(52) Participation in interventional drug trials among minority populations at an urban adult cystic fibrosis center

Background: Underrepresentation of minorities in clinical trials is currently an active area of research. Cystic fibrosis transmembrane receptor (CFTR) modulators are an efficacious treatment for specific CFTR mutations in persons with cystic fibrosis (pwCF), associated with improvement in lung function, and respiratory-related quality of life, decrease in pulmonary exacerbations, and reduced sweat chloride levels [1]. Although the majority of pwCF are Caucasian, prevalence among minority patients is increasing. Of the over 31,000 patients enrolled in the 2020 CF Foundation Patient Registry (CFFPR), 91.6% were White, 3.5% were Black, and 4.9% were Asian, mixed-race, or other. 9.6% identified as Hispanic [2]. Prior studies have demonstrated that pwCF from minority groups are less likely to be eligible for CFTR modulator therapy [3]; however, it is concerning that those who are eligible may still remain underrepresented in interventional clinical trials. Reasons for underrepresentation are not well characterized. As a CFF Therapeutics Development Network site, we conducted a study to describe eligibility for clinical intervention trials of minority patients at our adult CF program and determine if minorities were enrolled at an equitable rate.

Methods: A query of the Columbia University Adult CFFPR database was conducted to determine eligibility for the previously conducted CFTR modulator trials, VX659-102, VX659-103, and VX445-104. We compared the proportion of subjects that participated to the proportion of eligible subjects by race and ethnicity. The patient population is reported as Non-Hispanic White, Black, Hispanic (Black, White, and other race Hispanic), and Other races (Other races, 2 or more races, and Asians).

Results: 305 adult pwCF are enrolled in our CFFPR, 262 (86%) self-identify as Non-Hispanic White, 10 (3.2%) as Black, 24 (7.8%) as Hispanic, 9 (2.9%) as other. Of these, 92 patients met inclusion criteria for the VX659-102, VX659-103, and VX445-104 trials. Of the 92 eligible patients, 86 (93.5%) identified as Non-Hispanic White, 4 (4.4%) identified as Hispanic, and 2 (2.2%) identified as Black. At our center, a total of 22 pwCF were enrolled. Twenty-one (95.5%) of enrolled subjects were Non-Hispanic White and one enrolled subject (4.5%) was Hispanic; no Black pwCF were enrolled (Table 1).

Conclusions: Of the eligible Non-Hispanic White pwCF, 24.4% were enrolled in the triple combination modulator clinical trials. However, of 6 eligible minority pwCF, only 1 was enrolled, representing 17% of the eligible participants for that category. Our data demonstrate that at a single adult urban CF center, minorities were underrepresented in CFTR modulator interventional trials. Further efforts are needed at both center and national levels to identify barriers to recruitment and participation of minority pwCF in clinical trials.

Acknowledgements: The Gunnar Esiason Adult CF and Lung Program

References: [1] Zaher A, ElSaygh J, Elsori D, ElSaygh H, Sanni A. A review of Trikafta: Triple cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy. Cureus. 2021;13(7):e16144. https://dx.doi.org/10.7759/cureus.16144
[2] Cystic Fibrosis Foundation. Patient registry 2020 annual report. https://www.cff.org/sites/default/files/2021-11/Patient-Registry-Annual-Data-Report.pdf; 2021.
[3] McGarry ME, McColley SA. Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype. Pediatr Pulmonol. 2021;56(6):1496–503. https://doi.org/10.1002/ppul.25285