Designing Clinical Research An Epidemiologic Approach

by Hulley, Stephen B.; Cummings, Steven R.; Browner, Warren S.; Grady, Deborah; Hearst, Norman; Newman, Thomas B.

• ISBN13: 9780781722186
• ISBN10: 0781722187
• Edition: 2nd
• Format: Paperback
• Copyright: 2000-10-30
• Publisher: LWW

Summary

Designing Clinical Research has set the standard for providing a practical guide to planning, tabulating, formulating, and implementing clinical research, with an easy to read, uncomplicated presentation. Updated chapters incorporate current research methodology, with an updated syllabus for conducting a clinical research workshop. A Brandon-Hill recommended title.

Table of Contents

Introduction	xii
Acknowledgments	xv
Section I: Basic Ingredients
Getting Started: The Anatomy and Physiology of Clinical Research	3	(14)
Stephen B. Hulley
Thomas B. Newman
Steven R. Cummings
The Anatomy of Research: What It's Made of	3	(4)
The Physiology of Research: How It Works	7	(5)
Designing the Study	12	(1)
Summary	13	(1)
Exercises	14	(1)
Appendix: Study Outline	15	(2)
Conceiving the Research Question	17	(8)
Steven R. Cummings
Warren S. Browner
Stephen B. Hulley
Origins of a Research Question	17	(2)
Characteristics of a Good Research Question	19	(2)
Developing the Research Question and Study Plan	21	(1)
Summary	22	(1)
Exercises	23	(2)
Choosing the Study Subjects: Specification, Sampling, and Recruitment	25	(12)
Stephen B. Hulley
Thomas B. Newman
Steven R. Cummings
Basic Terms and Concepts	25	(2)
Selection Criteria	27	(3)
Sampling	30	(1)
Recruitment	31	(2)
Summary	33	(1)
Exercises	33	(2)
Appendix: Drawing a Random Sample	35	(2)
Planning the Measurements: Precision and Accuracy	37	(14)
Stephen B. Hulley
Jeffrey N. Martin
Steven R. Cummings
Measurement Scales	37	(2)
Precision	39	(2)
Accuracy	41	(4)
Other Features of Measurement Approaches	45	(1)
Measurements on Stored Materials	46	(1)
In Closing	47	(1)
Summary	47	(1)
Exercises	48	(1)
Appendix: Operationally Defining a Measurement	49	(2)
Getting Ready to Estimate Sample Size: Hypotheses and Underlying Principles	51	(14)
Warren S. Browner
Thomas B. Newman
Norman Hearst
Stephen B. Hulley
Hypotheses	51	(3)
Underlying Statistical Principles	54	(4)
Additional Points	58	(4)
Summary	62	(1)
Exercises	62	(3)
Estimating Sample Size and Power: The Nitty-gritty	65	(30)
Warren S. Browner
Thomas B. Newman
Steven R. Cummings
Stephen B. Hulley
Sample Size Techniques for Analytic Studies and Experiments	65	(5)
Other Considerations and Special Issues	70	(3)
Sample Size Techniques for Descriptive Studies	73	(2)
What to Do When Sample Size Is Fixed	75	(1)
Strategies for Minimizing Sample Size and Maximizing Power	76	(4)
How to Estimate Sample Size When There Is Insufficient Information	80	(1)
Common Errors to Avoid	81	(1)
Summary	81	(1)
Exercises	82	(3)
Appendix: Sample Size Tables	85	(10)
Section II: Study Designs
Designing an Observational Study: Cohort Studies	95	(12)
Steven R. Cummings
Thomas B. Newman
Stephen B. Hulley
Prospective Cohort Studies	96	(1)
Retrospective Cohort Studies	97	(2)
Nested Case-Control and Case-Cohort Studies	99	(2)
Multiple-Cohort Studies and External Controls	101	(2)
Planning a Cohort Study	103	(1)
Summary	104	(1)
Exercises	105	(2)
Designing an Observational Study: Cross-sectional and Case-control Studies	107	(18)
Thomas B. Newman
Warren S. Browner
Steven R. Cummings
Stephen B. Hulley
Cross-Sectional Studies	107	(3)
Case-Control Studies	110	(7)
Choosing Among Observational Designs	117	(2)
Summary	119	(1)
Exercises	119	(2)
Appendix: Measures of Association	121	(4)
Enhancing Causal Inference in Observational Studies	125	(18)
Thomas B. Newman
Warren S. Browner
Stephen B. Hulley
