Encyclopedia of Biopharmaceutical Statistics

Encyclopedia of

Biopharmaceutical Statistics, th 4  Edition Volume I – IV

Encyclopedia of

Biopharmaceutical Statistics, th 4  Edition Volume I – IV

edited by Shein-Chung Chow, PhD

CRC Press Taylor & Francis Group 6000 Broken Sound Parkway NW, Suite 300 Boca Raton, FL 33487-2742 © 2018 by Taylor & Francis Group, LLC CRC Press is an imprint of Taylor & Francis Group, an lnforma business No claim to original U.S. Government works Printed on acid-free paper International Standard Book Number-13: 978-1-4987-3395-3 (Set), 978-0-8153-6314-9 (Vol. 1) This book contains information obtained from authentic and highly regarded sources. Reasonable efforts have been made to publish reliable data and information, but the author and publisher cannot assume responsibility for the validity of all materials or the consequences of their use. The authors and publishers have attempted to trace the copyright holders of all material reproduced in this publication and apologize to copyright holders if permission to publish in this form has not been obtained. If any copyright material has not been acknowledged please write and let us know so we may rectify in any future reprint. Except as permitted under U.S. Copyright Law, no part of this book may be reprinted, reproduced, transmitted, or utilized in any form by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying, microfilming, and recording, or in any information storage or retrieval system, without written permission from the publishers. For permission to photocopy or use material electronically from this work, please access www.copyright.com (http:// www.copyright.com/) or contact the Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978-750-8400. CCC is a not-for-profit organization that provides licenses and registration for a variety of users. For organizations that have been granted a photocopy license by the CCC, a separate system of payment has been arranged. Trademark Notice: Product or corporate names may be trademarks or registered trademarks, and are used only for identification and explanation without intent to infringe. Visit the Taylor & Francis Web site at http://www.taylorandfrancis.com and the CRC Press Web site at http://www.crcpress.com

Brief Content

Biologics . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Biomarker Development with Applications to Genetics Data: Statistical Tests . . . . . . . . . . Biomarker: Clinical Trials . . . . . . . . . . . . . . . . . . Biopharmaceutics . . . . . . . . . . . . . . . . . . . . . . . . . . Biosimilar Studies: Sample Size . . . . . . . . . . . . . . Biosimilar Studies: Three-Arm Design . . . . . . . . Biosimilarity Assessment . . . . . . . . . . . . . . . . . . . Biosimilarity of Follow-On Biologics . . . . . . . . . . Bivariate Zero-Inflated Poisson Populations: Statistical Test for Homogeneity . . . . . . . . . . . Blinded Sample Size Re-Estimation . . . . . . . . . . . Blinding . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Bootstrap, The . . . . . . . . . . . . . . . . . . . . . . . . . . . . Botanical Drug Product Development: Scientific Issues . . . . . . . . . . . . . . . . . . . . . . . . . Bracketing Design . . . . . . . . . . . . . . . . . . . . . . . . . Bridging Studies . . . . . . . . . . . . . . . . . . . . . . . . . . . Bridging Studies: Statistical Methods . . . . . . . . . Calibration . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Canadian Health Products and Food Branch (HPFB) and Therapeutic Products Directorate (TPD) . . . . . . . . . . . . . . . . . . . . . . . Cancer Chemotherapy: Maximum Tolerable Dose . . . . . . . . . . . . . . . . . . . . . . . . Cancer Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Carcinogenicity Studies of Pharmaceuticals . . . . . . . . . . . . . . . . . . . . . . . . Carry-Forward Analysis . . . . . . . . . . . . . . . . . . . . Case-Control Studies: Inference in . . . . . . . . . . . Center Weighting: Multicenter Trials . . . . . . . . . Clinical Data Management . . . . . . . . . . . . . . . . . . Clinical Endpoint . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Endpoint BE Studies: Generic Drugs . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Inspection: Statistical Process . . . . . . . . Clinical Pharmacology . . . . . . . . . . . . . . . . . . . . . Clinical Research: Benefit and Risk Assessment . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Research: Comparing Variabilities . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Research: Outlier Detection . . . . . . . . . . Clinical Research: Reproducibility Probability . . . . . . . . . . . . . . . . . . . . . . . . . . . . .

