Dynamic Treatment Regimes: Statistical Methods for Precision Medicine provides a comprehensive introduction to statistical methodology for the evaluation and discovery of dynamic treatment regimes from data. Researchers and graduate students in statistics, data science, and related quantitative disciplines with a background in probability and statistical inference and popular statistical modeling techniques will be prepared for further study of this rapidly evolving field. A dynamic treatment regime is a set of sequential decision rules, each corresponding to a key decision point in a disease or disorder process, where each rule takes as input patient information and returns the treatment option he or she should receive. Thus, a treatment regime formalizes how a clinician synthesizes patient information and selects treatments in practice. Treatment regimes are of obvious relevance to precision medicine, which involves tailoring treatment selection to patient characteristics in an evidence-based way. Of critical importance to precision medicine is estimation of an optimal treatment regime, one that, if used to select treatments for the patient population, would lead to the most beneficial outcome on average. Key methods for estimation of an optimal treatment regime from data are motivated and described in detail. A dedicated companion website presents full accounts of application of the methods using a comprehensive R package developed by the authors. The authors’ website www.dtr-book.com includes updates, corrections, new papers, and links to useful websites.

Statistical Methods for Dynamic Treatment Regimes shares state of the art of statistical methods developed to address questions of estimation and inference for dynamic treatment regimes, a branch of personalized medicine. This volume demonstrates these methods with their conceptual underpinnings and illustration through analysis of real and simulated data. These methods are immediately applicable to the practice of personalized medicine, which is a medical paradigm that emphasizes the systematic use of individual patient information to optimize patient health care. This is the first single source to provide an overview of methodology and results gathered from journals, proceedings, and technical reports with the goal of orienting researchers to the field. The first chapter establishes context for the statistical reader in the landscape of personalized medicine. Readers need only have familiarity with elementary calculus, linear algebra, and basic large-sample theory to use this text. Throughout the text, authors direct readers to available code or packages in different statistical languages to facilitate implementation. In cases where code does not already exist, the authors provide analytic approaches in sufficient detail that any researcher with knowledge of statistical programming could implement the methods from scratch. This will be an important volume for a wide range of researchers, including statisticians, epidemiologists, medical researchers, and machine learning researchers interested in medical applications. Advanced graduate students in statistics and biostatistics will also find material in Statistical Methods for Dynamic Treatment Regimes to be a critical part of their studies.

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.? The authors provide a learning-by-seeing approach to the development of ATSs, aimed at a broad audience of health researchers. All estimation procedures used are described in sufficient heuristic and technical detail so that less quantitative readers can understand the broad principles underlying the approaches. At the same time, more quantitative readers can implement these practices. This book provides the most up-to-date summary of the current state of the statistical research in personalized medicine; contains chapters by leaders in the area from both the statistics and computer sciences fields; and also contains a range of practical advice, introductory and expository materials, and case studies.?

Cancer Clinical Trials: Current and Controversial Issues in Design and Analysis provides statisticians with an understanding of the critical challenges currently encountered in oncology trials. Well-known statisticians from academic institutions, regulatory and government agencies (such as the U.S. FDA and National Cancer Institute), and the pharmaceutical industry share their extensive experiences in cancer clinical trials and present examples taken from actual trials. The book covers topics that are often perplexing and sometimes controversial in cancer clinical trials. Most of the issues addressed are also important for clinical trials in other settings. After discussing general topics, the book focuses on aspects of early and late phase clinical trials. It also explores personalized medicine, including biomarker-based clinical trials, adaptive clinical trial designs, and dynamic treatment regimes.

This volume covers classic as well as cutting-edge topics on the analysis of clinical trial data in biomedical and psychosocial research and discusses each topic in an expository and user-friendly fashion. The intent of the book is to provide an overview of the primary statistical and data analytic issues associated with each of the selected topics, followed by a discussion of approaches for tackling such issues and available software packages for carrying out analyses. While classic topics such as survival data analysis, analysis of diagnostic test data and assessment of measurement reliability are well known and covered in depth by available topic-specific texts, this volume serves a different purpose: it provides a quick introduction to each topic for self-learning, particularly for those who have not done any formal coursework on a given topic but must learn it due to its relevance to their multidisciplinary research. In addition, the chapters on these classic topics will reflect issues particularly relevant to modern clinical trials such as longitudinal designs and new methods for analyzing data from such study designs. The coverage of these topics provides a quick introduction to these important statistical issues and methods for addressing them. As with the classic topics, this part of the volume on modern topics will enable researchers to grasp the statistical methods for addressing these emerging issues underlying modern clinical trials and to apply them to their research studies.

