Targeted therapy for biliary tract cancer: facts and fictions as well as demands and challenges.

[INTRODUCTION] Targeted therapy is an established treatment strategy for biliary tract cancer (BTC), yet its clinical efficacy remains limited when compared to promising and findings. This highlights the translational gap between preclinical research and real-world patient outcomes, emphasizing the urgent need for improved strategies to bridge laboratory insights with clinical practice.

[AREAS COVERED] This report explores the heterogeneous landscape of targeted therapy in BTC. It reviews established facts, including molecular mechanisms, signaling pathways, and clinical trial data, while addressing uncertainties such as patient- and tumor-related variables and infectious comorbidities. In addition, it discusses current demands and future challenges, with emphasis on integrating novel technologies, biomarker-driven approaches, and multidisciplinary collaborations to enhance therapeutic precision.

[EXPERT OPINION] Early and accurate diagnosis remains the cornerstone of successful BTC treatment. Noninvasive biomarkers, particularly liquid biopsy platforms, hold promise for improving detection and monitoring. Clinically, the establishment of high-volume BTC centers with integrated scientific and medical expertise is essential to translate molecular analyses into personalized therapies. Moreover, systematic incorporation of BTC-related clinical and molecular data into international multicenter trials is imperative. Such integration will refine therapeutic paradigms, accelerate drug development, and foster innovation through strategic collaborations with pharmaceutical industry consortia.