ABSTRACT
Since the publication of the first edition in 2000, there has been an explosive growth of literature in biopharmaceutical research and development of new medicines. This encyclopedia (1) provides a comprehensive and unified presentation of designs and analyses used at different stages of the drug development process, (2) gives a well-balanced summary of current regulatory requirements, and (3) describes recently developed statistical methods in the pharmaceutical sciences.
Features of the Fourth Edition:
1. 78 new and revised entries have been added for a total of 308 chapters and a fourth volume has been added to encompass the increased number of chapters.
2. Revised and updated entries reflect changes and recent developments in regulatory requirements for the drug review/approval process and statistical designs and methodologies.
3. Additional topics include multiple-stage adaptive trial design in clinical research, translational medicine, design and analysis of biosimilar drug development, big data analytics, and real world evidence for clinical research and development.
4. A table of contents organized by stages of biopharmaceutical development provides easy access to relevant topics.
About the Editor:
Shein-Chung Chow, Ph.D. is currently an Associate Director, Office of Biostatistics, U.S. Food and Drug Administration (FDA). Dr. Chow is an Adjunct Professor at Duke University School of Medicine, as well as Adjunct Professor at Duke-NUS, Singapore and North Carolina State University. Dr. Chow is the Editor-in-Chief of the Journal of Biopharmaceutical Statistics and the Chapman & Hall/CRC Biostatistics Book Series and the author of 28 books and over 300 methodology papers. He was elected Fellow of the American Statistical Association in 1995.
TABLE OF CONTENTS
entry
Acceptance Sampling
entry
Hypotheses and False Positive Rate in Active Control Non-Inferiority Trials
entry
Active Control Trials
entry
Active Controlled Clinical Trials: Noninferiority Analysis
entry
Adaptive Design Clinical Trials: Case Studies
entry
Adaptive Design Methods in Clinical Trials
entry
Adaptive Survival Trials
entry
Adverse Event Reporting
entry
Alpha Spending Function
entry
Ames Test
entry
Analysis of 2 × K Tables
entry
Analysis of Clustered Binary Data
entry
Analysis of Clustered Categorical Data
entry
Analysis of Heritability
entry
Analysis of Variance (ANOVA)
entry
Analytical Similarity Assessment
entry
ANCOVA Approach: Premarketing Shelf Life Determination with Multiple Factors
entry
Assay Development
entry
Assay Validation
entry
Assay Validation: Evaluation of Linearity
entry
Bayesian Designs: Phase II Oncology Clinical Trials
entry
Bayesian Estimate: Concordance Correlation Coefficient
entry
Bayesian Methods: Meta-Analysis
entry
Bayesian Methods: Stability Analysis
entry
Bayesian Model: Prior Effective Sample Size
entry
Bayesian Statistics
entry
Bayesian Statistics: Medical Device Clinical Trials
entry
Binary 2 × 2 Crossover Trials
entry
Bioassay
entry
Bioavailability and Bioequivalence
entry
Bioinformatics
entry
Biologics
entry
Biomarker Development with Applications to Genetics Data: Statistical Tests
entry
Biomarker: Clinical Trials
entry
Biopharmaceutics
entry
Biosimilar Studies: Sample Size
entry
Biosimilar Studies: Three-Arm Design
entry
Biosimilarity Assessment
entry
Biosimilarity of Follow-On Biologics
entry
Bivariate Zero-Inflated Poisson Populations: Statistical Test for Homogeneity
entry
Blinded Sample Size Re-Estimation
entry
Blinding
entry
Bootstrap, The
entry
Botanical Drug Product Development: Scientific Issues
entry
Bracketing Design
entry
Bridging Studies
entry
Bridging Studies: Statistical Methods
entry
Calibration
entry
Canadian Health Products and Food Branch (HPFB) and Therapeutic Products Directorate (TPD)
entry
Cancer Chemotherapy: Maximum Tolerable Dose
entry
Cancer Trials
entry
Carcinogenicity Studies of Pharmaceuticals
entry
Carry-Forward Analysis
entry
Case-Control Studies: Inference in
entry
Center Weighting: Multicenter Trials
entry
Clinical Data Management
entry
Clinical Endpoint
entry
Clinical Data Management
entry
Clinical Inspection: Statistical Process
entry
Clinical Pharmacology
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Clinical Research: Benefit and Risk Assessment
entry
Clinical Research: Comparing Variabilities
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Clinical Research: Outlier Detection
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Clinical Research: Reproducibility Probability
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Clinical Trial Design: Statistical Assurance
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Clinical Trial Designs with Prospective Patient Population Enrichment by Response
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Clinical Trial Process
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Clinical Trial Simulation
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Clinical Trial Simulations: Earlier Development Phases
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Clinical Trial Simulations: Later Development Phases
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Clinical Trial: Combination Drug Development
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Clinical Trial: Failure-Time Model
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Clinical Trial: N-of-1 Design Analysis
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Clinical Trials
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Clinical Trials: Controversial Issues
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Clinical Trials: Interval-censored Failure Time Data
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Clinical Trials: Investigating Quality-of-Life
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Clinical Trials: Meta-Analysis using R
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Clinical Trials: Response-Adaptive Repeated Measurement Designs
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Clinical Trials: Selection of Control
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Clinical Trials: Vaccine Development
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Cluster Randomized Trials: Sample Size
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Cluster Trials
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Clustered Study Designs: Power Analysis
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Companion Diagnostics
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Comparability Studies: Statistical Considerations
entry
Confidence Interval and Hypothesis Testing
entry
Confounding and Interaction
entry
Content Uniformity
entry
Continual Reassessment Methods
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Contract Research Organization (CRO): Early 2000s
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Contract Research Organization (CRO)
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Controlled Clinical Trials: P-Values, Evidence, and Multiplicity Considerations
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Correlated Probit Model
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Cost-Effectiveness Analysis
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Covariate-Adjusted Adaptive Dose-Finding: Early Phase Clinical Trials
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Covariates: Adjustment for
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Crossover Design
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Crossover Design: Rank-Based Robust Analysis
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Cutoff Designs
