Monitoring of clinical trials: Interim monitoring, data monitoring committees, and group sequential methods

Rickey E. Carter, Robert F. Woolson

Research output: Chapter in Book/Report/Conference proceedingChapter

Abstract

Introduction The aging population is increasing the global burden of neurological diseases and the need for safe and effective therapeutics for these disorders. While therapeutic targets for neurological disorders are increasingly tractable, neurology also has one of the highest failure rates in late stage clinical trials. There is an increasing need for proficiency in the design, conduct, analysis, and interpretation of clinical trials in neurology. This is especially true in the early and middle stages of therapeutic development, which determine if and how comparative efficacy studies should be conducted. The goal of this book is to describe how the principles of clinical trials can be applied to the challenges that arise in developing therapies for neurological disorders. The fundamentals of clinical trials are explored in several existing texts and are the same across different fields of medicine. Here we describe the application of those principles to the specific clinical questions that arise with the study of neurological diseases. There is no one trial design that meets all objectives for a particular phase of development. Rather there are parameters that need to be optimized for each intervention, question, and study. A clinical trial can be defined as an experiment in humans that is designed to test a medical, surgical, behavioral, or other type of intervention. This definition does not presuppose a particular design, type of control group, or analysis plan. When designing a trial and consulting this text for guidance, the reader should carefully consider the clinical question they are facing and how that question fits in the overall program of research for the intervention. The next step is to select a design that can practically and efficiently answer the question and guide decision-making about the intervention and the steps to further develop it.

Original languageEnglish (US)
Title of host publicationClinical Trials in Neurology: Design, Conduct, Analysis
PublisherCambridge University Press
Pages147-159
Number of pages13
ISBN (Print)9781139032445, 9780521762595
DOIs
StatePublished - Jan 1 2012

Fingerprint

Clinical Trials Data Monitoring Committees
Clinical Trials
Neurology
Nervous System Diseases
Therapeutics
Decision Making
Medicine
Control Groups
Research
Population

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Carter, R. E., & Woolson, R. F. (2012). Monitoring of clinical trials: Interim monitoring, data monitoring committees, and group sequential methods. In Clinical Trials in Neurology: Design, Conduct, Analysis (pp. 147-159). Cambridge University Press. https://doi.org/10.1017/CBO9781139032445.015

Monitoring of clinical trials : Interim monitoring, data monitoring committees, and group sequential methods. / Carter, Rickey E.; Woolson, Robert F.

Clinical Trials in Neurology: Design, Conduct, Analysis. Cambridge University Press, 2012. p. 147-159.

Research output: Chapter in Book/Report/Conference proceedingChapter

Carter, RE & Woolson, RF 2012, Monitoring of clinical trials: Interim monitoring, data monitoring committees, and group sequential methods. in Clinical Trials in Neurology: Design, Conduct, Analysis. Cambridge University Press, pp. 147-159. https://doi.org/10.1017/CBO9781139032445.015
Carter RE, Woolson RF. Monitoring of clinical trials: Interim monitoring, data monitoring committees, and group sequential methods. In Clinical Trials in Neurology: Design, Conduct, Analysis. Cambridge University Press. 2012. p. 147-159 https://doi.org/10.1017/CBO9781139032445.015
Carter, Rickey E. ; Woolson, Robert F. / Monitoring of clinical trials : Interim monitoring, data monitoring committees, and group sequential methods. Clinical Trials in Neurology: Design, Conduct, Analysis. Cambridge University Press, 2012. pp. 147-159
@inbook{9e43ea81fca94ac2be4e55af176abc8c,
title = "Monitoring of clinical trials: Interim monitoring, data monitoring committees, and group sequential methods",
abstract = "Introduction The aging population is increasing the global burden of neurological diseases and the need for safe and effective therapeutics for these disorders. While therapeutic targets for neurological disorders are increasingly tractable, neurology also has one of the highest failure rates in late stage clinical trials. There is an increasing need for proficiency in the design, conduct, analysis, and interpretation of clinical trials in neurology. This is especially true in the early and middle stages of therapeutic development, which determine if and how comparative efficacy studies should be conducted. The goal of this book is to describe how the principles of clinical trials can be applied to the challenges that arise in developing therapies for neurological disorders. The fundamentals of clinical trials are explored in several existing texts and are the same across different fields of medicine. Here we describe the application of those principles to the specific clinical questions that arise with the study of neurological diseases. There is no one trial design that meets all objectives for a particular phase of development. Rather there are parameters that need to be optimized for each intervention, question, and study. A clinical trial can be defined as an experiment in humans that is designed to test a medical, surgical, behavioral, or other type of intervention. This definition does not presuppose a particular design, type of control group, or analysis plan. When designing a trial and consulting this text for guidance, the reader should carefully consider the clinical question they are facing and how that question fits in the overall program of research for the intervention. The next step is to select a design that can practically and efficiently answer the question and guide decision-making about the intervention and the steps to further develop it.",
author = "Carter, {Rickey E.} and Woolson, {Robert F.}",
year = "2012",
month = "1",
day = "1",
doi = "10.1017/CBO9781139032445.015",
language = "English (US)",
isbn = "9781139032445",
pages = "147--159",
booktitle = "Clinical Trials in Neurology: Design, Conduct, Analysis",
publisher = "Cambridge University Press",

