SESSION I

This half‑day tutorial provides a focused yet comprehensive exploration of the science, strategy, and practical implementation of master protocol trials.  It is designed to give participants both a conceptual framework and a deep-dive into a real‑world case study on how these complex trial designs function in modern drug development.

The tutorial begins with an introduction to the fundamental concepts and framework of master protocols, followed by an overview of the evolving regulatory landscape, including recent guidance and increasing regulatory acceptance of such innovative trial designs. Participants will also examine trends in the adoption of master protocols across therapeutic areas and development settings, as well as the key challenges that continue to limit broader implementation, including operational complexity, stakeholder alignment, governance structures, statistical considerations, data sharing, and regulatory uncertainty.

Using I‑SPY2 as the central and unifying case study, the tutorial illustrates how platform trials have transformed clinical research by enabling more efficient, flexible, and patient‑centered development pathways.  In particular, the course highlights how Bayesian adaptive design principles, biomarker‑driven decision making, and the ability to evaluate multiple investigational agents within a single, continuously evolving protocol can substantially accelerate learning and improve clinical outcomes. Participants will gain insight into both the methodological innovations and the practical considerations that made I‑SPY2 a landmark example in the field.

By combining conceptual discussions with concrete examples and lessons learned, the tutorial equips participants with actionable knowledge to critically assess, design, or contribute to master protocol trials within their own organizations and research settings.

Instructors’ Biography:

Professor Donald A. Berry is a leading biostatistician and pioneer of Bayesian methods in medicine, widely recognized for his contributions to adaptive clinical trial design.   At MD Anderson, he served as the founding Chair of the Department of Biostatistics (1999–2010) and played a central role in advancing the use of Bayesian approaches for innovative oncology trials.   He founded Berry Consultants in 2000, a scientific consulting firm specializing in Bayesian and adaptive clinical trial design and implementation for pharmaceutical and medical device development.   Dr Berry has designed and led numerous innovative trials, including platform trials evaluating multiple therapies simultaneously, including the landmark I‑SPY 2 adaptive platform trial in breast cancer. He has published hundreds of scientific papers and multiple books in statistics and biostatistics.

Dr. Ruixiao Lu is a recognized leader in the biotech and biopharma industry, currently as Vice President and Head of Biostatistics and Statistical Programming at Alumis Inc. She has held senior leadership roles where she led end-to-end data science and analytical teams to support product development, regulatory submissions and approvals, medical affairs, and enterprise strategic initiatives. She is dedicated to advancing statistics and data science through professional organizations, as an elected Fellow and Board Director of of the American Statistical Association (ASA) (2020-2023, 2026–2028), elected President and Board Director of BBSW (2024–2026), and co-founder of DahShu (2015–present). She is widely engaged in cross-disciplinary collaborations, with a proven record of success in promoting scientific excellence and improving public health outcomes, including her leadership role at the 2023 FDA-AACR-ASA Overall Survival Workshop.

Dr. Chengxing (Cindy) Lu is a senior director of Oncology Biometrics in AstraZeneca, where she leads a team of statisticians dedicated to early-phase immunology and radioconjugate oncology development. Throughout her extensive career in drug discovery and development, her work has spanned the full spectrum of the field, encompassing early- and late-stage clinical development as well as post-marketing, medical affairs, reimbursement, and commercial strategies. Cindy is deeply committed to driving innovation and enhancing efficiency through multidisciplinary collaborations. She is actively engaged with the professional community through organizations such as the American Statistical Association (ASA), DahShu, DIA, and the International Society of Biopharmaceutical Statistics (ISBS), where she advocates for advanced statistical methodologies and promotes best practices that integrate multidisciplinary perspectives for real-world applications in drug development.