresented. We aimed to evaluate if the racial and ethnic backgrounds of patients with CF in clinical trials and pharmaceutical clinical trials at our CF Center reflect our overall patient diversity.

Methods: Leveraging the Cystic Fibrosis Foundation Patient Registry Reports, all patients with CF and at least one encounter at our CF Center between January 2016 and December 2020 were identified. Reported race and ethnicity were collected from the electronic medical record. Participation in clinical trials was tabulated from institutional records for the study period. A subgroup analysis, limiting the study population to the patients most likely to be eligible for a CF pharmaceutical clinical trial (age >= 12 years and heterozygosity or homozygosity for F508del), was also performed. Statistical analysis was performed using the Fisher’s exact test.

Results: 27.5% of patients with CF at our CF Center identify as part of a racial or ethnic minority group (Table 1). During the study period, 33.6% of patients with CF followed at our CF Center participated in one or more of 30 clinical trials and 25.5% participated in one or more of 22 pharmaceutical clinical trials. No pharmaceutical clinical trials enrolling children under the age of 12 years were performed. Overall, patients with CF identifying as non-Hispanic White were more likely to participate in a clinical trial than patients with CF from racial or ethnic minority groups (37.9% vs. 21.8%, P = 0.04). The same was true for pharmaceutical clinical trials (30.3% vs. 12.7%, P = 0.01). When limiting the study population to the patients most likely to be eligible for a CF pharmaceutical clinical trial, however, the disparity was no longer evident (40.2% vs. 54.5%, P = 0.5). Notably, patients with CF from racial or ethnic minority groups were more likely to be under the age of 12 years (56.4% vs. 11.7%, P = 0.0001) and less likely to be heterozygous or homozygous for F508del (60.0% vs. 73.1%, P = 0.09).

Conclusions: Ensuring that patients with CF from diverse backgrounds have access to and participate in clinical trials is key to advancing health equity. At first glance, patients with CF from racial or ethnic minority groups at our CF Center are underrepresented in clinical trials and pharmaceutical clinical trials. When considering common inclusion criteria for CF pharmaceutical clinical trials, however, our CF Center’s racial and ethnic diversity is, indeed, reflected in patient participation. We plan to further interrogate our data, evaluating each study and its unique inclusion and exclusion criteria, to delineate a more refined eligible subgroup. More broadly, further work should be directed toward obtaining complete and accurate demographic information for patients with CF participating in clinical trials and pursuing center-level self-study of clinical trial participation, evaluating for concordance with center patient diversity and assessing implemented recruitment strategies.

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