Spurious Associations	125	(3)
Real Associations Other Than Cause-Effect	128	(2)
Coping with Confounders in the Design Phase	130	(3)
Coping with Confounders in the Analysis Phase	133	(2)
Choosing a Strategy	135	(1)
Summary	136	(1)
Exercises	137	(2)
Appendix: Confounding and Adjustment	139	(4)
Designing an Experiment: Clinical Trials I	143	(14)
Steven R. Cummings
Deborah Grady
Stephen B. Hulley
Selecting the Paticipants	143	(3)
Measuring Baseline Variables	146	(1)
Randomizing	147	(2)
Applying the Interventions	149	(5)
Summary	154	(3)
Designing an Experiment: Clinical Trials II	157	(18)
Deborah Grady
Steven R. Cummings
Stephen B. Hulley
Follow-up and Adherence to the Protocol	157	(3)
Measuring the Outcome	160	(2)
Analyzing the Results	162	(2)
Monitoring Clinical Trials	164	(3)
Alternatives to the Randomized Blinded Trial	167	(3)
Trials for FDA Approval of New Therapies	170	(1)
Deciding to Do a Trial	171	(1)
Summary	172	(1)
Exercises	172	(1)
Appendix: Interim Monitoring	173	(2)
Designing Studies of Medical Tests	175	(20)
Thomas B. Newman
Warren S. Browner
Steven R. Cummings
Determining Whether a Test Is Useful	175	(2)
Studies of Test Reproducibility	177	(2)
Studies of the Accuracy of Tests	179	(4)
Studies of the Effect of Test Results on Clinical Decisions	183	(1)
Studies of Feasibility, Costs, and Risks of Tests	184	(2)
Studies of the Effect of Testing on Outcomes	186	(2)
Pitfalls in the Design or Analysis of Diagnostic Test Studies	188	(1)
Summary	189	(1)
Exercises	190	(2)
Appendix: Interobserver Reproducibility	192	(3)
Research Using Existing Data: Secondary Data Analysis, Ancillary Studies, and Systematic Reviews	195	(20)
Norman Hearst
Deborah Grady
Hal V. Barron
Karla Kerlikowske
Advantages and Disadvantages	195	(1)
Secondary Data Analysis	196	(5)
Ancillary Studies	201	(2)
Systematic Reviews	203	(5)
Summary	208	(1)
Exercises	209	(2)
Appendix: Meta-Analysis Methods	211	(4)
Section III: Implementation
Addressing Ethical Issues	215	(16)
Bernard Lo
Ethical Principles	215	(1)
Federal Regulations for Research on Human Subjects	216	(4)
Responsibilities of Investigators	220	(3)
Other Ethical Issues	223	(2)
Summary	225	(1)
Exercises	225	(3)
Appendix: Informed Consent	228	(3)
Designing Questionnaires and Data Collection Instruments	231	(16)
Steven R. Cummings
Anita L. Stewart
Stephen B. Hulley
Designing Good Questions and Instruments	231	(8)
Steps in Assembling the Instruments for the Study	239	(2)
Administering the Instruments	241	(2)
Summary	243	(1)
Exercises	244	(1)
Appendix: Example of a Questionnaire	245	(2)
Data Management	247	(12)
Deborah Grady
Thomas B. Newman
Eric Vittinghoff
Defining the Variables	247	(3)
Creating the Study Database and Data Dictionary	250	(2)
Entering the Data and Correcting Errors	252	(4)
Creating a Dataset for Analysis	256	(1)
Backing Up and Archiving the Dataset	256	(1)
Summary	256	(3)
Implementing the Study: Pretesting, Quality Control, and Protocol Revisions	259	(16)
Stephen B. Hulley
Steven R. Cummings
Pretesting	259	(2)
Quality Control	261	(7)
Protocol Revisions Once Data Collection Has Begun	268	(1)
Summary	269	(1)
Exercises	270	(1)
Appendix: Operations Manual and Checklists	271	(4)
Community and International Studies	275	(10)
Norman Hearst
Stephen B. Hulley
Why Community and International Research?	275	(2)
Community Research	277	(1)
International Research	278	(4)
Summary	282	(1)
Exercises	282	(3)
Writing and Funding a Research Proposal	285	(16)
Steven R. Cummings
Elizabeth A. Holly
Stephen B. Hulley
Writing Proposals	285	(3)
Elements of a Proposal	288	(5)
Characteristics of Good Proposals	293	(1)
Finding Support for Research	294	(4)
Summary	298	(3)
Answers to Chapter Exercises	301	(18)
Subject Index	319