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Biosimilars : design and analysis of follow-on biologics / / Shein-Chung Chow
Biosimilars : design and analysis of follow-on biologics / / Shein-Chung Chow
Autore Chow Shein-Chung <1955->
Edizione [1st ed.]
Pubbl/distr/stampa Boca Raton, : Taylor & Francis, 2014
Descrizione fisica 1 online resource (424 pages ) : illustrations
Disciplina 615.1/9
Collana Chapman & Hall/CRC biostatistics series
Soggetto topico Pharmaceutical biotechnology
Pharmaceutical biotechnology industry
Drugs - Generic substitution
Pharmaceutical policy
Biological products
ISBN 1-04-018910-5
0-429-07126-4
1-4665-7969-2
Classificazione MAT029000MED071000
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Nota di contenuto Introduction Background Fundamental Differences Regulatory Requirements Biosimilarity Interchangeability of Biological Drug Products Scientific Factors Aim and Scope of the Book Bioequivalence Experience for Small-Molecule Drug Products Background Process for Bioequivalence Assessment Issue of Drug Interchangeability Highly Variable Drugs Practical Issues Frequently Asked Questions Regulatory Requirements for Assessing Follow-On Biologics Background Definitions and Interpretations of Biosimilar Products Regulatory Requirements Review of the FDA Draft Guidances Global Harmonization Criteria for Similarity Introduction Criteria for Bioequivalence Similarity Factor for Dissolution Profile Comparison Measures of Consistency Comparison of Moment-Based and Probability-Based Criteria Alternative Criteria Statistical Methods for Assessing Average Biosimilarity Introduction Classic Methods for Assessing Biosimilarity Bayesian Methods Wilcoxon-Mann-Whitney Two One-Sided Tests Procedure Three-Arm Parallel Design General Approach for Assessing Biosimilarity Background Reproducibility Probability Development of the Biosimilarity Index Relationship of the Biosimilarity Criterion versus Variability Biosimilarity Index Based on the Bayesian Approach Consistency Approach Non-Inferiority versus Equivalence/Similarity Background Testing for Equality Testing for Noninferiority Testing for Superiority Testing for Equivalence Relationship among Testing for Noninferiority, Superiority, and Equivalence Determination of the Noninferiority Margin Sample Size Requirement When There Is a Switch in Hypothesis Testing Statistical Test for Biosimilarity in Variability Introduction Pitman-Morgan's Adjusted Test for Comparing Variabilities F -Type Test under Parallel Design Non-Parametrics Methods Alternative Methods Sample Size for Comparing Variabilities Introduction Comparing Intra-Subject Variability Comparing Inter-Subject Variability Comparing Total Variability Comparing Intra-Subject CVs Impact of Variability on Biosimilarity Limits for Assessing Follow-On Biologics Introduction Relationship between Variability and Biosimilarity Limits Scaled Biosimilarity Margins Simulations Discussions Drug Interchangeability Introduction Population and Individual Bioequivalence Interchangeability for Biosimilar Products Study Designs for Interchangeability Statistical Methods Issues on Immunogenicity Studies Introduction Regulatory Requirements Assay Development/Validation Design for Immunogenicity Studies Sample Size for Immunogenicity Studies CMC Requirements for Biological Products Introduction CMC Development Product Characterization and Specification Manufacture and Process Validation Quality Control/Assurance Reference Standards, Container Closure System, and Stability Test for Comparability in Manufacturing Process Introduction Biologic Manufacturing Process Consistency Index Test for Comparability Other Comparability Tests Stability Analysis of Biosimilar Products Introduction Regulatory Stability Guidelines on Biologicals Stability Indicating Profile and Expiration Dating Period Stability Designs Statistical Analysis Assessing Biosimilarity Using Biomarker Data Introduction Assessment of Biosimilarity Statistical Test for Biosimilarity Using Biomarker Data Numerical Study Current Issues in Biosimilar Studies Introduction Scientific Factors Current Issues References Index
Record Nr. UNINA-9910971108603321
Chow Shein-Chung <1955->  
Boca Raton, : Taylor & Francis, 2014
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui
Design and analysis of clinical trials : concepts and methodologies / / Shein-Chung Chow, Jen-pei Liu
Design and analysis of clinical trials : concepts and methodologies / / Shein-Chung Chow, Jen-pei Liu
Autore Chow Shein-Chung <1955->
Edizione [3rd ed.]
