A Bayesian sequential design with binary outcome

Several researchers have proposed solutions to control type I error rate in sequential designs. The use of Bayesian sequential design becomes more common; however, these designs are subject to inflation of the type I error rate. We propose a Bayesian sequential design for binary outcome using an alpha‐spending function to control the overall type I error rate. Algorithms are presented for calculating critical values and power for the proposed designs. We also propose a new stopping rule for futility. Sensitivity analysis is implemented for assessing the effects of varying the parameters of the prior distribution and maximum total sample size on critical values. Alpha‐spending functions are compared using power and actual sample size through simulations. Further simulations show that, when total sample size is fixed, the proposed design has greater power than the traditional Bayesian sequential design, which sets equal stopping bounds at all interim analyses. We also find that the proposed design with the new stopping for futility rule results in greater power and can stop earlier with a smaller actual sample size, compared with the traditional stopping rule for futility when all other conditions are held constant. Finally, we apply the proposed method to a real data set and compare the results with traditional designs.     

A simulation study of methods for selecting subgroup‐specific doses in phase 1 trials

Patient heterogeneity may complicate dose‐finding in phase 1 clinical trials if the dose‐toxicity curves differ between subgroups. Conducting separate trials within subgroups may lead to infeasibly small sample sizes in subgroups having low prevalence. Alternatively,it is not obvious how to conduct a single trial while accounting for heterogeneity. To address this problem,we consider a generalization of the continual reassessment method on the basis of a hierarchical Bayesian dose‐toxicity model that borrows strength between subgroups under the assumption that the subgroups are exchangeable. We evaluate a design using this model that includes subgroup‐specific dose selection and safety rules. A simulation study is presented that includes comparison of this method to 3 alternative approaches,on the basis of nonhierarchical models,that make different types of assumptions about within‐subgroup dose‐toxicity curves. The simulations show that the hierarchical model‐based method is recommended in settings where the dose‐toxicity curves are exchangeable between subgroups. We present practical guidelines for application and provide computer programs for trial simulation and conduct.     

Colorful Disclosures: Identifying Identity-Based Differences and Enhancing Critical Consciousness in Supervision

ABSTRACT

Managing microaggressions and marginalizing experiences while negotiating the ongoing oppression that occurs in micro, mezzo, and macro settings can be particularly challenging for clinicians of color. Thus, supervision with clinicians of color must include affirmation, empowerment, and exploration of the intracultural/intercultural dynamics inherent in the treatment-providing process and clinical supervision. Through case studies, autoethnographic studies of our supervision experiences, and interviews with supervisors and supervisees, we reflect on how axes of identity, including race, power, and privilege, inform practitioners’ clinical lenses and affect their vulnerability in treatment and the clinical supervision dyad. Special attention is placed on the clinical supervisor–clinician–client triad (the triple process) and the interpersonal dynamics of cultural sensitivity, cultural humility, and authentic responsiveness that supervisors aim to model and cultivate in the supervisory relationship. In addition to sustaining clinical growth for clinicians of color, adding this level of complexity to supervision supports equity in direct clinical practice, enhancing efficacy outcomes for clients and communities. Recommendations and pedagogical strategies are offered to support supervisors in initiating difficult dialogues and shifting the paradigm to promote this transformational perspective.     

Unifying CRM and EWOC designs for phase I cancer clinical trials

The main goal of phase I cancer clinical trials is to determine the highest dose of a new therapy associated with an acceptable level of toxicity for the use in a subsequent phase II trial. The continual reassessment method (CRM) [O’Quigley, J., Pepe, M., Fisher, L., 1990. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 46, 33–48] and escalation with overdose control (EWOC) [Babb, J., Rogatko, A., Zacks, S., 1998. Cancer phase I clinical trials: efficient dose escalation with overdose control. Statist. Med. 17 (10), 1103–1120] are two model-based designs used for phase I cancer clinical trials. A few modifications of the (original) CRM and EWOC have been made by many authors. In this paper, we show how CRM and EWOC can be unified and present a hybrid design. We study the characteristics of the approach of the hybrid design. The comparisons of the three designs (CRM, EWOC, and the hybrid design) are presented by convergence rates and overdose proportions. The simulation results show that the hybrid design generally has faster convergence rates than EWOC and smaller overdose proportions than CRM, especially when the true maximum tolerated dose (MTD) is above the mid-level of the dose range considered. The performance of these three designs is also evaluated in terms of sensitivity to outliers.     

