100 Key Clinical Trials

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.

Data sharing can accelerate new discoveries by avoiding duplicative trials, stimulating new ideas for research, and enabling the maximal scientific knowledge and benefits to be gained from the efforts of clinical trial participants and investigators. At the same time, sharing clinical trial data presents risks, burdens, and challenges. These include the need to protect the privacy and honor the consent of clinical trial participants; safeguard the legitimate economic interests of sponsors; and guard against invalid secondary analyses, which could undermine trust in clinical trials or otherwise harm public health. Sharing Clinical Trial Data presents activities and strategies for the responsible sharing of clinical trial data. With the goal of increasing scientific knowledge to lead to better therapies for patients, this book identifies guiding principles and makes recommendations to maximize the benefits and minimize risks. This report offers guidance on the types of clinical trial data available at different points in the process, the points in the process at which each type of data should be shared, methods for sharing data, what groups should have access to data, and future knowledge and infrastructure needs. Responsible sharing of clinical trial data will allow other investigators to replicate published findings and carry out additional analyses, strengthen the evidence base for regulatory and clinical decisions, and increase the scientific knowledge gained from investments by the funders of clinical trials. The recommendations of Sharing Clinical Trial Data will be useful both now and well into the future as improved sharing of data leads to a stronger evidence base for treatment. This book will be of interest to stakeholders across the spectrum of research--from funders, to researchers, to journals, to physicians, and ultimately, to patients.

A complete guide to the key statistical concepts essential for the design and construction of clinical trials As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis. Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials. Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features: Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials Over 100 contributions from leading academics, researchers, and practitioners An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology,and public health.

Using real examples from oncology trials, but keeping it simple, this concise resource explains the basic principles of medical statistics so that you can better appraise clinical trial results. Key concepts covered in this book include: • hypothesis testing • Kaplan–Meier curves and other graphic representations of data • calculating the power of a study • the stopping rules for efficacy and futility. ' Fast Facts: Medical Statistics' is aimed at all clinicians, clinical scientists, medical writers and regulatory personnel who need a better understanding of the statistical terms and methods used in the planning of studies and the analysis of clinical trial data. If you have ever wanted to know what a type I error is, how an odds ratio is calculated or what a forest plot is really all about, then this is the book for you. Contents: • Statistical inference • Analysis of time-to-event endpoints • Power and sample size • Multiplicity • Interim analysis • Modeling • Graphical methods

Using examples and case studies from industry, academia and research literature, Randomized Clinical Trials provides a detailed overview of the key issues involved in designing, conducting, analysing and reporting randomized clinical trials. It examines the methodology for conducting Phase III clinical trials, developing the protocols, the practice for capturing, measuring, and analysing the resulting clinical data and their subsequent reporting. Randomized clinical trials are the principal method for determining the relative efficacy and safety of alternative treatments, interventions or medical devices. They are conducted by groups comprising one or more of pharmaceutical and allied health-care organisations, academic institutions, and charity supported research groups. In many cases such trials provide the key evidence necessary for the regulatory approval of a new product for future patient use. Randomized Clinical Trials provides comprehensive coverage of such trials, ranging from elementary to advanced level. Written by authors with considerable experience of clinical trials, Randomized Clinical Trials is an authoritative guide for clinicians, nurses, data managers and medical statisticians involved in clinical trials research and for health care professionals directly involved in patient care in a clinical trial context.

Every year, hundreds of thousands of healthy volunteers and patients worldwide undertake the journey through the maze that can be clinical trials. Research participants take part in clinical trials for a variety of reasons. The healthy volunteers may be seeking extra money to pay off college tuition, or they may know someone who is suffering and would potentially benefit from the results of the trial. The patient who is terminally ill might participate in a clinical trial simply as a last hope for a cure. Whatever the goals, though, most participants will experience the same sense of bewilderment as they encounter the jargon and medical terminology that they will hear and have to read about and understand during the course of the clinical trial. Clinical Trials: What Patients and Volunteers Need to Know demystifies the entire process, focusing on the process of drug development, and the clinical trial itself. Writing from a lifetime of experience, the author provides important questions to ask those running a clinical trial, key definitions and terms for a participant to know and understand, as well as anecdotes illustrating the clinical trial process. The author also grapples with the idea of "informed consent," providing mechanisms for patients and volunteers to feel fully informed before signing up for the trial. A vital resource for those who are considering enrolling in a clinical trial, or for the parents, friends, or relatives of those involved in a clinical trial, this book takes away the mystery and allows the participant to enter a clinical trial feeling both informed and confident.

A complete guide to the key statistical concepts essential for the design and construction of clinical trials As the newest major resource in the field of medical research, Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs presents a timely and authoritative reviewof the central statistical concepts used to build clinical trials that obtain the best results. The referenceunveils modern approaches vital to understanding, creating, and evaluating data obtained throughoutthe various stages of clinical trial design and analysis. Accessible and comprehensive, the first volume in a two-part set includes newly-written articles as well as established literature from the Wiley Encyclopedia of Clinical Trials . Illustrating a variety of statistical concepts and principles such as longitudinal data, missing data, covariates, biased-coin randomization, repeated measurements, and simple randomization, the book also provides in-depth coverage of the various trial designs found within phase I-IV trials. Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs also features: Detailed chapters on the type of trial designs, such as adaptive, crossover, group-randomized, multicenter, non-inferiority, non-randomized, open-labeled, preference, prevention, and superiority trials Over 100 contributions from leading academics, researchers, and practitioners An exploration of ongoing, cutting-edge clinical trials on early cancer and heart disease, mother-to-child human immunodeficiency virus transmission trials, and the AIDS Clinical Trials Group Methods and Applications of Statistics in Clinical Trials, Volume 1: Concepts, Principles, Trials, and Designs is an excellent reference for researchers, practitioners, and students in the fields of clinicaltrials, pharmaceutics, biostatistics, medical research design, biology, biomedicine, epidemiology, and public health.