Pubbl/distr/stampa Hoboken, New Jersey, : Wiley, [2014]
Descrizione fisica 1 recurso en línea (893 páginas)
Altri autori (Persone) LiuJen-pei
Collana Wiley series in probability and statistics
Soggetto topico Biometría
Estudios clínicos - Métodos estadísticos
Estudios clínicos - Métodología
ISBN 1-118-45809-5
1-118-45816-8
1-118-45814-1
1-118-45813-3
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Record Nr. UNINA-9910139020803321
Chow Shein-Chung <1955->  
Hoboken, New Jersey, : Wiley, [2014]
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui
Design and analysis of clinical trials : concepts and methodologies / / Shein-Chung Chow, Jen-pei Liu
Design and analysis of clinical trials : concepts and methodologies / / Shein-Chung Chow, Jen-pei Liu
Autore Chow Shein-Chung <1955->
Edizione [3rd ed.]
Pubbl/distr/stampa Hoboken, New Jersey, : Wiley, [2014]
Descrizione fisica 1 recurso en línea (893 páginas)
Altri autori (Persone) LiuJen-pei
Collana Wiley series in probability and statistics
Soggetto topico Biometría
Estudios clínicos - Métodos estadísticos
Estudios clínicos - Métodología
ISBN 9781118458099
1118458095
9781118458167
1118458168
9781118458143
1118458141
9781118458136
1118458133
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Nota di contenuto Intro -- Design and Analysis of Clinical Trials -- Contents -- Preface -- PART I Preliminaries -- CHAPTER 1 Introduction -- 1.1 WHAT ARE CLINICAL TRIALS? -- 1.2 HISTORY OF CLINICAL TRIALS -- 1.3 REGULATORY PROCESS AND REQUIREMENTS -- 1.3.1 The Food and Drug Administration -- 1.3.2 FDA Regulations for Clinical Trials -- 1.3.3 Phases of Clinical Development -- 1.4 INVESTIGATIONAL NEW DRUG APPLICATION -- 1.4.1 Clinical Trial Protocol -- 1.4.2 Institutional Review Board -- 1.4.3 Safety Report -- 1.4.4 Treatment IND -- 1.4.5 Withdrawal and Termination of an IND -- 1.4.6 Communication with the FDA -- 1.5 NEW DRUG APPLICATION -- 1.5.1 Expanded Access -- 1.5.2 Abbreviated New Drug Application -- 1.5.3 Supplemental New Drug Application -- 1.5.4 Advisory Committee -- 1.6 CLINICAL DEVELOPMENT AND PRACTICE -- 1.6.1 Clinical Development Plan -- 1.6.2 Good Clinical Practice -- 1.7 AIMS AND STRUCTURE OF THE BOOK -- CHAPTER 2 Basic Statistical Concepts -- 2.1 INTRODUCTION -- 2.2 UNCERTAINTY AND PROBABILITY -- 2.2.1 Uncertainty -- 2.2.2 Probability -- 2.3 BIAS AND VARIABILITY -- 2.3.1 Bias -- 2.3.2 Variability -- 2.4 CONFOUNDING AND INTERACTION -- 2.4.1 Confounding -- 2.4.2 Interaction -- 2.5 DESCRIPTIVE AND INFERENTIAL STATISTICS -- 2.6 HYPOTHESES TESTING AND p-VALUES -- 2.6.1 p-Values -- 2.6.2 One-Sided Versus Two-Sided Hypotheses -- 2.7 CLINICAL SIGNIFICANCE AND CLINICAL EQUIVALENCE -- 2.8 REPRODUCIBILITY AND GENERALIZABILITY -- 2.8.1 Reproducibility -- 2.8.2 Generalizability -- CHAPTER 3 Basic Design Considerations -- 3.1 INTRODUCTION -- 3.2 GOALS OF CLINICAL TRIALS -- 3.3 TARGET POPULATION AND PATIENT SELECTION -- 3.3.1 Eligibility Criteria -- 3.3.2 Patient Selection Process -- 3.3.3 Ethical Considerations -- 3.4 SELECTION OF CONTROLS -- 3.4.1 Placebo Concurrent Control -- 3.4.2 Dose-Response Concurrent Control -- 3.4.3 Active (Positive) Concurrent Control.
