Efficient non-parametric sequential procedures for comparing the rates of change of two treatments

Consider a longitudinal experiment where subjects are allocated to one of two treatment arms and are subjected to repeated measurements over time. Two non-parametric group sequential procedures, based on the Wilcoxon rank sum test and fitted with asymptotically efficient allocation rules, are derived to test the equality of the rates of change over time of the two treatments, when the distribution of responses is unknown. The procedures are designed to allow for early stopping to reject the null hypothesis while allocating less subjects to the inferior treatment. Simulations – based on the normal, the logistic and the exponential distributions – showed that the proposed allocation rules substantially reduce allocations to the inferior treatment, but at the expense of a relatively small increase in the total sample size and a moderate decrease in power as compared to the pairwise allocation rule.     

An extension of the ranked set sampling theory

This paper is concerned with ranked set sampling theory which is useful to estimate the population mean when the order of a sample of small size can be found without measurements or with rough methods. Consider n sets of elements each set having size m. All elements of each set are ranked but only one is selected and quantified. The average of the quantified elements is adopted as the estimator. In this paper we introduce the notion of selective probability which is a generalization of a notion from Yanagawa and Shirahata (1976). Uniformly optimal unbiased procedures are found for some (n,m). Furthermore, procedures which are unbiased for all distributions and are good for symmetric distributions are studied for (n,m) which do not allow uniformly optimal unbiased procedures.     

Bayes procedures for detecting a shift in the probability of success in a series of Bernoulli trials

The determination of a stopping rule for the detection of the time of an increase in the success probability of a sequence of independent Bernoulli trials is discussed. Both success probabilities are assumed unknown. A Bayesian approach is applied; the distribution of the location of the shift in the success probability is assumed geometric and the success probabilities are assumed to have known joint prior distribution. The costs involved are penalties for late or early stoppings. The nature of the optimal dynamic programming solution is discussed and a procedure for obtaining a suboptimal stopping rule is determined. The results indicate that the detection procedure is quite effective.     

Partitioning k multivariate normal populations according to equivalence with respect to a standard vector

We propose optimal procedures to achieve the goal of partitioning k multivariate normal populations into two disjoint subsets with respect to a given standard vector. Definition of good or bad multivariate normal populations is given according to their Mahalanobis distances to a known standard vector as being small or large. Partitioning k multivariate normal populations is reduced to partitioning k non-central Chi-square or non-central F distributions with respect to the corresponding non-centrality parameters depending on whether the covariance matrices are known or unknown. The minimum required sample size for each population is determined to ensure that the probability of correct decision attains a certain level. An example is given to illustrate our procedures.     

Adaptive cluster sampling with a data driven stopping rule

The adaptive cluster sampling (ACS) is a suitable sampling design for rare and clustered populations. In environmental and ecological applications, biological populations are generally animals or plants with highly patchy spatial distribution. However, ACS would be a less efficient design when the study population is not rare with low aggregation since the final sample size could be easily out of control. In this paper, a new variant of ACS is proposed in order to improve the performance (in term of precision and cost) of ACS versus simple random sampling (SRS). The idea is to detect the optimal sample size by means of a data-driven stopping rule in order to determine when to stop the adaptive procedure. By introducing a stopping rule the theoretical basis of ACS are not respected and the behaviour of the ordinary estimators used in ACS is explored by using Monte Carlo simulations. Results show that the proposed variant of ACS allows to control the effective sample size and to prevent from excessive efficiency loss typical of ACS when the population is less clustered than anticipated. The proposed strategy may be recommended especially when no prior information about the population structure is available as it does not require a prior knowledge of the degree of rarity and clustering of the population of interest.     

Selecting the Best Exponential Population When the Scale Parameters are Bounded

Consider k (≥ 2) independent exponential populations with different location and scale parameters. Call a population associated with largest of unknown location parameters as the best population. For the goal of selecting the best population, it is established that if the scale parameters are completely unknown, then the indifference-zone probability requirement can not be guaranteed by any single sample decision rule which is just and translation invariant. Under the assumption that the scale parameters are bounded above by a known constant, a single sample selection procedure is proposed for which the indifference-zone probability requirement can be guaranteed. Under the same assumption, 100P*% simultaneous upper confidence intervals for all distances from the largest location parameter are also obtained.     