There is an increasing need for educational resources for statisticians and investigators. Reflecting this, the goal of this book is to provide readers with a sound foundation in the statistical design, conduct, and analysis of clinical trials. Furthermore, it is intended as a guide for statisticians and investigators with minimal clinical trial experience who are interested in pursuing a career in this area. The advancement in genetic and molecular technologies have revolutionized drug development. In recent years, clinical trials have become increasingly sophisticated as they incorporate genomic studies, and efficient designs (such as basket and umbrella trials) have permeated the field. This book offers the requisite background and expert guidance for the innovative statistical design and analysis of clinical trials in oncology. Key Features: Cutting-edge topics with appropriate technical background Built around case studies which give the work a "hands-on" approach Real examples of flaws in previously reported clinical trials and how to avoid them Access to statistical code on the book’s website Chapters written by internationally recognized statisticians from academia and pharmaceutical companies Carefully edited to ensure consistency in style, level, and approach Topics covered include innovating phase I and II designs, trials in immune-oncology and rare diseases, among many others

Discover best practices for real world data research with SAS code and examples Real world health care data is common and growing in use with sources such as observational studies, patient registries, electronic medical record databases, insurance healthcare claims databases, as well as data from pragmatic trials. This data serves as the basis for the growing use of real world evidence in medical decision-making. However, the data itself is not evidence. Analytical methods must be used to turn real world data into valid and meaningful evidence. Real World Health Care Data Analysis: Causal Methods and Implementation Using SAS brings together best practices for causal comparative effectiveness analyses based on real world data in a single location and provides SAS code and examples to make the analyses relatively easy and efficient. The book focuses on analytic methods adjusted for time-independent confounding, which are useful when comparing the effect of different potential interventions on some outcome of interest when there is no randomization. These methods include: propensity score matching, stratification methods, weighting methods, regression methods, and approaches that combine and average across these methods methods for comparing two interventions as well as comparisons between three or more interventions algorithms for personalized medicine sensitivity analyses for unmeasured confounding

This book examines recent developments in Operations Management, and focuses on four major application areas: dynamic pricing, assortment optimization, supply chain and inventory management, and healthcare operations. Data-driven optimization in which real-time input of data is being used to simultaneously learn the (true) underlying model of a system and optimize its performance, is becoming increasingly important in the last few years, especially with the rise of Big Data.

This issue of Clinics in Plastic Surgery, guest edited by Drs. Charles Scott Hultman and Michael W. Neumeister, is devoted to Burn Care: Reconstruction, Rehabilitation, and Recovery. Articles in this issue include: Role of Physiatry in the Management of Chronic Pain; New Concepts in OT, PT, RT: Focus on Functional Cutaneous Units; Rehabilitation in the Acute vs Outpatient Setting; Role of Anesthesia in the Management of Chronic Pain; Pathophysiology and Treatment of Hypertophic Burn Scars; Laser Modulation of Hypertrophic Scars; Fat Grafting for Burn, Traumatic, and Surgical Scars; Peripheral Neuropathy and Nerve Compression Syndromes; New Frontiers in Skin Grafting: The Painless, Scarless Donor Site; Tissue Rearrangements: The Power of the Z-pasty; Prefabricated Flaps for Burn Reconstruction; Microsurgery: Timing and Indications in Burn Patients; Hand Reconstruction: Functional Results; Head and Neck Reconstruction; Vascularized Composite Allotransplantation: Hand and Face; Ethics in the Setting of the Burned Patient; Psychosocial-spiritual Needs of Burn Patients; Aftercare, Survivorship, and Peer Support; Global Health Initiatives, Public Policy, and International Missions;Outcomes: Scar Scales, Physiologic Metrics, Measuring QOL; and Big Data: EBM and Best Practices.