多阶段抽样的精度控制及样本量计算

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Closed inverse sampling procedure for selecting the largest multinomial cell probability

A class of closed inverse sampling procedures R(n,m) for selecting the multinomial cell with the largest probability is considered; here n is the maximum sample size that an experimenter can take and m is the maximum frequency that a multinomial cell can have. The proposed procedures R(n,m) achieve the same probability of a correct selection as do the corresponding fixed sample size procedures and the curtailed sequential procedures when m is at least n/2. A monotonicity property on the probability of a correct selection is proved and it is used to find the least favorable configurations and to tabulate the necessary probabilities of a correct selection and corresponding expected sample sizes     

Adaptive interval estimation in one-way random effects models

Two-stage procedures are introduced to control the width and coverage (validity) of confidence intervals for the estimation of the mean, the between groups variance component and certain ratios of the variance components in one-way random effects models. The procedures use the pilot sample data to estimate an “optimal” group size and then proceed to determine the number of groups by a stopping rule. Such sampling plans give rise to unbalanced data, which are consequently analyzed by the harmonic mean method. Several asymptotic results concerning the proposed procedures are given along with simulation results to assess their performance in moderate sample size situations. The proposed procedures were found to effectively control the width and probability of coverage of the resulting confidence intervals in all cases and were also found to be robust in the presence of missing observations. From a practical point of view, the procedures are illustrated using a real data set and it is shown that the resulting unbalanced designs tend to require smaller sample sizes than is needed in a corresponding balanced design where the group size is arbitrarily pre-specified.     

Subset Selection Toward Optimizing the Best Performance at a Second Stage

In the search for the best of n candidates, two-stage procedures of the following type are in common use. In a first stage, weak candidates are removed, and the subset of promising candidates is then further examined. At a second stage, the best of the candidates in the subset is selected. In this article, optimization is not aimed at the parameter with largest value but rather at the best performance of the selected candidates at Stage 2. Under a normal model, a new procedure based on posterior percentiles is derived using a Bayes approach, where nonsymmetric normal (proper and improper) priors are applied. Comparisons are made with two other procedures frequently used in selection decisions. The three procedures and their performances are illustrated with data from a recent recruitment process at a Midwestern university.     

A Multiple Three-Decision Procedures for Comparing Several Treatments with a Control Under Heteroscedasticity

In this article, the design-oriented two-stage multiple three-decision procedure is proposed to classify a set of normal populations with respect to a control under heteroscedasticity. The statistical tables of percentage points and the power-related design constants, to implement our new two-stage procedure, are given. Sometimes when the sample for the second stage is not available, the one-stage data analysis procedure is proposed. Classifying a treatment better than control when it is actually worse (and vice versa) is known as type III error. Both the two-stage and one-stage procedures control the type III error rate at a specified level. The relationship between the two-stage and one-stage procedures is discussed. Finally, the application of the proposed procedures is illustrated with an example.     

Two stage fixed width confidence intervals for the common location parameter of several exponential distributins

Two stage sampling schemes are introduced for use in estimating the common location parameter (guarantee time) of two or more exponential distributions with a confidence interval of prespecified width whose coverage probability is at least a given nominal value. Exact expressions for all moments of order r ≥ 1 of the associated two stage sample sizes and for the actual coverage probabilities are derived. The performance of the procedures in a variety of two population, moderate fixed sample size cases is examined via numerical studies involving both exact calculations and Monte Carlo simulations. No new tables are needed to implement any of the proposed methods. A modified two stage procedure is recommended for practical use     

Classification procedures using principal component analysis and stepwise discriminant function

Classification procedures are examined in the case when the dimensionality exceeds the sample size. Two particular suggestions are (i) Principal components analysis and (ii) Two-step discriminant analysis. Comparisons are made in the two sample and the several sample cases. Extensions to growth curve model are investigated using the two stage discriminant analysis.     

New one-stage multiple comparison procedures with the average for exponential location parameters under heteroscedasticity

ABSTRACT

In this paper, we present new one-stage multiple comparison procedures with the average for location parameters of two-parameter exponential distributions under heteroscedasticity by modifying the existing one proposed by Wu [One stage multiple comparisons with the average for exponential location parameters under heteroscedasticity. Comput Stat Data Anal. 2013;68:352–360] with unequal sample sizes. A simulation study is done and the results show that the proposed procedures have shorter confidence length with coverage probabilities closer to the nominal ones. At last, an example of comparing the survival days of patients for four categories of lung cancer is given to demonstrate the proposed procedures.     

Closed testing procedures for all pairwise comparisons in a randomized block design

We consider multiple comparison test procedures among treatment effects in a randomized block design. We propose closed testing procedures based on maximum values of some two-sample t test statistics and based on F test statistics. It is shown that the proposed procedures are more powerful than single-step procedures and the REGW (Ryan/Einot–Gabriel/Welsch)-type tests. Next, we consider the randomized block design under simple ordered restrictions of treatment effects. We propose closed testing procedures based on maximum values of two-sample one-sided t test statistics and based on Batholomew’s statistics for all pairwise comparisons of treatment effects. Although single-step multiple comparison procedures are utilized in general, the power of these procedures is low for a large number of groups. The closed testing procedures stated in the present article are more powerful than the single-step procedures. Simulation studies are performed under the null hypothesis and some alternative hypotheses. In this studies, the proposed procedures show a good performance.     

