Mathematical And Statistical Skills In The Biopharmaceutical Industry

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.

This book examines statistical techniques that are critically important to Chemistry, Manufacturing, and Control (CMC) activities. Statistical methods are presented with a focus on applications unique to the CMC in the pharmaceutical industry. The target audience consists of statisticians and other scientists who are responsible for performing statistical analyses within a CMC environment. Basic statistical concepts are addressed in Chapter 2 followed by applications to specific topics related to development and manufacturing. The mathematical level assumes an elementary understanding of statistical methods. The ability to use Excel or statistical packages such as Minitab, JMP, SAS, or R will provide more value to the reader. The motivation for this book came from an American Association of Pharmaceutical Scientists (AAPS) short course on statistical methods applied to CMC applications presented by four of the authors. One of the course participants asked us for a good reference book, and the only book recommended was written over 20 years ago by Chow and Liu (1995). We agreed that a more recent book would serve a need in our industry. Since we began this project, an edited book has been published on the same topic by Zhang (2016). The chapters in Zhang discuss statistical methods for CMC as well as drug discovery and nonclinical development. We believe our book complements Zhang by providing more detailed statistical analyses and examples.

One of the hallmarks of the 21st century medicine is the emergence of digital therapeutics (DTx)—evidence-based, clinically validated digital technologies to prevent, diagnose, treat, and manage various diseases and medical conditions. DTx solutions have been gaining interest from patients, investors, healthcare providers, health authorities, and other stakeholders because of the potential of DTx to deliver equitable, massively scalable, personalized and transformative treatments for different unmet medical needs. Digital Therapeutics: Scientific, Statistical, Clinical, and Regulatory Aspects is an unparalleled summary of the current scientific, statistical, developmental, and regulatory aspects of DTx which is poised to become the fastest growing area of the biopharmaceutical and digital medicine product development. This edited volume intends to provide a systematic exposition to digital therapeutics through 19 peer-reviewed chapters written by subject matter experts in this emerging field. This edited volume is an invaluable resource for business leaders and researchers working in public health, healthcare, digital health, information technology, and biopharmaceutical industries. It will be also useful for regulatory scientists involved in the review of DTx products, and for faculty and students involved in an interdisciplinary research on digital health and digital medicine. Key Features: Provides the taxonomy of the concepts and a navigation tool for the field of DTx. Covers important strategic aspects of the DTx industry, thereby helping investors, developers, and regulators gain a better appreciation of the potential value of DTx. Expounds on many existing and emerging state-of-the art scientific and technological tools, as well as data privacy, ethical and regulatory considerations for DTx product development. Presents several case studies of successful development of some of the most remarkable DTx products. Provides some perspectives and forward-looking statements on the future of digital medicine.

Providing a general guide to statistical methods used in the pharmaceutical industry, and illustrating how to use S-PLUS to implement these methods, the book explains why S-PLUS is a useful software package and discusses the results and implications of each particular application. It is targeted at graduates in biostatistics, statisticians involved in the industry as research scientists, regulators, academics, and/or consultants who want to know more about how to use S-PLUS and learn about other sub-fields within the industry, as well as statisticians in other fields who want to know more about statistical applications in the pharmaceutical industry.

Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx Insights into commercial models, access hurdles, and health economics of gene therapies Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective

Geospatial health data are essential to inform public health and policy. These data can be used to quantify disease burden, understand geographic and temporal patterns, identify risk factors, and measure inequalities. Geospatial Health Data: Modeling and Visualization with R-INLA and Shiny describes spatial and spatio-temporal statistical methods and visualization techniques to analyze georeferenced health data in R. The book covers the following topics: Manipulating and transforming point, areal, and raster data, Bayesian hierarchical models for disease mapping using areal and geostatistical data, Fitting and interpreting spatial and spatio-temporal models with the integrated nested Laplace approximation (INLA) and the stochastic partial differential equation (SPDE) approaches, Creating interactive and static visualizations such as disease maps and time plots, Reproducible R Markdown reports, interactive dashboards, and Shiny web applications that facilitate the communication of insights to collaborators and policymakers. The book features fully reproducible examples of several disease and environmental applications using real-world data such as malaria in The Gambia, cancer in Scotland and USA, and air pollution in Spain. Examples in the book focus on health applications, but the approaches covered are also applicable to other fields that use georeferenced data including epidemiology, ecology, demography or criminology. The book provides clear descriptions of the R code for data importing, manipulation, modelling, and visualization, as well as the interpretation of the results. This ensures contents are fully reproducible and accessible for students, researchers and practitioners.

Medical Risk Prediction Models: With Ties to Machine Learning is a hands-on book for clinicians, epidemiologists, and professional statisticians who need to make or evaluate a statistical prediction model based on data. The subject of the book is the patient’s individualized probability of a medical event within a given time horizon. Gerds and Kattan describe the mathematical details of making and evaluating a statistical prediction model in a highly pedagogical manner while avoiding mathematical notation. Read this book when you are in doubt about whether a Cox regression model predicts better than a random survival forest. Features: All you need to know to correctly make an online risk calculator from scratch Discrimination, calibration, and predictive performance with censored data and competing risks R-code and illustrative examples Interpretation of prediction performance via benchmarks Comparison and combination of rival modeling strategies via cross-validation Thomas A. Gerds is a professor at the Biostatistics Unit at the University of Copenhagen and is affiliated with the Danish Heart Foundation. He is the author of several R-packages on CRAN and has taught statistics courses to non-statisticians for many years. Michael W. Kattan is a highly cited author and Chair of the Department of Quantitative Health Sciences at Cleveland Clinic. He is a Fellow of the American Statistical Association and has received two awards from the Society for Medical Decision Making: the Eugene L. Saenger Award for Distinguished Service, and the John M. Eisenberg Award for Practical Application of Medical Decision-Making Research.

Survival data analysis is a very broad field of statistics, encompassing a large variety of methods used in a wide range of applications, and in particular in medical research. During the last twenty years, several extensions of "classical" survival models have been developed to address particular situations often encountered in practice. This book aims to gather in a single reference the most commonly used extensions, such as frailty models (in case of unobserved heterogeneity or clustered data), cure models (when a fraction of the population will not experience the event of interest), competing risk models (in case of different types of event), and joint survival models for a time-to-event endpoint and a longitudinal outcome. Features Presents state-of-the art approaches for different advanced survival models including frailty models, cure models, competing risk models and joint models for a longitudinal and a survival outcome Uses consistent notation throughout the book for the different techniques presented Explains in which situation each of these models should be used, and how they are linked to specific research questions Focuses on the understanding of the models, their implementation, and their interpretation, with an appropriate level of methodological development for masters students and applied statisticians Provides references to existing R packages and SAS procedure or macros, and illustrates the use of the main ones on real datasets This book is primarily aimed at applied statisticians and graduate students of statistics and biostatistics. It can also serve as an introductory reference for methodological researchers interested in the main extensions of classical survival analysis.