Document Detail


A likelihood-based, counterfactual approach to accounting for treatment failures in clinical trials.
MedLine Citation:
PMID:  12921395     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Consider a two-armed, placebo-controlled trial in which subjects may experience treatment failure. For ethical reasons, it is necessary to administer emergency or rescue medications for such subjects. However, the emergency medications may bias the set of response measurements. When analyzing the data from clinical trials, the standard approach is to perform an intent-to-treat (ITT) analysis, wherein the data are analyzed according to treatment assignment. Secondary statistical analyses that supplement the ITT analysis can be performed to account for the impact of treatment failures and emergency medications. A likelihood-based, counterfactual approach to supplemental analyses uses the expectation maximization (EM) algorithm for parameter estimation and a likelihood ratio test to test the equality of the placebo and experimental treatment means for subjects who would not fail under either treatment assignment. A simulation study is performed to assess the operating characteristics of the likelihood ratio test. An example from the Asthma Clinical Research Network (ACRN) is used to draw comparisons between the standard ITT procedure and the developed supplemental analysis.
Authors:
Michele L Shaffer; Vernon M Chinchilli
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Publication Detail:
Type:  Journal Article; Research Support, U.S. Gov't, P.H.S.    
Journal Detail:
Title:  Journal of biopharmaceutical statistics     Volume:  13     ISSN:  1054-3406     ISO Abbreviation:  J Biopharm Stat     Publication Date:  2003 Aug 
Date Detail:
Created Date:  2003-08-18     Completed Date:  2004-03-09     Revised Date:  2007-11-15    
Medline Journal Info:
Nlm Unique ID:  9200436     Medline TA:  J Biopharm Stat     Country:  United States    
Other Details:
Languages:  eng     Pagination:  481-94     Citation Subset:  IM    
Affiliation:
Department of Health Evaluation Sciences, Penn State College of Medicine, Hershey, Pennsylvania 17033, USA. mshaffer@hes.hmc.psu.edu
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MeSH Terms
Descriptor/Qualifier:
Clinical Trials as Topic / statistics & numerical data*
Likelihood Functions*
Treatment Failure*
Grant Support
ID/Acronym/Agency:
U10 HL51845/HL/NHLBI NIH HHS

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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