Barriers for clinical trial enrollment in cholangiocarcinoma patients.
Authors: Pear Samantha , Bachini Melinda , Gardett Isabel , Lindsey Stacie , Delcorso-Ellmann Teresa , Abdel-Wahab Reham 
Published: January 2022

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Background: Cholangiocarcinoma (CCA) is a rare and aggressive cancer, with most patients presenting at an advanced stage with few treatment options. In the past decade, the number of clinical trials available to CCA patients has increased significantly. Recent clinical trials for FGFR2 and IDH1 inhibitors showed significant improvement in patients’ overall survival and progression-free survival and led to the approval of three new targeted therapies over the last year and a half. Unfortunately, a small percentage of CCA patients were eligible for this treatment. As the standard treatment has limited efficacy, there is a critical need for CCA patients to have access to and enroll in clinical trials. Our study aims to evaluate the current clinical trial enrollment rate in CCA and barriers that limit patient enrollment. Methods: We surveyed CCA patients and caregivers during the 2021 CCA Virtual Annual Conference to leverage the large gathering of the CCA community as they learned about the clinical trial landscape and opportunities available to them. Results: A total of 204 attendees participated in this study. 62.3% were females, 89.2% identified as white, 72.5% of patients were diagnosed with intrahepatic CCA, followed by 13.2% Perihilar, and 8.3% distal CCA. 60.3% of patients were receiving active cancer therapy at the time of the survey. Out of those patients, 56.9% were receiving chemotherapy, 25.2% targeted therapy, and 6.5% immunotherapy. The remaining 11.4% received surgery+/- neoadjuvant therapy, liver transplantation, local therapy, radiotherapy alone or combined chemoradiotherapy. Only 1.5% of the current patients surveyed did not carry medical insurance. Some patients reported that financial hardship negatively impacted pursuing medical care; including their inability to secure a second opinion, to travel long distance to a comprehensive cancer center, and to access clinical trials. 27% had enrolled in clinical trials, with 25.5% of those enrolled in two clinical trials over the course over their treatment. Among patients who were told that they were not eligible for clinical trials enrollment, 34.8% reported it was due to not having actionable mutations, 17.4% did not have measurable tumors, 8.7% had insufficient tissue biopsy, and 40% of patients had other barriers, including no clinical trials in their treating cancer center, advanced age, prior chemotherapy, prior tumor resection, presence of brain metastasis, or other primary cancer. Conclusions: Although, more CCA clinical trials are now available, the number of patients enrolled in clinical trials is still low. Based on our analysis, there is a need to provide more clinical trials to CCA patients. Additionally, there is a need for a greater geographic diversity of trials sites, more expanded access programs, and designing clinical trials for patients who don’t have actionable mutations will provide treatment opportunities and hope for more diverse patients.