3.4.4 No Treatment Concurrent Control -- 3.4.5 Historical Control -- 3.5 STATISTICAL CONSIDERATIONS -- 3.5.1 Efficacy and Safety Assessment -- 3.5.2 Sample Size Estimation -- 3.5.3 Interim Analysis and Data Monitoring -- 3.5.4 Statistical and Clinical Inference -- 3.6 OTHER ISSUES -- 3.6.1 Single Site Versus Multisites -- 3.6.2 Treatment Duration -- 3.6.3 Patient Compliance -- 3.6.4 Missing Value and Dropout -- 3.7 DISCUSSION -- CHAPTER 4 Randomization and Blinding -- 4.1 INTRODUCTION -- 4.2 RANDOMIZATION MODELS -- 4.2.1 Population Model -- 4.2.2 Invoked Population Model -- 4.2.3 Randomization Model -- 4.2.4 Stratification -- 4.3 RANDOMIZATION METHODS -- 4.3.1 Complete Randomization -- 4.3.2 Permuted-Block Randomization -- 4.3.3 Adaptive Randomization -- 4.4 IMPLEMENTATION OF RANDOMIZATION -- 4.4.1 Generation, Labeling, and Packaging -- 4.4.2 Random Assignment -- 4.5 GENERALIZATION OF CONTROLLED RANDOMIZED TRIALS -- 4.6 BLINDING -- 4.7 DISCUSSION -- PART II Designs and Their Classifications -- CHAPTER 5 Designs for Clinical Trials -- 5.1 INTRODUCTION -- 5.2 PARALLEL GROUP DESIGNS -- 5.2.1 Run-in Periods -- 5.2.2 Examples of Parallel Group Design in Clinical Trials -- 5.3 CLUSTERED RANDOMIZED DESIGNS -- 5.4 CROSSOVER DESIGNS -- 5.4.1 Higher-Order Crossover Designs -- 5.4.2 Williams Designs -- 5.4.3 Balanced Incomplete Block Design -- 5.4.4 Examples of Crossover Design in Clinical Trials -- 5.5 TITRATION DESIGNS -- 5.5.1 Standard Titration Design -- 5.5.2 Forced Dose-Escalation Design -- 5.6 ENRICHMENT DESIGNS -- 5.7 GROUP SEQUENTIAL DESIGNS -- 5.8 PLACEBO-CHALLENGING DESIGNS -- 5.8.1 Statistical Model and Inferences -- 5.9 BLINDED READER DESIGNS -- 5.10 DISCUSSION -- CHAPTER 6 Designs for Cancer Clinical Trials -- 6.1 INTRODUCTION -- 6.2 GENERAL CONSIDERATIONS FOR PHASE ICANCER CLINICAL TRIALS -- 6.3 SINGLE-STAGE UP-AND-DOWN PHASE I DESIGNS.
6.3.1 Design A-Standard Dose-Escalation Design -- 6.3.2 Design B -- 6.3.3 Design D -- 6.4 TWO-STAGE UP-AND-DOWN PHASE I DESIGNS -- 6.4.1 Design BD -- 6.4.2 Accelerated Titration Designs -- 6.5 CONTINUAL REASSESSMENT METHOD PHASE I DESIGNS -- 6.6 OPTIMAL AND FLEXIBLE MULTIPLE-STAGE DESIGNS -- 6.6.1 Single-Arm Trials -- 6.6.2 Multiple-Arm Trials -- 6.7 RANDOMIZED PHASE II DESIGNS -- 6.8 DISCUSSION -- CHAPTER 7 Classification of Clinical Trials -- 7.1 INTRODUCTION -- 7.2 MULTICENTER TRIALS -- 7.2.1 Treatment-by-Center Interaction -- 7.2.2 Practical Issues -- 7.3 SUPERIORITY TRIALS -- 7.4 ACTIVE CONTROL AND EQUIVALENCE/NONINFERIORITY TRIALS -- 7.4.1 Primary Objectives -- 7.4.2 Issues in Active Control Equivalence Trials -- 7.4.3 Interpretation of the Results of Active Control Trials -- 7.4.4 Equivalence/Noninferiority Limits -- 7.4.5 Statistical Methods -- 7.5 DOSE-RESPONSE TRIALS -- 7.5.1 Randomized Parallel