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Data Mining and Biopharmaceutical Research
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Data Monitoring Committees (DMC)
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Diagnostic Device Evaluation
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Diagnostic Imaging
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Diagnostic Procedure: Sensitivity and Specificity
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Disease Modification Effects: Design and Analysis
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Dissolution Profile Comparison: Statistical Methods
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Dissolution Profiles Comparison: Model-Independent Approaches
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Dosage Units Using Small and Large Sample Sizes: Uniformity
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Dose Finding Clinical Trials Using R
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Dose Proportionality
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Dose Response Analysis in Clinical Trials
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Dose Response Study Design
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Dose-Ranging Studies: Design Considerations
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Dropout
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Drug Development
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Drug Interchangeability: Biosimilar Products
entry
Drug Interchangeability: Criteria and Design
entry
Drug Interchangeability: Meta-Analysis for Safety Monitoring
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Drug Interchangeability: Generic Products, Prescribability and Switchability
entry
Ecologic Inference
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Ed50/Ed90
entry
Enrichment Design
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Enrichment Design with Patient Population Augmentation
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Equivalence Trials
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Ethnic Factors
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Expiration Dating Period
entry
Exploratory Factor Analysis
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Extra Variation Models
entry
Factor Analysis
entry
Factorial Designs
entry
False Discovery Rate (FDR)
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GEE in Cancer Research
entry
Generalizability Probability in Clinical Research
entry
Generalized Estimating Equation
entry
Generalized Inference
entry
Genetic Linkage and Linkage Disequilibrium Analysis
entry
Global Database and System
entry
Good Clinical Practice (GCP)
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Good Laboratory Practice (GLP)
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Good Programming Practice (GPP)
entry
Good Statistics Practice (GSP)
entry
Good Validation Practice (GVP)
entry
Group Sequential Methods
entry
Group Sequential Tests and Variance Heterogeneity: Clinical Trials
entry
Human Drug Abuse Trials
entry
Hypothesis Testing
entry
Imputation with Item Nonrespondents
entry
Imputation: Clinical Research
entry
In Vitro Testing: Bioequivalence
entry
In Vitro Testing: Dissolution Profile Comparison
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In Vitro Testing: Micronucleus Test
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Individual Bioequivalence
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Instrument Development and Validation
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Integrated Summary Report
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Intention-to-Treat Analyses (ITT)
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Interchangeability: Generic Drugs
entry
Interim Analysis
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International Conference on Harmonization (ICH)
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Item Response Theory
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IVRS/IWRS for Randomization
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Japan: Ministry of Health, Labour and Welfare and Pharmaceutical Administration
entry
Kaplan–Meier Estimator
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Kappa Coefficients: Medical Research
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Kruskal-Wallis Test for Ordered Categorical Data: Power Calculation
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Kullback–Leibler Divergence for Evaluating Equivalence
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Laboratory Analyses
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Latent Class Analysis
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Lilly Reference Ranges
entry
Local Influence Analysis
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LOCF: Validity
entry
Logistic Regression
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Logistic Regression Process: Predictive Model Building in Clinical Research
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Logistic Regression: Three-Point Designs
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McNemar’s Test
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MCP-Mod: Dose–Response Testing and Estimation
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Measuring Agreement
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MedDRA: Impact on Pharmaceutical Development
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Medical Devices
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Meta-Analysis of Therapeutic Trials
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Microarray Gene Expression
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Microdosing Studies
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Minimization Procedure
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Minimum Effective Dose
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Mixed Effects Models
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Mixed-Outcome Data
entry
MMRM with Missing Data
entry
Modified Large Sample Method
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Multicenter Trials
entry
Multicollinearity
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Multidimensional Data Analysis: Penalized Regression Methods
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Multinational Clinical Trials
entry
Multiple Comparisons
entry
Multiple Endpoints
entry
Multiple-Dose Bioequivalence Studies
entry
Multiple-Stage Designs: Phase II Cancer Trials
entry
Multiplicative Intensity Models
entry
Multiplicity in Clinical Trials
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Multi-Regional Clinical Trials: Consistency Test
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Multiregional Clinical Trials: Qualitative Consistency of Treatment Effects
entry
Multivariate Meta-Analysis
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Noninferiority Trials: Type I Error Rates
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Nonparametric Regression
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Odds Ratio
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Oncology Clinical Trials: Efficient Dose-Finding Designs
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Oncology Trials: Development Strategy
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Onset of Action
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Optimal Futility Interim Design
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Ordered Categorical Data: Test for