Simulation-based sequential Bayesian design

We consider simulation-based methods for exploration and maximization of expected utility in sequential decision problems. We consider problems which require backward induction with analytically intractable expected utility integrals at each stage. We propose to use forward simulation to approximate the integral expressions, and a reduction of the allowable action space to avoid problems related to an increasing number of possible trajectories in the backward induction. The artificially reduced action space allows strategies to depend on the full history of earlier observations and decisions only indirectly through a low dimensional summary statistic. The proposed rule provides a finite-dimensional approximation to the unrestricted infinite-dimensional optimal decision rule. We illustrate the proposed approach with an application to an optimal stopping problem in a clinical trial.     

The risks for usual sequential estimates and stopping times of multivariate normal mean for conjugate distribution

The exact formulas of optimal stopping times for usual problems are often difficult to derive. Biekej and Yahav (1965) had provided the large sample approximation known as the asymptotically pointwise optimal (A. P.O.) rule. In Nagao (1997a.b). he has derived the asymptotic formulas for Bayes stopping times for the problems of the mean of a multivariate normal distribution when a covariance matrix is completely unknown and has some structure, respectively. This paper gives the risks for estimate and stopping times which we use in common for some problems. From this result, we find that its increasing amount shows the deficiency of estimate and stopping usually used from the view of the Bayes risk.     

Interval estimation of a small proportion via inverse sampling

On the basis of a negative binomial sampling scheme, we consider a uniformly most accurate upper confidence limit for a small but unknown proportion, such as the proportion of defectives in a manufacturing process. The optimal stopping rule, with reference to the twin criteria of the expected length of the confidence interval and the expected sample size, is investigated. The proposed confidence interval has also been compared with several others that have received attention in the recent literature.     

Minimax optimality of the Shiryayev–Roberts change-point detection rule

The result of Pollak [1985. Optimal detection of a change in distribution. Ann. Statist. 13, 206–227] proving the asymptotic optimality in sequential change-point detection of a suitable Shirayayev–Roberts stopping rule up to terms that vanish in the limit is generalized from the case of two completely specified distributions to that of a composite alternative hypothesis in a multidimensional exponential family. An explicit asymptotic lower bound on the expected Kullback–Leibler information required to detect a change-point is derived and is shown to be attained by a Shirayayev–Roberts stopping rule.     

Three-stage and accelerated sequential point estimation of the normal mean using LINEX loss function

Consider a normal population with unknown mean μ and unknown variance σ². We estimate μ under an asymmetric LINEX loss function such that the associated risk is bounded from above by a known quantity w. This necessitates the use of a random number (N) of observations. Under a fairly broad set of assumptions on N, we derive the asymptotic second-order expansion of the associated risk function. Some examples have been included involving accelerated sequential and three-stage sampling techniques. Performance comparisons of these procedures are considered using a Monte-Carlo study.     

Selecting the better binomial population under a budget constraint

The problem of optimal non-sequential allocation of observations for the selection of the better binomial population is considered in the case of fixed sampling costs and budget. With the appropriate choice of selection rule it is shown that a 70% reduction in the probability of incorrect selection is possible by using an unequal rather than equal allocation. Simple formulae are given for the appropriate selection rule and unequal allocation in large samples.     

Multivariate inverse Gaussian distribution as a limit of multivariate waiting time distributions

Multivariate inverse Gaussian distribution proposed by Minami [2003. A multivariate extension of inverse Gaussian distribution derived from inverse relationship. Commun. Statist. Theory Methods 32(12), 2285–2304] was derived through multivariate inverse relationship with multivariate Gaussian distributions and characterized as the distribution of the location at a certain stopping time of a multivariate Brownian motion. In this paper, we show that the multivariate inverse Gaussian distribution is also a limiting distribution of multivariate Lagrange distributions, which is a family of waiting time distributions, under certain conditions.     

ON EMPIRICAL BAYES STOPPING TIMES

We consider the empirical Bayes decision theory where the component problems are the optimal fixed sample size decision problem and a sequential decision problem. With these components, an empirical Bayes decision procedure selects both a stopping rule function and a terminal decision rule function. Empirical Bayes stopping rules are constructed for each case and the asymptotic behaviours are investigated.     

