"This is truly an outstanding book. [It] brings together all of the latest research in clinical trials methodology and how it can be applied to drug development.... Chang et al provide applications to industry-supported trials. This will allow statisticians in the industry community to take these methods seriously." Jay Herson, Johns Hopkins University The pharmaceutical industry's approach to drug discovery and development has rapidly transformed in the last decade from the more traditional Research and Development (R & D) approach to a more innovative approach in which strategies are employed to compress and optimize the clinical development plan and associated timelines. However, these strategies are generally being considered on an individual trial basis and not as part of a fully integrated overall development program. Such optimization at the trial level is somewhat near-sighted and does not ensure cost, time, or development efficiency of the overall program. This book seeks to address this imbalance by establishing a statistical framework for overall/global clinical development optimization and providing tactics and techniques to support such optimization, including clinical trial simulations. Provides a statistical framework for achieve global optimization in each phase of the drug development process. Describes specific techniques to support optimization including adaptive designs, precision medicine, survival-endpoints, dose finding and multiple testing. Gives practical approaches to handling missing data in clinical trials using SAS. Looks at key controversial issues from both a clinical and statistical perspective. Presents a generous number of case studies from multiple therapeutic areas that help motivate and illustrate the statistical methods introduced in the book. Puts great emphasis on software implementation of the statistical methods with multiple examples of software code (both SAS and R). It is important for statisticians to possess a deep knowledge of the drug development process beyond statistical considerations. For these reasons, this book incorporates both statistical and "clinical/medical" perspectives.

Mathematical and Statistical Skills in the Biopharmaceutical Industry: A Pragmatic Approach describes a philosophy of efficient problem solving showcased using examples pertinent to the biostatistics function in clinical drug development. It was written to share a quintessence of the authors’ experiences acquired during many years of relevant work in the biopharmaceutical industry. The book will be useful will be useful for biopharmaceutical industry statisticians at different seniority levels and for graduate students who consider a biostatistics-related career in this industry. Features: Describes a system of principles for pragmatic problem solving in clinical drug development. Discusses differences in the work of a biostatistician in small pharma and big pharma. Explains the importance/relevance of statistical programming and data management for biostatistics and necessity for integration on various levels. Describes some useful statistical background that can be capitalized upon in the drug development enterprise. Explains some hot topics and current trends in biostatistics in simple, non-technical terms. Discusses incompleteness of any system of standard operating procedures, rules and regulations. Provides a classification of scoring systems and proposes a novel approach for evaluation of the safety outcome for a completed randomized clinical trial. Presents applications of the problem solving philosophy in a highly problematic transfusion field where many investigational compounds have failed. Discusses realistic planning of open-ended projects.

Artificial Intelligence for Drug Development, Precision Medicine, and Healthcare covers exciting developments at the intersection of computer science and statistics. While much of machine-learning is statistics-based, achievements in deep learning for image and language processing rely on computer science’s use of big data. Aimed at those with a statistical background who want to use their strengths in pursuing AI research, the book: · Covers broad AI topics in drug development, precision medicine, and healthcare. · Elaborates on supervised, unsupervised, reinforcement, and evolutionary learning methods. · Introduces the similarity principle and related AI methods for both big and small data problems. · Offers a balance of statistical and algorithm-based approaches to AI. · Provides examples and real-world applications with hands-on R code. · Suggests the path forward for AI in medicine and artificial general intelligence. As well as covering the history of AI and the innovative ideas, methodologies and software implementation of the field, the book offers a comprehensive review of AI applications in medical sciences. In addition, readers will benefit from hands on exercises, with included R code.

Humanized AI (HAI), emerging as the next of the AI waves, refers to artificial social beings that are very close to humans in various aspects, beings who are machine-race humans, not digital slaves. Foundation, Architecture, and Prototyping of HAI deploy a novel smalldata approach to vertically explore the spectrum of HAI. Different from the popular big-data philosophy that is based on the rigid notion that the connotation of each concept is fixed and the same to everyone, this book treats understanding as a process from simple to complex, and uses the similarity principle to effectively deal with novelties. Combining the efficiency of the Behaviorists’ goal-driven approach and the flexibility of a Constructivists’ approach, both the architecture of HAI and the philosophical discussions arising from it are elaborated upon. Advancing a unique approach to the concept of HAI, this book appeals to professors and students of both AI and philosophy, as well as industry professionals looking to stay at the forefront of developments within the field.