Notes on testing carry-over effects in continuous data under an incomplete block crossover design

We consider hypothesis testing and estimation of carry-over effects in continuous data under an incomplete block crossover design when comparing two experimental treatments with a placebo. We develop procedures for testing differential carry-over effects based on the weighted-least-squares (WLS) method. We apply Monte Carlo simulations to evaluate the performance of these test procedures in a variety of situations. We use the data regarding the forced expiratory volume in one second (FEV₁) readings taken from a double-blind crossover trial comparing two different doses of formoterol with a placebo to illustrate the use of test procedures proposed here.     

A stratified unrelated question randomized response model using Neyman allocation

This paper applies stratified random sampling using Neyman allocation to Mangat et al. (1992 Mangat, N.S., Singh, R., Singh, S. (1992). An improved unrelated question randomized response strategy. Cal. Stat. Assoc. Bull. 42:277–281.[Crossref] , [Google Scholar]) unrelated question randomized response (RR) strategy for both completely truthful reporting and less than completely truthful reporting. It is shown that, for the prior information given, our new model is more efficient in terms of variance (in the case of completely truthful reporting) and mean square error (in terms of less than completely truthful reporting) than Kim and Elam's (2007 Kim, J.M., Elam, M.E. (2007). A stratified unrelated question randomized response model. Stat. Papers 48:215–233.[Crossref], [Web of Science ®] , [Google Scholar]) model. Numerical illustrations and graphs are also given in support of the present study.     

An optional randomized response model for estimating a rare sensitive attribute using Poisson distribution

The crux of this article is to estimate the mean of the number of persons possessing a rare sensitive attribute based on the Mangat (1991 Mangat, N.S. (1991). An optional randomized response sampling technique using non–stigmatized attribute. Statistica. 51(4):595–602. [Google Scholar]) randomization device by utilizing the Poisson distribution in simple random sampling and stratified sampling. Properties of the proposed randomized response (RR) model have been studied along with recommendations. It is also shown that the proposed model is more efficient than that of Land et al. (2011 Land, M., Singh, S., Sedory, S.A. (2011). Estimation of a rare attribute using Poisson distribution. Statistics. 46(3):351–360.[Taylor &; Francis Online], [Web of Science ®] , [Google Scholar]) in simple random sampling and that of Lee et al. (2013 Lee, G.S., Uhm, D., Kim, J.M. (2013). Estimation of a rare sensitive attribute in stratified sampling using Poisson distribution. Statistics. 47(3):575–589.[Taylor &; Francis Online], [Web of Science ®] , [Google Scholar]) in stratified random sampling when the proportion of persons possessing a rare unrelated attribute is known. Numerical illustrations are also given in support of the present study.     

Optimal and lead-in adaptive allocation for binary outcomes: A comparison of Bayesian methods

We compare posterior and predictive estimators and probabilities in response-adaptive randomization designs for two- and three-group clinical trials with binary outcomes. Adaptation based upon posterior estimates are discussed, as are two predictive probability algorithms: one using the traditional definition, the other using a skeptical distribution. Optimal and natural lead-in designs are covered. Simulation studies show that efficacy comparisons lead to more adaptation than center comparisons, though at some power loss, skeptically predictive efficacy comparisons and natural lead-in approaches lead to less adaptation but offer reduced allocation variability. Though nuanced, these results help clarify the power-adaptation trade-off in adaptive randomization.     

Improved estimation methods for unrelated question randomized response techniques

The randomized response technique (RRT) is an important tool, commonly used to avoid biased answers in survey on sensitive issues by preserving the respondents’ privacy. In this paper, we introduce a data collection method for survey on sensitive issues combining both the unrelated-question RRT and the direct question design. The direct questioning method is utilized to obtain responses to a non sensitive question that is related to the innocuous question from the unrelated-question RRT. These responses serve as additional information that can be used to improve the estimation of the prevalence of the sensitive behavior. Furthermore, we propose two new methods for the estimation of the proportion of respondents possessing the sensitive attribute under a missing data setup. More specifically, we develop the weighted estimator and the weighted conditional likelihood estimator. The performances of our estimators are studied numerically and compared with that of an existing one. Both proposed estimators are more efficient than the Greenberg's estimator. We illustrate our methods using real data from a survey study on illegal use of cable TV service in Taiwan.