Selecting the best normal population better than a standard under unequal variances

In this article we consider a problem of selecting the best normal population that is better than a standard when the variances are unequal. Single-stage selection procedures are proposed when the variances are known. Wilcox (1984) and Taneja and Dudewicz (1992) proposed two-stage selection procedures when the variances are unknown. In addition to these procedures, we propose a two-stage selection procedure based on the method of Lam (1988). Comparisons are made between these selection procedures in terms of the sample sizes.     

Optimal treatment allocation and study duration for trials with discrete-time survival endpoints

Studies on event occurrence may be conducted in experiments, where one or more treatment groups are compared to a control group. Most of the randomized trials are designed with equally sized groups, but this design is not always the best one. The statistical power of the study may be larger with unequal sample sizes, and researchers may want to place more participants in one group relative to the other due to resource constraints or costs. The optimal designs for discrete-time survival endpoints in trials with two groups, where different proportions of subjects in the experimental group are taken into account, can be studied using the generalized linear model. Applying a cost function, the optimal combination of the number of subjects and periods in the study and the optimal allocation ratio can be found. It is observed that the ratio of the recruitment costs in both groups, the ratio of the recruitment cost in the control group to the cost of obtaining a measurement, the size of the treatment effect, and the shape of the survival distribution have the greatest influence on the optimal design.     

Optimizing trial design in pharmacogenetics research: comparing a fixed parallel group,group sequential,and adaptive selection design on sample size requirements

Two‐stage clinical trial designs may be efficient in pharmacogenetics research when there is some but inconclusive evidence of effect modification by a genomic marker. Two‐stage designs allow to stop early for efficacy or futility and can offer the additional opportunity to enrich the study population to a specific patient subgroup after an interim analysis. This study compared sample size requirements for fixed parallel group, group sequential, and adaptive selection designs with equal overall power and control of the family‐wise type I error rate. The designs were evaluated across scenarios that defined the effect sizes in the marker positive and marker negative subgroups and the prevalence of marker positive patients in the overall study population. Effect sizes were chosen to reflect realistic planning scenarios, where at least some effect is present in the marker negative subgroup. In addition, scenarios were considered in which the assumed ‘true’ subgroup effects (i.e., the postulated effects) differed from those hypothesized at the planning stage. As expected, both two‐stage designs generally required fewer patients than a fixed parallel group design, and the advantage increased as the difference between subgroups increased. The adaptive selection design added little further reduction in sample size, as compared with the group sequential design, when the postulated effect sizes were equal to those hypothesized at the planning stage. However, when the postulated effects deviated strongly in favor of enrichment, the comparative advantage of the adaptive selection design increased, which precisely reflects the adaptive nature of the design. Copyright © 2013 John Wiley & Sons, Ltd.     

Curtailed two-stage designs with two dependent binary endpoints

When phase I clinical trials were found to be unable to precisely estimate the frequency of toxicity, Brayan and Day proposed incorporating toxicity considerations into two-stage designs in phase II clinical trials. Conaway and Petroni further pointed out that it is important to evaluate the clinical activity and safety simultaneously in studying cancer treatments with more toxic chemotherapies in a phase II clinical trial. Therefore, they developed multi-stage designs with two dependent binary endpoints. However, the usual sample sizes in phase II trials make these designs difficult to control the type I error rate at a desired level over the entire null region and still have sufficient power against reasonable alternatives. Therefore, the curtailed sampling procedure summarized by Phatak and Bhatt will be applied to the two-stage designs with two dependent binary endpoints in this paper to reduce sample sizes and speed up the development process for drugs.     

Bayesian sample size determination for phase IIA clinical trials using historical data and semi‐parametric prior's elicitation

The Simon's two‐stage design is the most commonly applied among multi‐stage designs in phase IIA clinical trials. It combines the sample sizes at the two stages in order to minimize either the expected or the maximum sample size. When the uncertainty about pre‐trial beliefs on the expected or desired response rate is high, a Bayesian alternative should be considered since it allows to deal with the entire distribution of the parameter of interest in a more natural way. In this setting, a crucial issue is how to construct a distribution from the available summaries to use as a clinical prior in a Bayesian design. In this work, we explore the Bayesian counterparts of the Simon's two‐stage design based on the predictive version of the single threshold design. This design requires specifying two prior distributions: the analysis prior, which is used to compute the posterior probabilities, and the design prior, which is employed to obtain the prior predictive distribution. While the usual approach is to build beta priors for carrying out a conjugate analysis, we derived both the analysis and the design distributions through linear combinations of B‐splines. The motivating example is the planning of the phase IIA two‐stage trial on anti‐HER2 DNA vaccine in breast cancer, where initial beliefs formed from elicited experts' opinions and historical data showed a high level of uncertainty. In a sample size determination problem, the impact of different priors is evaluated.     

