Fundamentals of Clinical Trials

by Lawrence M. Friedman, David L. DeMets, Curt D. Furberg

The randomized control clinical trial has become the gold standard scientific method for the evaluation of pharmaceuticals, biologics, devices, procedures and diagnostic tests. This trial design has been successfully used in both therapeutic and disease prevention trials. It is superior to alternative designs by eliminating several sources of bias which exist in those designs. This role has evolved over the past three decades in a number of disease areas including cardiology, opthalmology, cancer and AIDS. While the specifics of using the randomized control design for a specific intervention and disease may differ, the basic fundamentals still apply in developing the study protocol and operational procedures. These fundamentals still apply in developing the study protocol and operational procedures. These fundamentals include identifying the specific questions to be tested and appropriate outcome measures, determining an adequate sample size, specifying the randomization procedure, detailing the intervention with visit schedules for subject evaluation, establishing an interim data and safety monitoring plan, detailing the final analysis plan and determining the organizational structure.This text is structured to address the fundamentals as the protocol for a clinical trial is being developed. A chapter is devoted to each of the critical areas of a protocol to aid the clinical trial researcher. The fundamentals described in this text are based on sound scientific methodology, statistical principles and years of accumulated experience by the three authors. Collectively, the authors have been active researchers in a broad area of clinical trials including cardiology, cancer, opthalmology, diabetes, osteoporosis, AIDS, women's health and screening tests. In these studies, the authors have served as members of the steering committee responsible for developing the protocol and as members of data and safety monitoring committees. The fundamentals were proposed in the first edition published in 1981 and have not changed substantially in the later editions. However, the number of examples illustrating the fundamentals has greatly expanded base on the collective experience of the authors.This text is intended for the clinical researcher who is interested in designing a clinical trial and developing a protocol. It is also of value to researchers and practitioners who must critically evaluate the literature of published clinical trials and assess the merits of each trial and the implications for the care and treatment of patients. The test uses numerous examples of published clinical trials from a variety of medical disciples to meaningfully illustrate the fundamentals. Technical design issues such as sample size are considered but the technical details have been suppressed as much as possible through the use of graphs and tables. While the technical material has been kept to a minimum, the statistician may still find the principles and fundamentals presented in this text useful both in a consulting and teaching capacity.The text assumes that the readers have only a modest formal statistical background. A basic introductory statistics course is helpful in maximizing the benefit of the text. However, a researcher or practitioner with no statistical background would still find most, if not all the chapters understandable and useful.

Genres: Textbooks, Health Care, Psychology, Reference

361 pages, Paperback

First published November 30, 1981

About the author

Professor of law.

Also author of mystery novels, The Frank May Chronicles.

Reviews

[Scott Pearson · Rating 5 out of 5]

In the past century, clinical research has grown dramatically as the number of questions about healthcare has increased. The current paradigm of evidence-based medicine describes the practice of prescribing medicines based on their tested strength instead of an individual’s limited experience. Of course, to make such judgments, a rich bed of research evidence needs to exist, and this book succinctly describes how such questions are investigated for eventual dissemination.

This book’s language is succinct and clearly intended for time-strapped researchers & physicians. It does not expand on or illustrate many of the concepts but presumes a high level of clinical knowledge among its readers. The audiobook version, which I listened to, is also abridged and does not contain contents of the full edition.

That said, it offers a comprehensive introduction to the topic. Indeed, I wonder whether re-listening to it might provide me with some benefits because I’m sure I missed a lot in its dense presentation. Even considered are newer topics like research designs besides a randomized clinical trial (the gold standard). The audiobook format provides a proper way to listen to-and-from work or during meal breaks.

[Mary Jean · Rating 4 out of 5]

Good first book on understanding clinical trial vocabulary, background, planning, and regulatory requirements. Will keep as reference.

[Monika]

PQHS 450

[Mike · Rating 4 out of 5]

A must-have for anyone just starting out in clinical trials. Clearly written and full of references for further exploration.

[Zhifei Ge · Rating 4 out of 5]

A well structured introduction to clinical trials. Something to improve is better examples of explaining key concepts.