Estimands Estimators And Sensitivity Analysis In Clinical Trials
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Estimands Estimators And Sensitivity Analysis In Clinical Trials
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Author : Craig Mallinckrodt
language : en
Publisher: CRC Press
Release Date : 2019-12-23
Estimands Estimators And Sensitivity Analysis In Clinical Trials written by Craig Mallinckrodt and has been published by CRC Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2019-12-23 with Medical categories.
The concepts of estimands, analyses (estimators), and sensitivity are interrelated. Therefore, great need exists for an integrated approach to these topics. This book acts as a practical guide to developing and implementing statistical analysis plans by explaining fundamental concepts using accessible language, providing technical details, real-world examples, and SAS and R code to implement analyses. The updated ICH guideline raises new analytic and cross-functional challenges for statisticians. Gaps between different communities have come to surface, such as between causal inference and clinical trialists, as well as among clinicians, statisticians, and regulators when it comes to communicating decision-making objectives, assumptions, and interpretations of evidence. This book lays out a path toward bridging some of these gaps. It offers A common language and unifying framework along with the technical details and practical guidance to help statisticians meet the challenges A thorough treatment of intercurrent events (ICEs), i.e., postrandomization events that confound interpretation of outcomes and five strategies for ICEs in ICH E9 (R1) Details on how estimands, integrated into a principled study development process, lay a foundation for coherent specification of trial design, conduct, and analysis needed to overcome the issues caused by ICEs: A perspective on the role of the intention-to-treat principle Examples and case studies from various areas Example code in SAS and R A connection with causal inference Implications and methods for analysis of longitudinal trials with missing data Together, the authors have offered the readers their ample expertise in clinical trial design and analysis, from an industrial and academic perspective.
A Sensitivity Analysis For Clinical Trials With Informatively Censored Survival Endpoints
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Author : Eric Norbert Meier
language : en
Publisher:
Release Date : 2012
A Sensitivity Analysis For Clinical Trials With Informatively Censored Survival Endpoints written by Eric Norbert Meier and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2012 with categories.
Analyses of clinical trials with time-to-event endpoints typically employ the assumption of non-informative censoring. While this assumption is usually appropriate for end-of-study (EOS) censoring, its applicability to lost-to-follow-up (LTFU) censoring is often suspect and may result in biased estimates of the treatment effect. To assess the robustness of estimates to departures from non-informative censoring, authors have proposed sensitivity analyses that assume a semiparametric model for the censoring mechanism, with the parameters representing associations between censoring and increased or decreased rates of survival. The parameters are varied over a plausible range resulting in a corresponding range of estimates for the treatment effect. We consider such an approach for two-arm trials in which the sensitivity parameters represent hazard ratios within a proportional hazards model with a time varying covariate comparing subjects who have been lost to follow-up to all other subjects. Using hypothesized hazard ratios for each arm separately, we multiply impute the unobserved data as it might have been observed in the absence of informative censoring. The treatment effect estimates computed using the imputed data are then summarized in a graphical display. Of particular interest in this research is the robustness of our approach to violations of the proportional hazards assumptions used when imputing the missing data. On the basis of extensive simulation studies, we find that the accuracy of the sensitivity analyses are relatively unaffected by departures from the semiparametric assumptions.
Clinical Trials With Missing Data
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Author : Michael O'Kelly
language : en
Publisher: John Wiley & Sons
Release Date : 2014-02-14
Clinical Trials With Missing Data written by Michael O'Kelly and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2014-02-14 with Medical categories.
This book provides practical guidance for statisticians, clinicians, and researchers involved in clinical trials in the biopharmaceutical industry, medical and public health organisations. Academics and students needing an introduction to handling missing data will also find this book invaluable. The authors describe how missing data can affect the outcome and credibility of a clinical trial, show by examples how a clinical team can work to prevent missing data, and present the reader with approaches to address missing data effectively. The book is illustrated throughout with realistic case studies and worked examples, and presents clear and concise guidelines to enable good planning for missing data. The authors show how to handle missing data in a way that is transparent and easy to understand for clinicians, regulators and patients. New developments are presented to improve the choice and implementation of primary and sensitivity analyses for missing data. Many SAS code examples are included – the reader is given a toolbox for implementing analyses under a variety of assumptions.
Sensitivity Analysis For Non Ignorable Dropout Of Marginal Treatment Effect In Longitudinal Trials For G Computation Based Estimators
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Author : Emin Tahirovic
language : en
Publisher:
Release Date : 2016
Sensitivity Analysis For Non Ignorable Dropout Of Marginal Treatment Effect In Longitudinal Trials For G Computation Based Estimators written by Emin Tahirovic and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2016 with categories.
