Biomarkers in Drug Development: Statistical Methods and Applications

Addresses the central role biomarkers play in modern drug development. Across therapeutic areas and modalities, biomarkers inform disease classification, patient selection, trial design, and regulatory decision‑making. Biomarker Statistical Considerations and Approaches for Clinical Trials addresses the central role biomarkers play in modern drug development. Across therapeutic areas and modalities, biomarkers inform disease classification, patient selection, trial design, and regulatory decision‑making. As precision medicine becomes standard practice, the ability to rigorously evaluate and interpret biomarker evidence has become essential. Yet effective biomarker use requires more than biological insight alone—it demands sound statistical methodology, principled study design, and careful consideration of emerging statistical approaches. This book provides a comprehensive, end‑to‑end treatment of biomarker methodology from a quantitative perspective. Bringing together contributions from leading experts in biostatistics, machine learning, and clinical development, it offers an integrated view of biomarkers across the full lifecycle of drug development—from discovery and validation to modeling, confirmation, and interpretation. Rather than presenting isolated techniques, the volume emphasizes unifying principles that connect classical biostatistics with modern causal inference, high‑dimensional data analysis, and AI‑driven approaches, reflecting real‑world biomarker development and deployment. Key Features: Covers core biomarker types, including prognostic, predictive, and surrogate biomarkers, with clear distinctions between roles and evidentiary requirements Addresses advanced topics such as treatment effect heterogeneity, companion diagnostics, generalizability and transportability, and biomarker‑driven trial designs Includes modern methodologies for high‑dimensional omics data, machine learning, artificial intelligence, and digital twins Tackles practical challenges such as multiplicity, missing and censored data, and statistical validation across development stages Integrates regulatory and applied perspectives throughout, linking methodology to clinical and development decision‑making Designed for statisticians, data scientists, and quantitative researchers in industry, academia, and regulatory agencies—as well as clinicians seeking a deeper understanding of the statistical foundations of biomarker‑based evidence—this book serves as both a practical reference and a conceptual guide. It provides a durable foundation for navigating today’s biomarker‑rich landscape and for engaging thoughtfully with innovations shaping the future of precision medicine.