Volume I Acceptance Sampling . . . . . . . . . . . . . . . . . . . . . . . Active Control Non-Inferiority Trials: Hypotheses and False Positive Rate . . . . . . . . . Active Control Trials . . . . . . . . . . . . . . . . . . . . . . . Active Controlled Clinical Trials: Noninferiority Analysis . . . . . . . . . . . . . . . . . . Adaptive Design Clinical Trials: Case Studies . . . . . . . . . . . . . . . . . . . . . . . . . . . . Adaptive Design Methods in Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Adaptive Survival Trials . . . . . . . . . . . . . . . . . . . . Adverse Event Reporting . . . . . . . . . . . . . . . . . . . . Alpha Spending Function . . . . . . . . . . . . . . . . . . . Ames Test . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Analysis of 2 × K Tables . . . . . . . . . . . . . . . . . . . . Analysis of Clustered Binary Data . . . . . . . . . . . . Analysis of Clustered Categorical Data . . . . . . . . Analysis of Heritability . . . . . . . . . . . . . . . . . . . . . Analysis of Variance (ANOVA) . . . . . . . . . . . . . . . Analytical Similarity Assessment . . . . . . . . . . . . . ANCOVA Approach: Premarketing Shelf Life Determination with Multiple Factors . . . . . . . . . . . . . . . . . . . . . . . . Assay Development . . . . . . . . . . . . . . . . . . . . . . . . Assay Validation . . . . . . . . . . . . . . . . . . . . . . . . . . . Assay Validation: Evaluation of Linearity . . . . . . . . . . . . . . . . . . . . . . . . . . . . Bayesian Designs: Phase II Oncology Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . Bayesian Estimate: Concordance Correlation Coefficient . . . . . . . . . . . . . . . . . . . Bayesian Methods: Meta-Analysis . . . . . . . . . . . . Bayesian Methods: Stability Analysis . . . . . . . . . Bayesian Model: Prior Effective Sample Size . . . . . . . . . . . . . . . . . . . . . . . . . . . . Bayesian Statistics . . . . . . . . . . . . . . . . . . . . . . . . . Bayesian Statistics: Medical Device Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . Binary 2 × 2 Crossover Trials . . . . . . . . . . . . . . . Bioassay . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Bioavailability and Bioequivalence . . . . . . . . . . . . Bioinformatics . . . . . . . . . . . . . . . . . . . . . . . . . . . .

1 8 14 23 28 36 50 53 59 67 75 82 88 98 104 116

125 130 138 146 154 164 173 180 186 190 198 206 213 216 224 v

233 243 251 254 274 277 281 304 316 323 336 342 348 359 361 366 370

378 384 390 398 416 422 435 444 451 455 462 469 472 486 502 510

vi

Brief Content

Volume I (cont’d .) Clinical Trial Design: Statistical Assurance . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trial Designs with Prospective Patient Population Enrichment by Response . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trial Process . . . . . . . . . . . . . . . . . . . . . . Clinical Trial Simulation . . . . . . . . . . . . . . . . . . . Clinical Trial Simulations: Earlier Development Phases . . . . . . . . . . . . . . . . . . . . . Clinical Trial Simulations: Later Development Phases . . . . . . . . . . . . . . . . . . . . . Clinical Trial: Combination Drug Development . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trial: Failure-Time Model . . . . . . . . . . . Clinical Trial: N-of-1 Design Analysis . . . . . . . . . Clinical Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trials: Controversial Issues . . . . . . . . . .

523

526 532 536 539 544 552 556 564 572 580

Volume II Clinical Trials: Interval-censored Failure Time Data . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trials: Investigating Quality-of-Life . . . . . . . . . . . . . . . . . . . . . . . . . . Clinical Trials: Meta-Analysis using R . . . . . . . . Clinical Trials: Response-Adaptive Repeated Measurement Designs . . . . . . . . . . . Clinical Trials: Selection of Control . . . . . . . . . . Clinical Trials: Vaccine Development . . . . . . . . . Cluster Randomized Trials: Sample Size . . . . . . Cluster Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Clustered Study Designs: Power Analysis . . . . . . Companion Diagnostics . . . . . . . . . . . . . . . . . . . . . Comparability Studies: Statistical Considerations . . . . . . . . . . . . . . . . . . . . . . . . . . Confidence Interval and Hypothesis Testing . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . Confounding and Interaction . . . . . . . . . . . . . . . . Content Uniformity . . . . . . . . . . . . . . . . . . . . . . . . Continual Reassessment Methods . . . . . . . . . . . . Contract Research Organization (CRO): Early 2000s . . . . . . . . . . . . . . . . . . . . . . . . . . . . Contract Research Organization (CRO) . . . . . . . Controlled Clinical Trials: P-Values, Evidence, and Multiplicity Considerations . . . . . . . . . . . . . . . . . . . . . . . . . . Correlated Probit Model . . . . . . . . . . . . . . . . . . . .

589 597 603 612 622 628 645 648 658 672 682 699 703 709 712 716 721

725 741

Cost-Effectiveness Analysis ....