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Ordered Multiple Class Receiver Operating Characteristic (ROC) Analysis
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Ordinal Data: Crossover Design Analysis
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Parallel Design
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Patient Compliance
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Patient-Reported Outcomes
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Percentile Charts on Correlated Measures
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Personalized Medicine: Validation of Diagnostic Medical Devices
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Pharmaceutical Development: Statistical Designs
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Pharmacodynamic Issues
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Pharmacodynamics with Covariates
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Pharmacodynamics with No Covariates
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Pharmacoeconomics
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Pharmacoeconomics: Cost-Effectiveness Analysis
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Phase I Cancer Clinical Trials
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Phase II Clinical Development
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Phase III Clinical Trials with Response-Adaptive Allocation
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Placebo Effect
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Population Bioequivalence
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Population PK/PD Analysis
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Postmarketing Adverse Drug Event Signaling
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Postmarketing Surveillance
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Power
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Precision Medicine: Statistical Issues
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Prediction Trees
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Premarket Applications: Registries
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Principal Component Analysis
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Process Validation
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Profile Analysis
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Propensity Score Analysis and Its Application in Regulatory Settings
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Propensity Score: Methodology and Application to Clinical Studies
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Proportion of Treatment Effect
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Proportional Hazards Regression Model
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Protocol Development
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P-Values
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QT Analysis
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Quality by Design in Biopharmaceutical Development
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Randomization
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Randomized Trials with Clusters: Design and Analysis
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Rank Regression: Stability Analysis
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Regulatory Statistics
entry
Release Targets
entry
Reliability
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Repeated Measurement Designs: Missing Values
entry
Repeated Measures Data with Missing Values Analysis: Overview of Methods
entry
Reproductive and Developmental Studies
entry
Response Surface Methodology
entry
Response-Adaptive Designs
entry
Risk Ratio Analysis
entry
ROC Curve
entry
Sample Size Calculation: Nonparametrics
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Sample Size Calculation: Survival Data
entry
Sample Size Determination
entry
Sample Size Determination: Three-arm Ratio of Mean Differences Equivalence Test
entry
Sample Size Estimation: Generalized Estimating Equations (GEE) Method
entry
Sample Size for Multiregional Clinical Trials
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Sample Size Re-estimation Based on Observed Treatment Difference
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Sample Size Re-estimation Design with Robust Power
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Sample Size: Negative Binomial With Unequal Follow-Up Times
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Scaled Average Bioequivalence (SABE)
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Screening Design
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Seamless Adaptive Trial Design: Dose Selection Criteria
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Semi-Parametric Time-Varying Regression Models
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Sequential Estimation: Additive Hazards Rate Model with Staggered Entry
entry
Signal Detection in Pharmacovigilance
entry
Slope Approach: Assessment of Dose Proportionality and Linearity Under a Crossover Design
entry
Spatio-Temporal Modeling
entry
Specifications
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SROC Curve
entry
Stability Analysis: Frozen Drug Products
entry
Stability Matrix Designs
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Statistical Genetics
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Statistical Principles for Clinical Trials
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Statistical Process Control
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Statistical Significance
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Structural Equation Model
entry
Stuart–Maxwell Test
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Subgroup Analysis
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Subject-Treatment Interaction
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Surrogate Endpoint
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Survival Analysis
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Targeted Clinical Trials
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Testing for Qualitative Interaction
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Therapeutic Equivalence
entry
Thorough QT Studies
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Tiered Approach of Analytical Similarity Assessment: Statistical Methods
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Titration Design
entry
Toxicological Studies
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Traditional Chinese Medicine (TCM): Clinical Development
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Traditional Chinese Medicine (TCM): General Considerations
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Traditional Chinese Medicine (TCM): Unified Approach for Evaluation
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Translational Medicine: Concepts, Statistical Methods, and Related Issues
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Trend Estimation
entry
Two-Stage Adaptive Design: Analysis
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Two-Stage Design: Phase II Cancer Clinical Trials
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US Food and Drug Administration
entry
USP Tests
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Validation of Quantitative and Qualitative Assays
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Weibull Model: Group Sequential Design
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Z-Score
