Start-up designs for response-adaptive randomization procedures with sequential estimation. 2015

Linda M Haines, and Hassan Sadiq
Department of Statistical Sciences, University of Cape Town, Private Bag X3, Rondebosch, 7701, South Africa.

Response-adaptive randomization procedures are appropriate for clinical trials in which two or more treatments are to be compared, patients arrive sequentially and the response of each patient is recorded before the next patient arrives. However, for those procedures that involve sequential estimation of model parameters, start-up designs are commonly required in order to provide initial estimates of the parameters. In this paper, a suite of such start-up designs for two treatments and binary patient responses are considered and compared in terms of the numbers of patients required in order to give meaningful parameters estimates, the number of patients allocated to the better treatment, and the bias in the parameter estimates. It is shown that permuted block designs with blocks of size 4 are to be preferred over a wide range of parameter values. For the case of two treatments, normal responses and selected start-up procedures, a design incorporating complete randomization followed appropriately by repeats of one of the treatments yields the minimum expected number of patients and is to be preferred.

UI MeSH Term Description Entries
D011897 Random Allocation A process involving chance used in therapeutic trials or other research endeavor for allocating experimental subjects, human or animal, between treatment and control groups, or among treatment groups. It may also apply to experiments on inanimate objects. Randomization,Allocation, Random
D012107 Research Design A plan for collecting and utilizing data so that desired information can be obtained with sufficient precision or so that an hypothesis can be tested properly. Experimental Design,Data Adjustment,Data Reporting,Design, Experimental,Designs, Experimental,Error Sources,Experimental Designs,Matched Groups,Methodology, Research,Problem Formulation,Research Methodology,Research Proposal,Research Strategy,Research Technics,Research Techniques,Scoring Methods,Adjustment, Data,Adjustments, Data,Data Adjustments,Design, Research,Designs, Research,Error Source,Formulation, Problem,Formulations, Problem,Group, Matched,Groups, Matched,Matched Group,Method, Scoring,Methods, Scoring,Problem Formulations,Proposal, Research,Proposals, Research,Reporting, Data,Research Designs,Research Proposals,Research Strategies,Research Technic,Research Technique,Scoring Method,Source, Error,Sources, Error,Strategies, Research,Strategy, Research,Technic, Research,Technics, Research,Technique, Research,Techniques, Research
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D001699 Biometry The use of statistical and mathematical methods to analyze biological observations and phenomena. Biometric Analysis,Biometrics,Analyses, Biometric,Analysis, Biometric,Biometric Analyses
D015982 Bias Any deviation of results or inferences from the truth, or processes leading to such deviation. Bias can result from several sources: one-sided or systematic variations in measurement from the true value (systematic error); flaws in study design; deviation of inferences, interpretations, or analyses based on flawed data or data collection; etc. There is no sense of prejudice or subjectivity implied in the assessment of bias under these conditions. Aggregation Bias,Bias, Aggregation,Bias, Ecological,Bias, Statistical,Bias, Systematic,Ecological Bias,Outcome Measurement Errors,Statistical Bias,Systematic Bias,Bias, Epidemiologic,Biases,Biases, Ecological,Biases, Statistical,Ecological Biases,Ecological Fallacies,Ecological Fallacy,Epidemiologic Biases,Experimental Bias,Fallacies, Ecological,Fallacy, Ecological,Scientific Bias,Statistical Biases,Truncation Bias,Truncation Biases,Bias, Experimental,Bias, Scientific,Bias, Truncation,Biase, Epidemiologic,Biases, Epidemiologic,Biases, Truncation,Epidemiologic Biase,Error, Outcome Measurement,Errors, Outcome Measurement,Outcome Measurement Error
D018401 Sample Size The number of units (persons, animals, patients, specified circumstances, etc.) in a population to be studied. The sample size should be big enough to have a high likelihood of detecting a true difference between two groups. (From Wassertheil-Smoller, Biostatistics and Epidemiology, 1990, p95) Sample Sizes,Size, Sample,Sizes, Sample

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