Incorporating intermediate binary responses into interim analyses of clinical trials: a comparison of four methods.
In clinical trials with a long period of time between randomization and the primary assessment of the patient, the same assessments are often undertaken at intermediate times. When an interim analysis is conducted, in addition to the patients who have completed the primary assessment, there will be those who have till then undergone only intermediate assessments. The efficiency of the interim analysis can be increased by the inclusion of data from these additional patients. This paper compares four methods of increasing information based on model-free estimates of transition probabilities to incorporate intermediate assessments from patients who have not completed the trial. It is assumed that the observations are binary and that there is one intermediate assessment. The methods are the score and Wald approaches, each with the log-odds ratio and probability difference parameterizations. Simulations show that all four approaches have good properties in moderate to large sample sizes.
Whitehead
Anne
A
The University of Reading, Reading RG6 6FN, U.K. p.a.whitehead@reading.ac.uk
Sooriyarachchi
Marina Roshini
MR
Whitehead
John
J
Bolland
Kim
K
D002986:Clinical Trials as Topic / Q000379:methods*; D017326:Clinical Trials, Phase III as Topic / Q000379:methods; D003198:Computer Simulation; D003627:Data Interpretation, Statistical; D006801:Humans; D016013:Likelihood Functions; D016017:Odds Ratio; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D020521:Stroke / Q000188:drug therapy; D013997:Time Factors