}

TY - CHAP

T1 - Monitoring of clinical trials

T2 - Interim monitoring, data monitoring committees, and group sequential methods

AU - Carter, Rickey E.

AU - Woolson, Robert F.

PY - 2012/1/1

Y1 - 2012/1/1

N2 - Introduction The aging population is increasing the global burden of neurological diseases and the need for safe and effective therapeutics for these disorders. While therapeutic targets for neurological disorders are increasingly tractable, neurology also has one of the highest failure rates in late stage clinical trials. There is an increasing need for proficiency in the design, conduct, analysis, and interpretation of clinical trials in neurology. This is especially true in the early and middle stages of therapeutic development, which determine if and how comparative efficacy studies should be conducted. The goal of this book is to describe how the principles of clinical trials can be applied to the challenges that arise in developing therapies for neurological disorders. The fundamentals of clinical trials are explored in several existing texts and are the same across different fields of medicine. Here we describe the application of those principles to the specific clinical questions that arise with the study of neurological diseases. There is no one trial design that meets all objectives for a particular phase of development. Rather there are parameters that need to be optimized for each intervention, question, and study. A clinical trial can be defined as an experiment in humans that is designed to test a medical, surgical, behavioral, or other type of intervention. This definition does not presuppose a particular design, type of control group, or analysis plan. When designing a trial and consulting this text for guidance, the reader should carefully consider the clinical question they are facing and how that question fits in the overall program of research for the intervention. The next step is to select a design that can practically and efficiently answer the question and guide decision-making about the intervention and the steps to further develop it.

AB - Introduction The aging population is increasing the global burden of neurological diseases and the need for safe and effective therapeutics for these disorders. While therapeutic targets for neurological disorders are increasingly tractable, neurology also has one of the highest failure rates in late stage clinical trials. There is an increasing need for proficiency in the design, conduct, analysis, and interpretation of clinical trials in neurology. This is especially true in the early and middle stages of therapeutic development, which determine if and how comparative efficacy studies should be conducted. The goal of this book is to describe how the principles of clinical trials can be applied to the challenges that arise in developing therapies for neurological disorders. The fundamentals of clinical trials are explored in several existing texts and are the same across different fields of medicine. Here we describe the application of those principles to the specific clinical questions that arise with the study of neurological diseases. There is no one trial design that meets all objectives for a particular phase of development. Rather there are parameters that need to be optimized for each intervention, question, and study. A clinical trial can be defined as an experiment in humans that is designed to test a medical, surgical, behavioral, or other type of intervention. This definition does not presuppose a particular design, type of control group, or analysis plan. When designing a trial and consulting this text for guidance, the reader should carefully consider the clinical question they are facing and how that question fits in the overall program of research for the intervention. The next step is to select a design that can practically and efficiently answer the question and guide decision-making about the intervention and the steps to further develop it.

UR - http://www.scopus.com/inward/record.url?scp=84925633599&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84925633599&partnerID=8YFLogxK

U2 - 10.1017/CBO9781139032445.015

DO - 10.1017/CBO9781139032445.015

M3 - Chapter

AN - SCOPUS:84925633599

SN - 9781139032445

SN - 9780521762595

SP - 147

EP - 159

BT - Clinical Trials in Neurology: Design, Conduct, Analysis

PB - Cambridge University Press

ER -