Cholangiocarcinoma (CCA) is a rare, aggressive, highly heterogeneous gastrointestinal cancer with high mortality and a rising incidence. CCA is typically asymptomatic in the early stages, resulting in frequent late-stage diagnoses with an overall poor prognosis. Surgery is the only available curative treatment approach; however, it is only effective in the early stages of the disease. Available therapeutic options for later stages are limited and offer only modest benefits. Thus, there is a desperate need for patients with CCA to gain access to clinical trials to help improve survival.
The past 2 years have seen breakthroughs in the CCA clinical trial space. Three new targeted therapies—including 2 FGFR2 inhibitors infigratinib (Truseltiq) and pemigatinib (Pemazyre) and 1 IDH1 inhibitor ivosidenib (Tibsovo)—received FDA approval after showing a significant improvement in overall patient survival and progression-free survival. Yet, despite 141 phase 1 through 4 clinical trials currently enrolling patients with CCA worldwide, inclusion and exclusion criteria are limiting, affording few patients access to cutting-edge trials.
Hence, the Cholangiocarcinoma Foundation (CCF) Research Team created a survey to evaluate clinical trial enrollment rates for patients with CCA and the barriers that limit enrollment. We shared this survey with patient and caregiver attendees at the 2021 CCF Annual Conference, and 204 attendees participated. The survey results were presented at the 2022 American Society of Clinical Oncology Gastrointestinal Symposium.
Of the respondents, 62.3% were women and 89.2% were white. Specific patient diagnoses included intrahepatic (72.5%), perihilar (13.2%), and distal (8.3%) CCA. At the time of the survey, 60.3% of patients were receiving active cancer treatment (56.9% chemotherapy, 25.2% targeted therapy, and 6.5% immunotherapy). Despite 98.5% of survey participants having health insurance, a number of respondents reported financial difficulty in pursuing medical care.
Although 77.5% of patients reported having received biomarker testing, only 27% had enrolled in a clinical trial. Respondents cited several reasons for clinical trial ineligibility:
Unfortunately, the clinical trial enrollment rate for patients with CCA remains low. Thus, in collaboration with all stakeholders, CCF is working with the FDA on guidance to minimize clinical trial enrollment eligibility criteria—a critical barrier leading to low enrollment rates. Moreover, CCF continues to work collaboratively with industry partners to open more clinical trials, allow expanded access programs, and design clinical trials for patients who do not have actionable mutations to provide treatment opportunities and hope for more patients with CCA.
Dr Abdel-Wahab is Chief Scientific Officer and Director of Research for the Cholangiocarcinoma Foundation (CCF), Salt Lake City, UT, and Associate Professor of Clinical Oncology at Assiut University Faculty of Medicine, Egypt. Ms Pear is a patient advocate volunteer at CCF and a master’s degree student at Mel and Enid Zuckerman College of Public Health, University of Arizona, Tucson, AZ.
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