A cluster‐sample approach for Monte Carlo integration using multiple samplers

A computational problem in many fields is to estimate simultaneously multiple integrals and expectations, assuming that the data are generated by some Monte Carlo algorithm. Consider two scenarios in which draws are simulated from multiple distributions but the normalizing constants of those distributions may be known or unknown. For each scenario, existing estimators can be classified as using individual samples separately or using all the samples jointly. The latter pooled‐sample estimators are statistically more efficient but computationally more costly to evaluate than the separate‐sample estimators. We develop a cluster‐sample approach to obtain computationally effective estimators, after draws are generated for each scenario. We divide all the samples into mutually exclusive clusters and combine samples from each cluster separately. Furthermore, we exploit a relationship between estimators based on samples from different clusters to achieve variance reduction. The resulting estimators, compared with the pooled‐sample estimators, typically yield similar statistical efficiency but have reduced computational cost. We illustrate the value of the new approach by two examples for an Ising model and a censored Gaussian random field. The Canadian Journal of Statistics 41: 151–173; 2013 © 2012 Statistical Society of Canada     

Fixed-width interval estimation of the minimum point of a regression function based on the Kiefer-Wolfowitz procedure

A version of the central limit theorem for the Kiefer-Wolfowitz procedure is stated. One constructs an asymptotically consistent fixed-width confidence interval for the minimum of a regression function. It is shown that the first moment of the corresponding stopping rule is finite. Both the construction and properties of the estimates of unknown parameters and an adaptive version of the procedure are presented.     

Optimal,nearly optimal and robust procedures for the exponential distribution with relative linear exponential loss

In this paper, the problem of sequentially estimating the mean of the exponential distribution with relative linear exponential loss and fixed cost for each observation is considered within the Bayesian framework. An optimal procedure with a deterministic stopping rule is derived. Since the corresponding value of the optimal deterministic stopping rule cannot be obtained directly, an approximate optimal deterministic stopping rule and an asymptotically pointwise optimal rule are proposed. In addition, we propose a robust procedure with a deterministic stopping rule, which does not depend on the parameters of the prior distribution. All of the proposed procedures are shown to be asymptotically optimal. Some numerical studies are conducted to investigate the performances of the proposed procedures. A real data set is provided to illustrate the use of the proposed procedures.     

Early stopping in seamless phase I/II clinical trials

In recent years, seamless phase I/II clinical trials have drawn much attention, as they consider both toxicity and efficacy endpoints in finding an optimal dose (OD). Engaging an appropriate number of patients in a trial is a challenging task. This paper attempts a dynamic stopping rule to save resources in phase I/II trials. That is, the stopping rule aims to save patients from unnecessary toxic or subtherapeutic doses. We allow a trial to stop early when widths of the confidence intervals for the dose-response parameters become narrower or when the sample size is equal to a predefined size, whichever comes first. The simulation study of dose-response scenarios in various settings demonstrates that the proposed stopping rule can engage an appropriate number of patients. Therefore, we suggest its use in clinical trials.     

A nonparametric Bayesian model for inference in related longitudinal studies

Summary.  We discuss a method for combining different but related longitudinal studies to improve predictive precision. The motivation is to borrow strength across clinical studies in which the same measurements are collected at different frequencies. Key features of the data are heterogeneous populations and an unbalanced design across three studies of interest. The first two studies are phase I studies with very detailed observations on a relatively small number of patients. The third study is a large phase III study with over 1500 enrolled patients, but with relatively few measurements on each patient. Patients receive different doses of several drugs in the studies, with the phase III study containing significantly less toxic treatments. Thus, the main challenges for the analysis are to accommodate heterogeneous population distributions and to formalize borrowing strength across the studies and across the various treatment levels. We describe a hierarchical extension over suitable semiparametric longitudinal data models to achieve the inferential goal. A nonparametric random-effects model accommodates the heterogeneity of the population of patients. A hierarchical extension allows borrowing strength across different studies and different levels of treatment by introducing dependence across these nonparametric random-effects distributions. Dependence is introduced by building an analysis of variance (ANOVA) like structure over the random-effects distributions for different studies and treatment combinations. Model structure and parameter interpretation are similar to standard ANOVA models. Instead of the unknown normal means as in standard ANOVA models, however, the basic objects of inference are random distributions, namely the unknown population distributions under each study. The analysis is based on a mixture of Dirichlet processes model as the underlying semiparametric model.     

Asymptotically pointwise optimal and asymptotically optimal stopping times in the Bayesian inference

The paper deals with the problem of determining asymptotically pointwise optimal and asymptotically optimal stopping times in the Bayesian inference. The sufficient conditions are given for a family of stopping times to be asymptotically pointwise optimal and asymptotically optimal with respect to a continuous time process. As an example a sequential estimation of the intensity of the Poisson process is considered. Under a gamma prior distribution, an asymptotically pointwise optimal and asymptotically optimal rule is given using a LINEX loss function and the cost c per unit time.