Interface between regulation and statistics in drug development /

Interface between regulation and statistics in drug development /

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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...

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Bibliographic Details
Main Authors: Alemayehu, Demissie (Author), Emir, Birol (Author), Gaffney, Michael, active 1983 (Author)
Format: Electronic eBook
Language:English
Published: Boca Raton : Chapman & Hall/CRC, 2021.
Edition:1st.
Series:Chapman & Hall/CRC biostatistics series
Subjects:
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.
MEDICAL / Biostatistics
MATHEMATICS / Probability & Statistics / General
MEDICAL / Pharmacology
Online Access:Taylor & Francis
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Table of 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
  • 2.5.7.1 Basket Trials
  • 2.5.7.2 Umbrella Trials
  • 2.5.7.3 Platform Trials
  • 2.5.7.4 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

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