Adaptive Treatment Strategies in Practice – Planning Trials and Analyzing Data for Personalized Medicine

Personalized medicine is a medical paradigm that emphasizes systematic use of individual patient information to optimize that patient's health care, particularly in managing chronic conditions and treating cancer. In the statistical literature, sequential decision making is known as an adaptive treatment strategy (ATS) or a dynamic treatment regime (DTR). The field of DTRs emerges at the interface of statistics, machine learning and biomedical science to provide a data-driven framework for precision medicine. A learning-by-seeing approach to the development of ATSs is provided in this book. While estimation procedures are described in sufficient heuristic and technical detail, so that less quantitative readers can understand the broad principles underlying the approaches, practices can also be implemented by more quantitative readers. As the most up-to-date summary of the current state of statistical research in personalized medicine, this book is ideal for a broad audience of health researchers.

Michael R. Kosorok is W. R. Kenan, Jr Distinguished Professor and Chair of Biostatistics and Professor of Statistics and Operations Research at the University of North Carolina, Chapel Hill. He is an honorary fellow of both the American Statistical Association and the Institute of Mathematical Statistics and an Associate Editor of The Annals of Statistics, the Journal of the American Statistical Association, and the Journal of the Royal Statistical Society, Series B. He is the contact principal investigator for a program project (P01) from the US National Cancer Institute, entitled 'Statistical Methods for Cancer Clinical Trials'. His main research interests are in precision medicine, clinical trials, machine learning, and related areas.

Erica E. M. Moodie is a William Dawson Scholar and an Associate Professor of Biostatistics in the Department of Epidemiology, Biostatistics and Occupational Health at McGill University. She is an Elected Member of the International Statistical Institute, and an Associate Editor of Biometrics and the Journal of the American Statistical Association. She holds a Chercheur–Boursier Junior 2 career award from the Fonds de Recherche du Québec-Santé. Her main research interests are in causal inference and longitudinal data, with a focus on dynamic treatment regimes.