We specify identifying assumptions under which linear increments (LI) estimator can be used to estimate unconditional expectation for longitudinal data from a clinical trial in the presence of dropout. We show that these are analog conditions under which extended linear SWEEP estimator achieves unbiased estimation of the identical parameter in the same setting. Within a class of linear autoregressive models we specify how strategies implemented in LI and extended SWEEP relate to each other w.r.t. the conditional expectation of increments and outcomes respectively. We utilize conceptual overlap of these two methods to define a sensitivity analysis for both of them in presence of non-ignorable dropout. Interdependency of these two approaches offers a natural solution to a prominent problem of asynchronous association between outcome and dropout inevitably encountered in sensitivity analysis for dropout in longitudinal data. Validation of our approach is done on the data coming from a randomized, longitudinal trial of behavioral economic interventions to reduce CVD risk. We subsequently show that our approach to sensitivity analysis can be perceived as extension of the pattern mixture method defined by Daniels and Hogan in 2007. to longer sequences of observations. For T=3 we give the explicit expression for bias of our approach w.r.t. mentioned pattern mixture approach. We further show on a subset of the data from the same study that this bias does not invalidate our sensitivity analysis for LI when it comes to evaluating the robustness of findings under increasingly less ignorable dropout.
The Prevention And Treatment Of Missing Data In Clinical Trials
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Author : National Research Council
language : en
Publisher: National Academies Press
Release Date : 2010-12-21
The Prevention And Treatment Of Missing Data In Clinical Trials written by National Research Council and has been published by National Academies Press this book supported file pdf, txt, epub, kindle and other format this book has been release on 2010-12-21 with Medical categories.
Randomized clinical trials are the primary tool for evaluating new medical interventions. Randomization provides for a fair comparison between treatment and control groups, balancing out, on average, distributions of known and unknown factors among the participants. Unfortunately, these studies often lack a substantial percentage of data. This missing data reduces the benefit provided by the randomization and introduces potential biases in the comparison of the treatment groups. Missing data can arise for a variety of reasons, including the inability or unwillingness of participants to meet appointments for evaluation. And in some studies, some or all of data collection ceases when participants discontinue study treatment. Existing guidelines for the design and conduct of clinical trials, and the analysis of the resulting data, provide only limited advice on how to handle missing data. Thus, approaches to the analysis of data with an appreciable amount of missing values tend to be ad hoc and variable. The Prevention and Treatment of Missing Data in Clinical Trials concludes that a more principled approach to design and analysis in the presence of missing data is both needed and possible. Such an approach needs to focus on two critical elements: (1) careful design and conduct to limit the amount and impact of missing data and (2) analysis that makes full use of information on all randomized participants and is based on careful attention to the assumptions about the nature of the missing data underlying estimates of treatment effects. In addition to the highest priority recommendations, the book offers more detailed recommendations on the conduct of clinical trials and techniques for analysis of trial data.
Statistical Thinking For Non Statisticians In Drug Regulation
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Author : Richard Kay
language : en
Publisher: John Wiley & Sons
Release Date : 2022-11-29
Statistical Thinking For Non Statisticians In Drug Regulation written by Richard Kay and has been published by John Wiley & Sons this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-11-29 with Medical categories.
STATISTICAL THINKING FOR NON-STATISTICIANS IN DRUG REGULATION Statistical methods in the pharmaceutical industry are accepted as a key element in the design and analysis of clinical studies. Increasingly, the medical and scientific community are aligning with the regulatory authorities and recognizing that correct statistical methodology is essential as the basis for valid conclusions. In order for those correct and robust methods to be successfully employed there needs to be effective communication across disciplines at all stages of the planning, conducting, analyzing and reporting of clinical studies associated with the development and evaluation of new drugs and devices. Statistical Thinking for Non-Statisticians in Drug Regulation provides a comprehensive in-depth guide to statistical methodology for pharmaceutical industry professionals, including physicians, investigators, medical science liaisons, clinical research scientists, medical writers, regulatory personnel, statistical programmers, senior data managers and those working in pharmacovigilance. The author’s years of experience and up-to-date familiarity with pharmaceutical regulations and statistical practice within the wider clinical community make this an essential guide for the those working in and with the industry. The third edition of Statistical Thinking for Non-Statisticians in Drug Regulation includes: A detailed new chapter on Estimands in line with the 2019 Addendum to ICH E9 Major new sections on topics including Combining Hierarchical Testing and Alpha Adjustment, Biosimilars, Restricted Mean Survival Time, Composite Endpoints and Cumulative Incidence Functions, Adjusting for Cross-Over in Oncology, Inverse Propensity Score Weighting, and Network Meta-Analysis Updated coverage of many existing topics to reflect new and revised guidance from regulatory authorities and author experience Statistical Thinking for Non-Statisticians in Drug Regulation is a valuable guide for pharmaceutical and medical device industry professionals, as well as statisticians joining the pharmaceutical industry and students and teachers of drug development.