Selecting logistic populations using the sample medians

This paper is concerned primarily with subset selection procedures based on the sample mediansof logistic populations. A procedure is given which chooses a nonempty subset from among kindependent logistic populations, having a common known variance, so that the populations with thelargest location parameter is contained in the subset with a pre‐specified probability. Theconstants required to apply the median procedure with small sample sizes (≤= 19) are tabulated and can also be used to construct simultaneous confidence intervals. Asymptotic formulae are provided for application with larger sample sizes. It is shown that, under certain situations, rules based on the median are substantially more efficient than analogous procedures based either on sample means or on the sum of joint ranks.     

Estimation of variance components in staggered nested designs

SUMMARY Variance components are estimated by two different methods for a general p stage random-effects staggered nested design. In addition to estimation from an analysis of variance, a new approach is introduced. The main features of this new technique are its simplicity and its ability to yield non-negative estimates of the variance components. The performances of the two procedures are compared using simulation and the meansquared-error criterion.     

A new response-adaptive design for continuous treatment responses for phase III clinical trials

A new response-adaptive design, applicable for general class of continuous response distributions, is proposed. The allocation design is studied both theoretically and numerically and compared with some existing procedures. The applicability of the proposed procedure is also illustrated using real life data sets.     

Efficiency and Validity Analyses of Two-Stage Estimation Procedures and Derived Testing Procedures in Quantitative Linear Models with AR(1) Errors

Abstract

In a quantitative linear model with errors following a stationary Gaussian, first-order autoregressive or AR(1) process, Generalized Least Squares (GLS) on raw data and Ordinary Least Squares (OLS) on prewhitened data are efficient methods of estimation of the slope parameters when the autocorrelation parameter of the error AR(1) process, ρ, is known. In practice, ρ is generally unknown. In the so-called two-stage estimation procedures, ρ is then estimated first before using the estimate of ρ to transform the data and estimate the slope parameters by OLS on the transformed data. Different estimators of ρ have been considered in previous studies. In this article, we study nine two-stage estimation procedures for their efficiency in estimating the slope parameters. Six of them (i.e., three noniterative, three iterative) are based on three estimators of ρ that have been considered previously. Two more (i.e., one noniterative, one iterative) are based on a new estimator of ρ that we propose: it is provided by the sample autocorrelation coefficient of the OLS residuals at lag 1, denoted r(1). Lastly, REstricted Maximum Likelihood (REML) represents a different type of two-stage estimation procedure whose efficiency has not been compared to the others yet. We also study the validity of the testing procedures derived from GLS and the nine two-stage estimation procedures. Efficiency and validity are analyzed in a Monte Carlo study. Three types of explanatory variable x in a simple quantitative linear model with AR(1) errors are considered in the time domain: Case 1, x is fixed; Case 2, x is purely random; and Case 3, x follows an AR(1) process with the same autocorrelation parameter value as the error AR(1) process. In a preliminary step, the number of inadmissible estimates and the efficiency of the different estimators of ρ are compared empirically, whereas their approximate expected value in finite samples and their asymptotic variance are derived theoretically. Thereafter, the efficiency of the estimation procedures and the validity of the derived testing procedures are discussed in terms of the sample size and the magnitude and sign of ρ. The noniterative two-stage estimation procedure based on the new estimator of ρ is shown to be more efficient for moderate values of ρ at small sample sizes. With the exception of small sample sizes, REML and its derived F-test perform the best overall. The asymptotic equivalence of two-stage estimation procedures, besides REML, is observed empirically. Differences related to the nature, fixed or random (uncorrelated or autocorrelated), of the explanatory variable are also discussed.     

Estimation procedures with delayed observations

The statistical model is considered in which the collection of data from several independent populations is available only at random times determined by order statistics of lifetimes of a given number of objects. Each of the populations is distributed according to a general multiparameter exponential family. The problem is to estimate the mean value vector parameter of the multiparameter exponential family of distributions of the forthcoming observations. Under the loss function involving a weighted squared error loss, the cost proportional to the events appeared and a cost of observing the process, a class of optimal sequential procedures is established. The procedures are derived in two situations: when the lifetime distribution is completely known and in the case when it is unknown but assumed to belong to an exponential subfamily with an unknown failure rate parameter.     

Bayesian two-stage optimal design for mixture models

In this paper, a Bayesian two-stage D–D optimal design for mixture experimental models under model uncertainty is developed. A Bayesian D-optimality criterion is used in the first stage to minimize the determinant of the posterior variances of the parameters. The second stage design is then generated according to an optimalityprocedure that collaborates with the improved model from the first stage data. The results show that a Bayesian two-stage D–D-optimal design for mixture experiments under model uncertainty is more efficient than both the Bayesian one-stage D-optimal design and the non-Bayesian one-stage D-optimal design in most situations. Furthermore, simulations are used to obtain a reasonable ratio of the sample sizes between the two stages.