Mixture Modelling for Medical and Health Sciences provides a direct connection between theoretical developments in mixture modelling and their applications in real world problems. The book describes the development of the most important concepts through comprehensive analyses of real and practical examples taken from real-life research problems in

Cancer is a major healthcare burden across the world and impacts not only the people diagnosed with various cancers but also their families, carers, and healthcare systems. With advances in the diagnosis and treatment, more people are diagnosed early and receive treatments for a disease where few treatments options were previously available. As a result, the survival of patients with cancer has steadily improved and, in most cases, patients who are not cured may receive multiple lines of treatment, often with financial consequences for the patients, insurers and healthcare systems. Although many books exist that address economic evaluation, Economic Evaluation of Cancer Drugs using Clinical Trial and Real World Data is the first unified text that specifically addresses the economic evaluation of cancer drugs. The authors discuss how to perform cost-effectiveness analyses while emphasising the strategic importance of designing cost-effectiveness into cancer trials and building robust economic evaluation models that have a higher chance of reimbursement if truly cost-effective. They cover the use of real-world data using cancer registries and discuss how such data can support or complement clinical trials with limited follow up. Lessons learned from failed reimbursement attempts, factors predictive of successful reimbursement and the different payer requirements across major countries including US, Australia, Canada, UK, Germany, France and Italy are also discussed. The book includes many detailed practical examples, case studies and thought-provoking exercises for use in classroom and seminar discussions. Iftekhar Khan is a medical statistician and health economist and a lead statistician at Oxford Unviersity’s Center for Statistics in Medicine. Professor Khan is also a Senior Research Fellow in Health Economics at University of Warwick and is a Senior Statistical Assessor within the Licensing Division of the UK Medicine and Health Regulation Agency. Ralph Crott is a former professor in Pharmacoeconomics at the University of Montreal in Quebec, Canada and former head of the EORTC Health Economics Unit and former senior health economist at the Belgian HTA organization. Zahid Bashir has over twelve years experience working in the pharmaceutical industry in medical affairs and oncology drug development where he is involved in the design and execution of oncology clinical trials and development of reimbursement dossiers for HTA submission.

Drug development is an iterative process. The recent publications of regulatory guidelines further entail a lifecycle approach. Blending data from disparate sources, the Bayesian approach provides a flexible framework for drug development. Despite its advantages, the uptake of Bayesian methodologies is lagging behind in the field of pharmaceutical development. Written specifically for pharmaceutical practitioners, Bayesian Analysis with R for Drug Development: Concepts, Algorithms, and Case Studies, describes a wide range of Bayesian applications to problems throughout pre-clinical, clinical, and Chemistry, Manufacturing, and Control (CMC) development. Authored by two seasoned statisticians in the pharmaceutical industry, the book provides detailed Bayesian solutions to a broad array of pharmaceutical problems. Features Provides a single source of information on Bayesian statistics for drug development Covers a wide spectrum of pre-clinical, clinical, and CMC topics Demonstrates proper Bayesian applications using real-life examples Includes easy-to-follow R code with Bayesian Markov Chain Monte Carlo performed in both JAGS and Stan Bayesian software platforms Offers sufficient background for each problem and detailed description of solutions suitable for practitioners with limited Bayesian knowledge Harry Yang, Ph.D., is Senior Director and Head of Statistical Sciences at AstraZeneca. He has 24 years of experience across all aspects of drug research and development and extensive global regulatory experiences. He has published 6 statistical books, 15 book chapters, and over 90 peer-reviewed papers on diverse scientific and statistical subjects, including 15 joint statistical works with Dr. Novick. He is a frequent invited speaker at national and international conferences. He also developed statistical courses and conducted training at the FDA and USP as well as Peking University. Steven Novick, Ph.D., is Director of Statistical Sciences at AstraZeneca. He has extensively contributed statistical methods to the biopharmaceutical literature. Novick is a skilled Bayesian computer programmer and is frequently invited to speak at conferences, having developed and taught courses in several areas, including drug-combination analysis and Bayesian methods in clinical areas. Novick served on IPAC-RS and has chaired several national statistical conferences.