Principles And Practice Of Clinical Trials
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Author : Steven Piantadosi
language : en
Publisher: Springer Nature
Release Date : 2022-07-19
Principles And Practice Of Clinical Trials written by Steven Piantadosi and has been published by Springer Nature this book supported file pdf, txt, epub, kindle and other format this book has been release on 2022-07-19 with Medical categories.
This is a comprehensive major reference work for our SpringerReference program covering clinical trials. Although the core of the Work will focus on the design, analysis, and interpretation of scientific data from clinical trials, a broad spectrum of clinical trial application areas will be covered in detail. This is an important time to develop such a Work, as drug safety and efficacy emphasizes the Clinical Trials process. Because of an immense and growing international disease burden, pharmaceutical and biotechnology companies continue to develop new drugs. Clinical trials have also become extremely globalized in the past 15 years, with over 225,000 international trials ongoing at this point in time. Principles in Practice of Clinical Trials is truly an interdisciplinary that will be divided into the following areas: 1) Clinical Trials Basic Perspectives 2) Regulation and Oversight 3) Basic Trial Designs 4) Advanced Trial Designs 5) Analysis 6) Trial Publication 7) Topics Related Specific Populations and Legal Aspects of Clinical Trials The Work is designed to be comprised of 175 chapters and approximately 2500 pages. The Work will be oriented like many of our SpringerReference Handbooks, presenting detailed and comprehensive expository chapters on broad subjects. The Editors are major figures in the field of clinical trials, and both have written textbooks on the topic. There will also be a slate of 7-8 renowned associate editors that will edit individual sections of the Reference.
Report Of The Technical Consultation On Innovative Clinical Trial Designs For Development Of New Tb Treatments
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Author :
language : en
Publisher: World Health Organization
Release Date : 2021-07-28
Report Of The Technical Consultation On Innovative Clinical Trial Designs For Development Of New Tb Treatments written by and has been published by World Health Organization this book supported file pdf, txt, epub, kindle and other format this book has been release on 2021-07-28 with Medical categories.
The Global Tuberculosis Programme of the World Health Organization (WHO) convened a virtual technical consultation on “Innovative Clinical Trial Designs for Development of New TB Treatments” on 5 days between 20 August and 13 October 2021. The main objective of the meeting was to establish evidence-based approaches to trial designs and use of data to inform policy guidance of new regimens for the treatment of TB with the view to accelerate future regimen development. The consultation brought together researchers, academics, technical partners, TB drugs and regimens developers including pharmaceutical industry, trialists, regulators, guideline developers, programme managers, nongovernmental organizations and civil society.
Missing Data In Clinical Studies
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Author : Geert Molenberghs
language : en
Publisher: Wiley
Release Date : 2007-04-16
Missing Data In Clinical Studies written by Geert Molenberghs and has been published by Wiley this book supported file pdf, txt, epub, kindle and other format this book has been release on 2007-04-16 with Medical categories.
Missing Data in Clinical Studies provides a comprehensive account of the problems arising when data from clinical and related studies are incomplete, and presents the reader with approaches to effectively address them. The text provides a critique of conventional and simple methods before moving on to discuss more advanced approaches. The authors focus on practical and modeling concepts, providing an extensive set of case studies to illustrate the problems described. Provides a practical guide to the analysis of clinical trials and related studies with missing data. Examines the problems caused by missing data, enabling a complete understanding of how to overcome them. Presents conventional, simple methods to tackle these problems, before addressing more advanced approaches, including sensitivity analysis, and the MAR missingness mechanism. Illustrated throughout with real-life case studies and worked examples from clinical trials. Details the use and implementation of the necessary statistical software, primarily SAS. Missing Data in Clinical Studies has been developed through a series of courses and lectures. Its practical approach will appeal to applied statisticians and biomedical researchers, in particular those in the biopharmaceutical industry, medical and public health organisations. Graduate students of biostatistics will also find much of benefit.
Statistical Issues In The Design And Analysis Of Clinical Trials
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Author : Yanning Liu
language : en
Publisher:
Release Date : 2017-01-11
Statistical Issues In The Design And Analysis Of Clinical Trials written by Yanning Liu and has been published by this book supported file pdf, txt, epub, kindle and other format this book has been release on 2017-01-11 with categories.
