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Title Interface between regulation and statistics in drug development / Demissie Alemayehu, Birol Emir, Michael Gaffney.
Imprint Boca Raton : Chapman & Hall/CRC, 2020.


 Internet  Electronic Book    AVAILABLE
Edition 1st.
Description 1 online resource : illustrations (black and white).
Series Chapman and Hall/CRC Biostatistics Ser.
Chapman and Hall/CRC Biostatistics Ser.
Note Available only to authorized UTEP users.
Bio/Hist Note Demissie Alemayehu, PhD, is Vice President and Head of the Statistical Research and Data Science Center at Pfizer Inc. He is a Fellow of the American Statistical Association, has published widely, and has served on the editorial boards of major journals, including the "Journal of the American Statistical Association" and the "Journal of Nonparametric Statistics." Additionally, he has been on the faculties of both Columbia University and Western Michigan University. He has co-authored a monograph entitled, "Patient-Reported Outcomes: Measurement, Implementation and Interpretation," and co-edited another, "Statistical Topics in Health Economics and Outcome Research" both published by Chapman & Hall/CRC Press. Birol Emir, PhD, is Senior Director and Statistics Lead of Real-World Evidence (RWE) at Pfizer Inc. In addition, Dr. Emir has served as Adjunct Professor of Statistics and Lecturer at Columbia University in New York and as an External PhD Committee Member, Graduate School of Arts and Sciences, Rutgers, The State University of New Jersey. Recently, his primary focuses have been on big data, predictive modelling and genomic data analysis. He has numerous publications in refereed journals, and he has co-edited "Statistical Topics in Health Economics and Outcome Research" published by Chapman & Hall/CRC Press. He has taught many short courses and has given several invited presentations. Michael Gaffney, PhD, is Vice President, Statistics at Pfizer, and received his Ph.D. from New York University School of Environmental Medicine with his dissertation in the area of multistage model of cancer induction. Dr. Gaffney has spent his 43-year career in pharmaceutical research concentrating in the areas of design and analysis of clinical trials and regulatory interaction for drug approval and product defense. He has interacted with FDA, EMA, MHRA and regulators in Canada and Japan on over 25 distinct regulatory approvals and product issues in many therapeutic areas. Dr. Gaffney has published 40 peer-reviewed articles and presented at numerous scientific meetings in diverse areas of modelling cancer induction, variance components, harmonic regression, factor analysis, propensity scores, meta-analysis, large safety trials and sample size re-estimation. Dr. Gaffney was recently a member of the Council for International Organizations of Medical Sciences (CIOMS) X committee and was a co-author of, CIOMS X: Evidence Synthesis and Meta-Analysis for Drug Safety.
Note Description based on CIP data; resource not viewed.
Subject United States. Food and Drug Administration -- Rules and practice.
Drugs -- United States -- Testing -- Statistical methods.
Drugs -- Testing -- Law and legislation -- United States.
Clinical trials -- United States -- Statistical methods.
Clinical trials -- Law and legislation -- United States.
Clinical trials -- Reporting -- United States.
Drug development -- United States.
Pharmaceutical policy -- United States.
Contents Cover -- Half Title -- Series Information -- Title Page -- Copyright Page -- Table of contents -- Figures -- Abbreviations -- Authors' Disclosure -- Acknowledgment -- Preface -- About the Authors -- Chapter 1 Fundamental Principles of Clinical Trials -- 1.1 Introduction -- 1.2 General Statistical Considerations -- 1.2.1 Statistical Analysis Plan -- 1.2.2 Trial Design -- 1.2.3 Randomization and Blinding -- 1.2.4 Statistical Methodology -- 1.2.5 Reporting and Interpretation of Study Results -- 1.2.6 Data Quality and Software Validity -- 1.3 Evolving Roles of the Statistician in Drug Development.
