Design And Analysis Of Non Inferiority Trials PDF Download
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Author | : Mark D. Rothmann |
Publisher | : CRC Press |
Total Pages | : 451 |
Release | : 2016-04-19 |
Genre | : Mathematics |
ISBN | : 1584888059 |
Download Design and Analysis of Non-Inferiority Trials Book in PDF, ePub and Kindle
The increased use of non-inferiority analysis has been accompanied by a proliferation of research on the design and analysis of non-inferiority studies. Using examples from real clinical trials, Design and Analysis of Non-Inferiority Trials brings together this body of research and confronts the issues involved in the design of a non-inferiority tr
Author | : Bernard Ravina |
Publisher | : Cambridge University Press |
Total Pages | : 387 |
Release | : 2012-04-12 |
Genre | : Medical |
ISBN | : 1107376572 |
Download Clinical Trials in Neurology Book in PDF, ePub and Kindle
Translating laboratory discoveries into successful therapeutics can be difficult. Clinical Trials in Neurology aims to improve the efficiency of clinical trials and the development of interventions in order to enhance the development of new treatments for neurologic diseases. It introduces the reader to the key concepts underpinning trials in the neurosciences. This volume tackles the challenges of developing therapies for neurologic disorders from measurement of agents in the nervous system to the progression of clinical signs and symptoms through illustrating specific study designs and their applications to different therapeutic areas. Clinical Trials in Neurology covers key issues in Phase I, II and III clinical trials, as well as post-marketing safety surveillance. Topics addressed include regulatory and implementation issues, outcome measures and common problems in drug development. Written by a multidisciplinary team, this comprehensive guide is essential reading for neurologists, psychiatrists, neurosurgeons, neuroscientists, statisticians and clinical researchers in the pharmaceutical industry.
Author | : Institute of Medicine |
Publisher | : National Academies Press |
Total Pages | : 221 |
Release | : 2001-01-01 |
Genre | : Medical |
ISBN | : 0309171148 |
Download Small Clinical Trials Book in PDF, ePub and Kindle
Clinical trials are used to elucidate the most appropriate preventive, diagnostic, or treatment options for individuals with a given medical condition. Perhaps the most essential feature of a clinical trial is that it aims to use results based on a limited sample of research participants to see if the intervention is safe and effective or if it is comparable to a comparison treatment. Sample size is a crucial component of any clinical trial. A trial with a small number of research participants is more prone to variability and carries a considerable risk of failing to demonstrate the effectiveness of a given intervention when one really is present. This may occur in phase I (safety and pharmacologic profiles), II (pilot efficacy evaluation), and III (extensive assessment of safety and efficacy) trials. Although phase I and II studies may have smaller sample sizes, they usually have adequate statistical power, which is the committee's definition of a "large" trial. Sometimes a trial with eight participants may have adequate statistical power, statistical power being the probability of rejecting the null hypothesis when the hypothesis is false. Small Clinical Trials assesses the current methodologies and the appropriate situations for the conduct of clinical trials with small sample sizes. This report assesses the published literature on various strategies such as (1) meta-analysis to combine disparate information from several studies including Bayesian techniques as in the confidence profile method and (2) other alternatives such as assessing therapeutic results in a single treated population (e.g., astronauts) by sequentially measuring whether the intervention is falling above or below a preestablished probability outcome range and meeting predesigned specifications as opposed to incremental improvement.
Author | : Yulia Sidi |
Publisher | : |
Total Pages | : 0 |
Release | : 2020 |
Genre | : |
ISBN | : |
Download New Approaches To The Design And Analysis Of Non-Inferiority Clinical Trials Book in PDF, ePub and Kindle
Clinical trials are an essential part of the drug development life cycle. There are different types of clinical trials, and in this dissertation, we focus on non-inferiority (NI) trials. In NI trials the goal is to show that the effectiveness of a new treatment is not considerably worse than of a standard one by an acceptable margin. Although, the new treatment could be slightly less efficacious, it can offer other benefits such as less severe adverse reactions. Several methodological challenges have been reported regarding the design, analysis and interpretation of NI trials. These include incomplete data analysis, specification of an acceptable margin, and overall benefit of the new non-inferior treatment. Therefore, the aim of this dissertation was to address each of these challenges and provide practical solutions for researchers involved with NI trials. First, we focus on incomplete data. Specifically, we evaluate how different statistical strategies perform under several NI scenarios and various types of missingness. We provide a set of recommendations for practitioners to use when confronted with incomplete data to avoid false non-inferiority conclusions. Second, while performing a thorough investigation of proper statistical strategies for incomplete data analysis, we discovered that combination rules of multiply imputed data when inference is done using a Newcombe's method did not exist. As a result, we developed these combination rules. Third, we proposed a new framework that allows for a transparent and objective justification of an acceptable margin. The framework is based on combining results of NI study and clinical experts survey data using multiple imputation (MI). Fourth, we developed a new approach for a comprehensive benefit-risk assessment of a non-inferior treatment. We focus on preference elicitation regarding benefits and risks from a small sample of NI trial participants, and use MI to restore preferences of all study participants. This dissertation provides an important contribution to the field of Statistics, and drug development. The novel methods and techniques outlined in this dissertation facilitate practitioners involved with NI trails to make more efficient and transparent evaluations of treatment effectiveness.
