Multiple Testing Problems in Pharmaceutical Statistics

Multiple Testing Problems in Pharmaceutical Statistics

Useful Statistical Approaches for Addressing Multiplicity IssuesIncludes practical examples from recent trials Bringing together leading statisticians, scientists, and clinicians from the pharmaceutical industry, academia, and regulatory ...

Author: Alex Dmitrienko

Publisher: CRC Press

ISBN: 1584889853

Category: Mathematics

Page: 320

View: 752

Useful Statistical Approaches for Addressing Multiplicity IssuesIncludes practical examples from recent trials Bringing together leading statisticians, scientists, and clinicians from the pharmaceutical industry, academia, and regulatory agencies, Multiple Testing Problems in Pharmaceutical Statistics explores the rapidly growing area of multiple c
Categories: Mathematics

Biopharmaceutical Applied Statistics Symposium

Biopharmaceutical Applied Statistics Symposium

The power issue would generally be like those for testing multiple hypotheses in a non-GS trial. Acknowledgements The authors are ... Statistics in Medicine, 33(4), 693–713. ... Boca Raton, FL: Chapman & Hall/CRC Biostatistics Series.

Author: Karl E. Peace

Publisher: Springer

ISBN: 9789811078200

Category: Medical

Page: 426

View: 125

This BASS book Series publishes selected high-quality papers reflecting recent advances in the design and biostatistical analysis of biopharmaceutical experiments – particularly biopharmaceutical clinical trials. The papers were selected from invited presentations at the Biopharmaceutical Applied Statistics Symposium (BASS), which was founded by the first Editor in 1994 and has since become the premier international conference in biopharmaceutical statistics. The primary aims of the BASS are: 1) to raise funding to support graduate students in biostatistics programs, and 2) to provide an opportunity for professionals engaged in pharmaceutical drug research and development to share insights into solving the problems they encounter. The BASS book series is initially divided into three volumes addressing: 1) Design of Clinical Trials; 2) Biostatistical Analysis of Clinical Trials; and 3) Pharmaceutical Applications. This book is the third of the 3-volume book series. The topics covered include: Targeted Learning of Optimal Individualized Treatment Rules under Cost Constraints, Uses of Mixture Normal Distribution in Genomics and Otherwise, Personalized Medicine – Design Considerations, Adaptive Biomarker Subpopulation and Tumor Type Selection in Phase III Oncology Trials, High Dimensional Data in Genomics; Synergy or Additivity - The Importance of Defining the Primary Endpoint, Full Bayesian Adaptive Dose Finding Using Toxicity Probability Interval (TPI), Alpha-recycling for the Analyses of Primary and Secondary Endpoints of Clinical Trials, Expanded Interpretations of Results of Carcinogenicity Studies of Pharmaceuticals, Randomized Clinical Trials for Orphan Drug Development, Mediation Modeling in Randomized Trials with Non-normal Outcome Variables, Statistical Considerations in Using Images in Clinical Trials, Interesting Applications over 30 Years of Consulting, Uncovering Fraud, Misconduct and Other Data Quality Issues in Clinical Trials, Development and Evaluation of High Dimensional Prognostic Models, and Design and Analysis of Biosimilar Studies.
Categories: Medical

Understanding Biostatistics

Understanding Biostatistics

Dmitrienko, A., Tamhane, AC. and Bretz, F. (2010) Multiple Testing Problems in Pharmaceutical Statistics, CRC Biostatistics Series. Boca Raton, FL: Chapman & Hall/CRC. Feynman, RP. and Leighton, R. (1992) Surely You're Joking Mr Feynman ...

Author: Anders Källén

Publisher: John Wiley & Sons

ISBN: 9781119993506

Category: Medical

Page: 388

View: 718

Understanding Biostatistics looks at the fundamentals of biostatistics, using elementary statistics to explore the nature of statistical tests. This book is intended to complement first-year statistics and biostatistics textbooks. The main focus here is on ideas, rather than on methodological details. Basic concepts are illustrated with representations from history, followed by technical discussions on what different statistical methods really mean. Graphics are used extensively throughout the book in order to introduce mathematical formulae in an accessible way. Key features: Discusses confidence intervals and p-values in terms of confidence functions. Explains basic statistical methodology represented in terms of graphics rather than mathematical formulae, whilst highlighting the mathematical basis of biostatistics. Looks at problems of estimating parameters in statistical models and looks at the similarities between different models. Provides an extensive discussion on the position of statistics within the medical scientific process. Discusses distribution functions, including the Guassian distribution and its importance in biostatistics. This book will be useful for biostatisticians with little mathematical background as well as those who want to understand the connections in biostatistics and mathematical issues.
Categories: Medical

Clinical Trials Design in Operative and Non Operative Invasive Procedures

Clinical Trials Design in Operative and Non Operative Invasive Procedures

Some methods for strengthening the common v2 tests. Biometrics. ... Dmitrienko A, Tamhane AC, Bretz F. Multiple testing problems in pharmaceutical statistics. 1st ed. Boca Raton: Chapman and Hall, CRC Biostatistics Series; 2009.

