Confidence intervals versus p-values for interpretation of clinical trial results: introduction. 1993

G W Pledger
R. W. Johnson Pharmaceutical Research Institute, Raritan, New Jersey.

The following three papers summarize the presentations at a Society for Clinical Trials annual meeting session on the relative merits of estimation versus testing for analysis of randomized clinical trials. By design, randomized clinical trials have internal validity. Whether they also possess quantitative external validity--generalizability of effect size to some population represented by the trial subjects--is one of the main points of disagreement among the three authors. It may be unrealistic to expect a resolution that applies across the wide variety of therapeutic areas and clinical trial goals. Extrapolation from clinical trial to clinical practice is often endorsed in connection with large trials having loose entry criteria and focusing on an objective, clearly meaningful clinical endpoint. By contrast, the relevance of estimates of effect size is less clear in the case of many clinical trials conducted in the course of drug development.

UI MeSH Term Description Entries
D002986 Clinical Trials as Topic Works about pre-planned studies of the safety, efficacy, or optimum dosage schedule (if appropriate) of one or more diagnostic, therapeutic, or prophylactic drugs, devices, or techniques selected according to predetermined criteria of eligibility and observed for predefined evidence of favorable and unfavorable effects. This concept includes clinical trials conducted both in the U.S. and in other countries. Clinical Trial as Topic
D003627 Data Interpretation, Statistical Application of statistical procedures to analyze specific observed or assumed facts from a particular study. Data Analysis, Statistical,Data Interpretations, Statistical,Interpretation, Statistical Data,Statistical Data Analysis,Statistical Data Interpretation,Analyses, Statistical Data,Analysis, Statistical Data,Data Analyses, Statistical,Interpretations, Statistical Data,Statistical Data Analyses,Statistical Data Interpretations
D015195 Drug Design The molecular designing of drugs for specific purposes (such as DNA-binding, enzyme inhibition, anti-cancer efficacy, etc.) based on knowledge of molecular properties such as activity of functional groups, molecular geometry, and electronic structure, and also on information cataloged on analogous molecules. Drug design is generally computer-assisted molecular modeling and does not include PHARMACOKINETICS, dosage analysis, or drug administration analysis. Computer-Aided Drug Design,Computerized Drug Design,Drug Modeling,Pharmaceutical Design,Computer Aided Drug Design,Computer-Aided Drug Designs,Computerized Drug Designs,Design, Pharmaceutical,Drug Design, Computer-Aided,Drug Design, Computerized,Drug Designs,Drug Modelings,Pharmaceutical Designs
D016001 Confidence Intervals A range of values for a variable of interest, e.g., a rate, constructed so that this range has a specified probability of including the true value of the variable. Confidence Interval,Interval, Confidence,Intervals, Confidence
D016032 Randomized Controlled Trials as Topic Works about clinical trials that involve at least one test treatment and one control treatment, concurrent enrollment and follow-up of the test- and control-treated groups, and in which the treatments to be administered are selected by a random process, such as the use of a random-numbers table. Clinical Trials, Randomized,Controlled Clinical Trials, Randomized,Trials, Randomized Clinical

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