At the 2025 Annual Cholangiocarcinoma Foundation Conference, a working group that included co-chairs Juan Valle and Wendy Selig presented on the development of a consensus with focus on the key discussion points from the conference. Industry and regulatory processes including endpoints in cholangiocarcinoma (CCA) clinical trials were also discussed.
CCA poses significant challenges due to its rarity, molecular heterogeneity, and limited treatment options. To address these issues, consensus-driven frameworks have been adopted to align research priorities and optimize resources. The recent Cholangiocarcinoma Consensus Conference brought together experts, patient advocates, and international networks such as the International Cholangiocarcinoma Research Network and the European BTC Precision Network. Ten working groups focused on key areas, including diagnostics, biomarkers, systemic therapy, surgery, and patient advocacy, identifying critical gaps in CCA research. Key priorities include advancing early detection methods, improving molecular characterization, and developing personalized treatments through precision medicine.
A significant outcome of the conference was the recognition of the need for enhanced collaboration and funding to overcome the fragmented nature of CCA research. Historically, research efforts have occurred in silos, limiting progress. The conference emphasized the importance of aligning funding priorities across government agencies, private sectors, and international organizations. Collaborative networks like the COST Action Precision BTC Network, funded by the European Union, exemplify how interdisciplinary cooperation can accelerate progress in rare cancer research.
The recommendations from the conference will be compiled into a publication to serve as a strategic roadmap for future initiatives. By bridging funding gaps, aligning research priorities, and integrating patient advocacy perspectives, the scientific community aims to drive transformative changes in CCA research. The success of this collaborative framework highlights its potential to advance research in other rare cancers, offering hope for improved outcomes and therapeutic innovation.
The field of oncology is rapidly evolving, driven by innovations in therapeutic development and adaptive regulatory frameworks. In biliary tract cancer (BTC), a rare and aggressive malignancy, progress has been made by redefining clinical trial endpoints, integrating patient-centered tools, and adopting innovative treatment strategies. Traditional endpoints, such as overall survival (OS), remain the gold standard but are often time-intensive to collect and confounded by sequential therapies. Surrogate endpoints like progression-free survival (PFS), PFS from first to second treatment (PFS2), and time to next treatment have emerged as practical alternatives. Among these, PFS2 has shown strong correlation with OS, enabling faster trial completion and drug approval. Regulatory agencies such as the European Medicines Agency have begun recognizing PFS2 as a valid endpoint, setting a precedent for its broader use in oncology trials.
Patient-reported outcomes (PROs), particularly the PROs version of the Common Terminology Criteria for Adverse Events system, are also reshaping clinical trials by capturing patients’ perspectives on treatment toxicities and quality of life. These tools bypass clinician interpretations, providing unbiased and accurate assessments of adverse events and longitudinal symptom trajectories. However, challenges in standardizing data analysis and interpretation remain, necessitating consensus-driven guidelines for statistical modeling. Despite these hurdles, the growing adoption of PROs underscores the importance of patient-centered research in improving cancer care.
In BTC, downstaging to resection has also emerged as a promising trial endpoint. High response rates to targeted therapies and chemoimmunotherapy combinations have shown potential in converting unresectable tumors into resectable ones, offering patients the chance for long-term survival. While current data on downstaging are limited, structured clinical trials are essential to validate its feasibility and impact. This strategy represents a pivotal shift in treatment paradigms, with the potential to redefine therapeutic goals for BTC patients.
These developments reflect a broader trend in oncology toward interdisciplinary collaboration and patient-centered approaches, paving the way for meaningful progress in rare and challenging malignancies.
Selig W, Valle J. WG3-Industry and Regulatory. Presented at: 2025 Annual Cholangiocarcinoma Foundation Conference. April 9-11, 2025; Salt Lake City, UT.
Consensus Process. Presented at: 2025 Annual Cholangiocarcinoma Foundation Conference. April 9-11, 2025; Salt Lake City, UT.
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