Author: Kamal M.F. Itani

Publisher: Springer

ISBN: 9783319538778

Category: Medical

Page: 495

View: 821

The aim of this text is to provide the framework for building a clinical trial as it pertains to operative and non operative invasive procedures, how to get it funded and how to conduct such a trial up to publication of results The text provides all details of building a scientifically and ethically valid proposal, including how to build the infrastructure for a clinical trial and how to move it forward through various funding agencies. The text also presents various types of clinical trials, the use of implantable devices and FDA requirements, and adjuncts to clinical trials and interaction with industry Clinical Trials Design in Invasive Operative and Non Operative Procedures will be of interest to all specialists of surgery, anesthesiologists, interventional radiologists, gastroenterologists, cardiologists, and pulmonologists
Categories: Medical

Modern Issues and Methods in Biostatistics

Modern Issues and Methods in Biostatistics

Chapman and Hall/CRC, Boca Raton (2010) Dunnett, C.W.: A multiple comparison procedure for comparing several treatments with a control. J. Am. Stat. Assoc. 50, 1096–1121 (1955) European Medicines Agency: ICH Topic E 9 Statistical ...

Author: Mark Chang

Publisher: Springer Science & Business Media

ISBN: 9781441998422

Category: Medical

Page: 307

View: 908

Classic biostatistics, a branch of statistical science, has as its main focus the applications of statistics in public health, the life sciences, and the pharmaceutical industry. Modern biostatistics, beyond just a simple application of statistics, is a confluence of statistics and knowledge of multiple intertwined fields. The application demands, the advancements in computer technology, and the rapid growth of life science data (e.g., genomics data) have promoted the formation of modern biostatistics. There are at least three characteristics of modern biostatistics: (1) in-depth engagement in the application fields that require penetration of knowledge across several fields, (2) high-level complexity of data because they are longitudinal, incomplete, or latent because they are heterogeneous due to a mixture of data or experiment types, because of high-dimensionality, which may make meaningful reduction impossible, or because of extremely small or large size; and (3) dynamics, the speed of development in methodology and analyses, has to match the fast growth of data with a constantly changing face. This book is written for researchers, biostatisticians/statisticians, and scientists who are interested in quantitative analyses. The goal is to introduce modern methods in biostatistics and help researchers and students quickly grasp key concepts and methods. Many methods can solve the same problem and many problems can be solved by the same method, which becomes apparent when those topics are discussed in this single volume.
Categories: Medical

Handbook of Statistical Methods for Randomized Controlled Trials

Handbook of Statistical Methods for Randomized Controlled Trials

Springer texts in statistics. Springer, third edition, 2002. [2] A. Dmitrienko, A.C. Tamhane, and F. Bretz. Multiple Testing Problems in Pharmaceutical Statistics. Chapman & Hall/CRC Biostatistics Series. CRC Press, 2009.

Author: KyungMann Kim

Publisher: CRC Press

ISBN: 9781498714648

Category: Mathematics

Page: 654

View: 593

Statistical concepts provide scientific framework in experimental studies, including randomized controlled trials. In order to design, monitor, analyze and draw conclusions scientifically from such clinical trials, clinical investigators and statisticians should have a firm grasp of the requisite statistical concepts. The Handbook of Statistical Methods for Randomized Controlled Trials presents these statistical concepts in a logical sequence from beginning to end and can be used as a textbook in a course or as a reference on statistical methods for randomized controlled trials. Part I provides a brief historical background on modern randomized controlled trials and introduces statistical concepts central to planning, monitoring and analysis of randomized controlled trials. Part II describes statistical methods for analysis of different types of outcomes and the associated statistical distributions used in testing the statistical hypotheses regarding the clinical questions. Part III describes some of the most used experimental designs for randomized controlled trials including the sample size estimation necessary in planning. Part IV describe statistical methods used in interim analysis for monitoring of efficacy and safety data. Part V describe important issues in statistical analyses such as multiple testing, subgroup analysis, competing risks and joint models for longitudinal markers and clinical outcomes. Part VI addresses selected miscellaneous topics in design and analysis including multiple assignment randomization trials, analysis of safety outcomes, non-inferiority trials, incorporating historical data, and validation of surrogate outcomes.
Categories: Mathematics

Encyclopedia of Biopharmaceutical Statistics Four Volume Set

Encyclopedia of Biopharmaceutical Statistics   Four Volume Set

Sullivan, L.M.; D'Agostino, R.B. SrRobustness and power of analysis of covariance applied to ordinal scaled data as arising ... Bretz, F. Multiple Testing Problems in Pharmaceutical Statistics; Chapman & Hall/ CRC: Boca Raton, FL, 2010.

