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Reproducibility probability in clinical trials
Author(s) -
Shao Jun,
Chow SheinChung
Publication year - 2002
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.1177
Subject(s) - clinical trial , protocol (science) , food and drug administration , medical physics , medicine , sample size determination , bayesian probability , clinical research , clinical study design , reproducibility , randomized controlled trial , drug development , risk analysis (engineering) , computer science , drug , statistics , alternative medicine , pharmacology , artificial intelligence , surgery , mathematics , pathology
For marketing approval of a new drug product, the United States Food and Drug Administration (FDA) requires that substantial evidence of the effectiveness of the drug product be provided through the conduct of at least two adequate and well‐controlled clinical trials. The purpose of conducting the second clinical trial is to study whether the clinical result from the first trial is reproducible in the second trial with the same study protocol. Under certain circumstance, the FDA Modernization Act of 1997 includes a provision to allow data from one adequate and well‐controlled clinical trial investigation and confirmatory evidence to establish effectiveness for risk/benefit assessment of drug and biological candidates for approval. In this paper, we introduce the concept of reproducibility probability for a given clinical trial, which is useful in providing important information for regulatory agencies in deciding whether a single clinical trial is sufficient and for pharmaceutical companies in adjusting the sample size in a future clinical trial. Three approaches, the estimated power approach, the method of confidence bounds and the Bayesian approach, are studied in evaluating reproducibility probabilities under several study designs commonly used in clinical trials. Copyright © 2002 John Wiley & Sons, Ltd.