Dose-Response Designs -- 7.5.2 Crossover Dose-Response Design -- 7.5.3 Forced Titration (Dose-Escalation) Design -- 7.5.4 Optional Titration Design (Placebo-Controlled Titration to Endpoint) -- 7.6 COMBINATION TRIALS -- 7.6.1 Fixed-Combination Prescription Drugs -- 7.6.2 Multilevel Factorial Design -- 7.6.3 Global Superiority of Combination Drugs -- 7.6.4 Method of Response Surface -- 7.7 BRIDGING STUDIES AND GLOBAL TRIALS -- 7.7.1 Introduction -- 7.7.2 Ethnic Sensitivity and Necessity of Bridging Studies -- 7.7.3 Types of Bridging Studies -- 7.7.4 Assessment of Similarity Based on Bridging Evidence -- 7.8 VACCINE CLINICAL TRIALS -- 7.8.1 Basic Design and Statistical Considerations -- 7.8.2 Types of Vaccine Immunogenicity Trials -- 7.8.3 Statistical Methods -- 7.9 QT STUDIES -- 7.9.1 Study Designs and Models -- 7.9.2 Power and Sample Size Calculation -- 7.9.3 Allocation Optimization -- 7.9.4 Remarks -- 7.10 DISCUSSION.
PART III Analysis of Clinical Data -- CHAPTER 8 Analysis of Continuous Data -- 8.1 INTRODUCTION -- 8.2 ESTIMATION -- 8.3 TEST STATISTICS -- 8.3.1 Paired t Test -- 8.3.2 Two-Sample t Test -- 8.4 ANALYSIS OF VARIANCE -- 8.4.1 One-Way Classification -- 8.4.2 Simultaneous Confidence Intervals -- 8.4.3 Two-Way Classification -- 8.5 ANALYSIS OF COVARIANCE -- 8.6 NONPARAMETRIC METHODS -- 8.6.1 Wilcoxon Signed Rank Test -- 8.6.2 Wilcoxon Rank Sum Test -- 8.6.3 Kruskal-Wallis Test -- 8.7 REPEATED MEASURES -- 8.7.1 Assessment of Overall Average Effect Across Time -- 8.7.2 Detection of Time Effect -- 8.7.3 Treatment-by-Time Interaction -- 8.7.4 Method of Generalized Estimating Equations (GEEs) -- 8.8 DISCUSSION -- CHAPTER 9 Analysis of Categorical Data -- 9.1 INTRODUCTION -- 9.2 STATISTICAL INFERENCE FOR ONE SAMPLE -- 9.3 INFERENCE OF INDEPENDENT SAMPLES -- 9.4 ORDERED CATEGORICAL DATA -- 9.5 COMBINING CATEGORICAL DATA -- 9.6 MODEL-BASED METHODS -- 9.7 REPEATED CATEGORICAL DATA -- 9.8 DISCUSSION -- CHAPTER 10 Censored Data and Interim Analysis -- 10.1 INTRODUCTION -- 10.2 ESTIMATION OF THE SURVIVAL FUNCTION -- 10.3 COMPARISON BETWEEN SURVIVAL FUNCTIONS -- 10.4 COX'S PROPORTIONAL HAZARD MODEL -- 10.5 CALENDAR TIME AND INFORMATION TIME -- 10.6 GROUP SEQUENTIAL METHODS -- 10.7 DISCUSSION -- CHAPTER 11 Sample Size Determination -- 11.1 INTRODUCTION -- 11.2 BASIC CONCEPT -- 11.2.1 Study Objectives and Hypotheses -- 11.2.2 Type I and Type II Errors -- 11.2.3 Precision Analysis -- 11.2.4 Power Analysis -- 11.3 TWO SAMPLES -- 11.3.1 One-Sample Test for Mean -- 11.3.2 Two-Sample Test for Comparing Means -- 11.4 MULTIPLE SAMPLES -- 11.4.1 Sample Size Calculations for Analysis of Variance Models -- 11.4.2 Sample Size Calculations for Generalized Linear Models -- 11.5 CENSORED DATA -- 11.6 DOSE-RESPONSE STUDIES -- 11.6.1 Dose-Response Relationship.
