Advancing Precision Medicine: Statistical Approaches and Design Strategies to Integrate Biomarkers for Targeted Therapies

This full-day short course will focus on precision medicine, AI/ML approaches, design and companion diagnostics. The first part will provide the audience with a concise understanding of precision medicine and the statistical considerations to find a predictive biomarker for patient stratification and enrichment. Methods around accelerating from early phase to late phase, particularly around implementing historical data, Bayesian borrowing approaches, and AI/ML approaches will be leveraged to take advantage of available data and discuss solutions to address the challenges of limited data. The second part of this course focuses on clinical trial design with an emphasis on biomarker-driven approaches. The third part of the short course aims to equip clinical statisticians with a comprehensive understanding of the statistical principles and methodologies that underpin successful companion diagnostics (CDx) development. Participants will gain insights into fundamental design elements, key analytical strategies, and emerging advanced methods, thereby strengthening their capacity to navigate the evolving regulatory landscape and successfully contribute to CDx study teams. Key issues, evolution of the statistical field, and new statistical and design approaches around precision medicine and biomarkers will be covered so the participants will be more knowledgeable and successful in their study team contributions.