1.4 Potential Statistical Issues in Regulatory Review -- 1.4.1 Data Quality -- 1.4.2 Endpoint Definition -- 1.4.3 Design and Analysis Issues -- 1.4.4 Evaluation of Safety -- 1.4.5 Analysis Populations and Subgroups -- 1.4.6 Assessing Interpretation and Reliability of Results -- 1.5 Concluding Remarks -- Bibliography -- Chapter 2 Selected Statistical Topics of Regulatory Importance -- 2.1 Introduction -- 2.2 Multiplicity -- 2.2.1 Multiple Endpoints -- 2.2.2 Multiple Testing Over the Course of the Study -- 2.3 Missing Values and Estimands -- 2.3.1 General Considerations.
2.3.2 Missingness Mechanisms -- 2.3.3 Approaches for Missing Data -- 2.3.4 Sensitivity Analyses -- 2.3.5 Estimands and Other Recent Regulatory Developments -- 2.3.6 Concluding Remarks -- 2.4 Non-inferiority Study -- 2.4.1 Efficacy Objective -- 2.4.2 Non-inferiority Hypothesis / Non-inferiority Margin -- 2.4.3 Determination of NIM -- 2.4.4 Example: FDA Guidance Document -- 2.4.5 Implications of Choice of NIM -- 2.4.6 Strength of a Non-inferiority Study -- 2.4.7 Synthesis Method for Non-inferiority -- 2.4.8 Summary Points -- 2.4.9 Non-inferiority Study with a Safety Objective.
2.4.10 Summary Points -- 2.5 Innovative Trial Designs -- 2.5.1 Adaptive Designs -- 2.5.2 Adaptive Randomization -- 2.5.3 Sample Size Reestimation -- 2.5.4 Sequential Designs -- 2.5.5 Adaptive Designs for Dose and Treatment Selection -- 2.5.6 Adaptive Enrichment Designs -- 2.5.7 Master Protocols -- Basket Trials -- Umbrella Trials -- Platform Trials -- Regulatory and Operational Considerations with Novel Trials -- 2.6 Bayesian Analysis in a Regulatory Framework -- 2.6.1 Introduction -- 2.6.2 Potential Areas of Application -- 2.6.3 Regulatory Considerations.
2.6.4 Challenges with Bayesian Statistics -- 2.6.5 Concluding Remarks -- 2.7 Surrogate Endpoints and Biomarkers -- 2.7.1 Introduction -- 2.7.2 Statistical Considerations -- 2.7.3 Regulatory Considerations -- 2.7.4 Concluding Remarks -- 2.8 Subgroup Analyses -- 2.8.1 Introduction -- 2.8.2 Subgroup Analyses in the Traditional Confirmatory Clinical-Trial Setting -- 2.8.3 Statistical Approaches -- 2.8.4 Reporting and Interpretation of Subgroup Results -- 2.8.5 Subgroup Analyses in the Changing Clinical-Trial and Regulatory Setting -- 2.8.6 Conclusion -- 2.9 Benefit-Risk Assessment.
Summary With the critical role of statistics in the design, conduct, analysis and reporting of clinical trials or observational studies intended for regulatory purposes, numerous guidelines have been issued by regulatory authorities around the world focusing on statistical issues related to drug development. However, the available literature on this important topic is sporadic, and often not readily accessible to drug developers or regulatory personnel. This book provides a systematic exposition of the interplay between the two disciplines, including emerging themes pertaining to the acceleration of the development of pharmaceutical medicines to serve patients with unmet needs. Features: Regulatory and statistical interactions throughout the drug development continuum The critical role of the statistician in relation to the changing regulatory and healthcare landscapes Statistical issues that commonly arise in the course of drug development and regulatory interactions Trending topics in drug development, with emphasis on current regulatory thinking and the associated challenges and opportunities The book is designed to be accessible to readers with an intermediate knowledge of statistics, and can be a useful resource to statisticians, medical researchers, and regulatory personnel in drug development, as well as graduate students in the health sciences. The authors' decades of experience in the pharmaceutical industry and academia, and extensive regulatory experience, comes through in the many examples throughout the book.
Other Author Emir, Birol, author.
Gaffney, Michael, active 1983, author.
Other Title Print version: 9780367490485.