Author | : Stefan Wellek |
Publisher | : CRC Press |
Total Pages | : 431 |
Release | : 2010-06-24 |
Genre | : Mathematics |
ISBN | : 9781439808191 |
Download Testing Statistical Hypotheses of Equivalence and Noninferiority Book in PDF, ePub and Kindle
While continuing to focus on methods of testing for two-sided equivalence, Testing Statistical Hypotheses of Equivalence and Noninferiority, Second Edition gives much more attention to noninferiority testing. It covers a spectrum of equivalence testing problems of both types, ranging from a one-sample problem with normally distributed observations
Author | : Stefan Wellek |
Publisher | : CRC Press |
Total Pages | : 301 |
Release | : 2002-11-12 |
Genre | : Mathematics |
ISBN | : 1420035967 |
Download Testing Statistical Hypotheses of Equivalence Book in PDF, ePub and Kindle
Equivalence testing has grown significantly in importance over the last two decades, especially as its relevance to a variety of applications has become understood. Yet published work on the general methodology remains scattered in specialists' journals, and for the most part, it focuses on the relatively narrow topic of bioequivalence assessment.
Author | : Steven A. Julious |
Publisher | : CRC Press |
Total Pages | : 330 |
Release | : 2009-08-26 |
Genre | : Mathematics |
ISBN | : 1584887400 |
Download Sample Sizes for Clinical Trials Book in PDF, ePub and Kindle
Drawing on various real-world applications, Sample Sizes for Clinical Trials takes readers through the process of calculating sample sizes for many types of clinical trials. It provides descriptions of the calculations with a practical emphasis.Focusing on normal, binary, ordinal, and survival data, the book explores a range of trials, including su
Author | : Tie-Hua Ng |
Publisher | : CRC Press |
Total Pages | : 212 |
Release | : 2014-12-01 |
Genre | : Mathematics |
ISBN | : 1466561491 |
Download Noninferiority Testing in Clinical Trials Book in PDF, ePub and Kindle
Take Your NI Trial to the Next Level Reflecting the vast research on noninferiority (NI) designs from the past 15 years, Noninferiority Testing in Clinical Trials: Issues and Challenges explains how to choose the NI margin as a small fraction of the therapeutic effect of the active control in a clinical trial. Requiring no prior knowledge of NI testing, the book is easily accessible to both statisticians and nonstatisticians involved in drug development. With over 20 years of experience in this area, the author introduces the basic elements of the NI trials one at a time in a logical order. He discusses issues with estimating the effect size based on historical placebo control trials of the active control. The book covers fundamental concepts related to NI trials, such as assay sensitivity, constancy assumption, discounting, and preservation. It also describes patient populations, three-arm trials, and the equivalence of three or more groups.
Author | : Shein-Chung Chow |
Publisher | : CRC Press |
Total Pages | : 368 |
Release | : 2011-12-01 |
Genre | : Mathematics |
ISBN | : 1439839883 |
Download Adaptive Design Methods in Clinical Trials Book in PDF, ePub and Kindle
With new statistical and scientific issues arising in adaptive clinical trial design, including the U.S. FDA's recent draft guidance, a new edition of one of the first books on the topic is needed. Adaptive Design Methods in Clinical Trials, Second Edition reflects recent developments and regulatory positions on the use of adaptive designs in clini
Author | : Peter G. Smith |
Publisher | : |
Total Pages | : 479 |
Release | : 2015 |
Genre | : Health & Fitness |
ISBN | : 0198732864 |
Download Field Trials of Health Interventions Book in PDF, ePub and Kindle
This is an open access title available under the terms of a CC BY-NC 4.0 International licence. It is free to read at Oxford Scholarship Online and offered as a free PDF download from OUP and selected open access locations. Before new interventions are released into disease control programmes, it is essential that they are carefully evaluated in field trials'. These may be complex and expensive undertakings, requiring the follow-up of hundreds, or thousands, of individuals, often for long periods. Descriptions of the detailed procedures and methods used in the trials that have been conducted have rarely been published. A consequence of this, individuals planning such trials have few guidelines available and little access to knowledge accumulated previously, other than their own. In this manual, practical issues in trial design and conduct are discussed fully and in sufficient detail, that Field Trials of Health Interventions may be used as a toolbox' by field investigators. It has been compiled by an international group of over 30 authors with direct experience in the design, conduct, and analysis of field trials in low and middle income countries and is based on their accumulated knowledge and experience. Available as an open access book via Oxford Medicine Online, this new edition is a comprehensive revision, incorporating the new developments that have taken place in recent years with respect to trials, including seven new chapters on subjects ranging from trial governance, and preliminary studies to pilot testing.