Authors: Michael Li, MD, et al.

Journal: Hepatology. 78(S1):S1-S2154, October 2023.

Abstract:

Background: New AASLD guidance recommends consideration of all patients with primary sclerosing cholangitis (PSC) as potential candidates for clinical trials. However, the factors influencing patient interest and the barriers preventing inclusive and accessible participation in trials for PSC have yet to be elucidated. We investigated PSC patient interest in trial participation and identified factors associated with willingness to participate in drug trials.

Methods: Patient-Focused Drug Development (PFDD) is an FDA initiative to ensure that patient experiences and perspectives on drug development are captured. PSC Partners developed the international Our Voices survey to inform the development of the Externally-Led PSC PFDD meeting. Our team of researchers and patient advocacy representatives analyzed data from this survey, which included 797 adults with PSC.

Results: Patients identified slowing disease progression as the most important drug development outcome (67%). 89% of patients identified their hepatologist/gastroenterologist as who they would approach for advice about trials. 61% of patients reported being willing to participate in drug trials, while only 26% of patients reported ever being asked to participate. Notable frequently reported barriers to trial involvement included unknown long-term risks (71%), long travel times to study center (32%), a liver biopsy requirement (27%), and concerns about taking too much time off work (22%). On multivariable logistic regression, pruritus (aOR 1.61, 95% CI 1.07-2.44, p=0.023), jaundice (aOR 0.34, 95% CI 0.19-0.62, p<0.001), and inflammatory bowel disease (aOR 0.64, 95% CI 0.42-0.98, p=0.042) were associated with willingness to participate in disease-modifying therapy trials (Figure). Pruritus (aOR 2.25, 95% CI 1.50-3.39, p<0.001) was also independently associated with willingness to participate in symptom treatment trials.

Conclusion: Hepatologists/gastroenterologists are trusted by PSC patients and must be responsible for closing the gap between trial interest and participation. Pruritus is one of the most influential symptoms associated with willingness to participate in drug trials and can serve as an indicator of patient interest in trial participation. Patient advocacy groups like PSC Partners can play a crucial role in further investigation to understand the gaps between clinical trial interest, referral, and participation, with an emphasis on promoting equity and diversity in clinical trials.