Author: Shein-Chung Chow

Publisher: CRC Press

ISBN: 9781351110259

Category: Mathematics

Page: 2780

View: 996

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 third 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.
Categories: Mathematics

Pediatric Kidney Disease

Pediatric Kidney Disease

Dmitrienko A, Tamhane AC, Bretz F, editors. Multiple testing problems in pharmaceutical statistics. Boca Raton: Chapman & Hall/CRC Biostatistics Series; 2009. CPMP. Points to consider on multiplicity issues in clinical trials. 2002.

Author: Denis F. Geary

Publisher: Springer

ISBN: 9783662529720

Category: Medical

Page: 2005

View: 556

The new edition of this valuable clinical resource offers a state of the art, comprehensive review on every clinical condition encountered in pediatric nephrology. International experts present the latest knowledge on epidemiology, diagnosis, management, and prognosis in one concise, clinically focused text, in which care has been taken to couple just the right amount of "need-to-know" basic science with practical clinical guidance that will enable the reader to make efficient, informed decisions. The topics covered include: disorders of renal development, glomerular disorders, the kidney and systemic disease, renal tubular disorders, tubulointerstitial disease, urinary tract disorders, acute kidney injury, hypertension, chronic and end-stage renal disease, and renal replacement therapy. The full-color, highly visual, meticulously crafted format will ensure that the practitioner is able to source and apply information with remarkable ease.
Categories: Medical

Clinical Trial Methodology

Clinical Trial Methodology

Basic Statistics and Pharmaceutical Statistical Applications, James E. De Muth . ... Ming T. Tan, Guo-Liang Tian, and Kai Wang Ng Multiple Testing Problems in Pharmaceutical Statistics, Alex Dmitrienko, Ajit C. Tamhane, and Frank Bretz ...

Author: Karl E. Peace

Publisher: CRC Press

ISBN: 1584889187

Category: Mathematics

Page: 420

View: 923

Now viewed as its own scientific discipline, clinical trial methodology encompasses the methods required for the protection of participants in a clinical trial and the methods necessary to provide a valid inference about the objective of the trial. Drawing from the authors’ courses on the subject as well as the first author’s more than 30 years working in the pharmaceutical industry, Clinical Trial Methodology emphasizes the importance of statistical thinking in clinical research and presents the methodology as a key component of clinical research. From ethical issues and sample size considerations to adaptive design procedures and statistical analysis, the book first covers the methodology that spans every clinical trial regardless of the area of application. Crucial to the generic drug industry, bioequivalence clinical trials are then discussed. The authors describe a parallel bioequivalence clinical trial of six formulations incorporating group sequential procedures that permit sample size re-estimation. The final chapters incorporate real-world case studies of clinical trials from the authors’ own experiences. These examples include a landmark Phase III clinical trial involving the treatment of duodenal ulcers and Phase III clinical trials that contributed to the first drug approved for the treatment of Alzheimer’s disease. Aided by the U.S. FDA, the U.S. National Institutes of Health, the pharmaceutical industry, and academia, the area of clinical trial methodology has evolved over the last six decades into a scientific discipline. This guide explores the processes essential for developing and conducting a quality clinical trial protocol and providing quality data collection, biostatistical analyses, and a clinical study report, all while maintaining the highest standards of ethics and excellence.
Categories: Mathematics

Statistical Methods in Biomarker and Early Clinical Development

Statistical Methods in Biomarker and Early Clinical Development

Permutation criteria to evaluate multiple clinical endpoints in a proof-of-concept study: lessons from Pre-RELAX-AHF. ... F., ed., Multiple Testing Problems in Pharmaceutical Statistics (2009), Chapman & Hall/CRC Biostatistics Series.

Author: Liang Fang

Publisher: Springer Nature

ISBN: 9783030315030

Category: Medical

Page: 348

View: 386

This contributed volume offers a much-needed overview of the statistical methods in early clinical drug and biomarker development. Chapters are written by expert statisticians with extensive experience in the pharmaceutical industry and regulatory agencies. Because of this, the data presented is often accompanied by real world case studies, which will help make examples more tangible for readers. The many applications of statistics in drug development are covered in detail, making this volume a must-have reference. Biomarker development and early clinical development are the two critical areas on which the book focuses. By having the two sections of the book dedicated to each of these topics, readers will have a more complete understanding of how applying statistical methods to early drug development can help identify the right drug for the right patient at the right dose. Also presented are exciting applications of machine learning and statistical modeling, along with innovative methods and state-of-the-art advances, making this a timely and practical resource. This volume is ideal for statisticians, researchers, and professionals interested in pharmaceutical research and development. Readers should be familiar with the fundamentals of statistics and clinical trials.
Categories: Medical