11.6.2 Minimum Effective Dose -- 11.7 CROSSOVER DESIGNS -- 11.7.1 Point Hypotheses for Equality -- 11.7.2 Interval Hypotheses for Equivalence -- 11.7.3 Higher-Order Crossover Designs -- 11.8 EQUIVALENCE AND NONINFERIORITY TRIALS -- 11.8.1 Independent Binary Endpoints -- 11.8.2 Paired Binary Endpoints -- 11.8.3 Independent Censored Endpoints -- 11.9 MULTIPLE-STAGE DESIGN IN CANCER TRIALS -- 11.10 MULTINATIONAL TRIALS -- 11.10.1 Selection of the Number of Sites -- 11.10.2 Sample Size Calculation and Allocation -- 11.11 COMPARING VARIABILITIES -- 11.11.1 Comparing Intrasubject Variabilities -- 11.11.2 Comparing Intersubject Variabilities -- 11.12 DISCUSSION -- PART IV Issues in Evaluation -- CHAPTER 12 Issues in Efficacy Evaluation -- 12.1 INTRODUCTION -- 12.2 BASELINE COMPARISON -- 12.3 INTENTION-TO-TREAT PRINCIPLE AND EFFICACY ANALYSIS -- 12.4 ADJUSTMENT FOR COVARIATES -- 12.5 MULTICENTER TRIALS -- 12.6 MULTIPLICITY -- 12.6.1 Multiple Comparisons -- 12.6.2 Multiple Endpoints -- 12.6.3 Subgroup Analysis -- 12.7 DATA MONITORING -- 12.7.1 Definition and Objectives -- 12.7.2 Regulatory Concerns -- 12.7.3 Early and Late Stages of Drug Development -- 12.7.4 Administrative Interim Analyses -- 12.7.5 Data Monitoring Committee -- 12.8 USE OF GENETIC INFORMATION FOR EVALUATION OF EFFICACY -- 12.9 SAMPLE SIZE REESTIMATION -- 12.10 DISCUSSION -- CHAPTER 13 Safety Assessment -- 13.1 INTRODUCTION -- 13.2 EXTENT OF EXPOSURE -- 13.2.1 Risk of Exposure -- 13.2.2 Absorbing Events -- 13.2.3 Recurring Events with Negligible Duration -- 13.2.4 Recurring Events with Nonnegligible Duration -- 13.2.5 Laboratory Data -- 13.3 CODING OF ADVERSE EVENTS -- 13.3.1 Definitions of Adverse Events -- 13.3.2 COSTART -- 13.3.3 MedDRA -- 13.3.4 Common Toxicity Criteria -- 13.4 ANALYSIS OF ADVERSE EVENTS -- 13.4.1 Adverse Event Data Listing -- 13.4.2 Summary Tables of Adverse Events.
13.4.3 Graphical Presentation.
Record Nr. UNINA-9910822202603321
Chow Shein-Chung <1955->  
Hoboken, New Jersey, : Wiley, [2014]
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui
Statistics in regulatory science / / by Shein-Chung Chow
Statistics in regulatory science / / by Shein-Chung Chow
Autore Chow Shein-Chung <1955->
Edizione [1st ed.]
Pubbl/distr/stampa Boca Raton : , : CRC Press, , [2018]
Descrizione fisica 1 online resource (553 pages)
Disciplina 615.19
Collana Chapman and Hall/CRC Biostatistics Series
Soggetto topico Drugs - Testing
Clinical trials - Statistical methods
Pharmacy - Research
Research - Statistical methods
ISBN 1-000-71081-5
1-000-71003-3
0-429-27506-4
Formato Materiale a stampa
Livello bibliografico Monografia
Lingua di pubblicazione eng
Nota di contenuto Totality-of-the-evidence -- Hypotheses testing versus confidence interval -- Endpoint selection -- Non-inferiority margin -- Missing data -- Multiplicity -- Sample size -- Reproducible research -- Extrapolation -- Consistency evaluation -- Drug products with multiple components -- Adaptive trial design -- Selection criteria in adaptive dose finding -- Generic drugs and biosimilars -- Precision and personalized medicine -- Big data analytics -- Rare disease drug development.
Record Nr. UNINA-9910861081003321
Chow Shein-Chung <1955->  
Boca Raton : , : CRC Press, , [2018]
Materiale a stampa
Lo trovi qui: Univ. Federico II
Opac: Controlla la disponibilità qui