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Articles 1 - 30 of 82
Full-Text Articles in Clinical Trials
An Improved Bayesian Pick-The-Winner (Ibpw) Design For Randomized Phase Ii Clinical Trials, Wanni Lei, Maosen Peng, Xi K. Zhou
An Improved Bayesian Pick-The-Winner (Ibpw) Design For Randomized Phase Ii Clinical Trials, Wanni Lei, Maosen Peng, Xi K. Zhou
COBRA Preprint Series
Phase II clinical trials play a pivotal role in drug development by screening a large number of drug candidates to identify those with promising preliminary efficacy for phase III testing. Trial designs that enable efficient decision-making with small sample sizes and early futility stopping while controlling for type I and II errors in hypothesis testing, such as Simon’s two-stage design, are preferred. Randomized multi-arm trials are increasingly used in phase II settings to overcome the limitations associated with using historical controls as the reference. However, how to effectively balance efficiency and accurate decision-making continues to be an important research topic. …
A Modular Framework For Early-Phase Seamless Oncology Trials, Philip S. Boonstra, Thomas M. Braun, Elizabeth C. Chase
A Modular Framework For Early-Phase Seamless Oncology Trials, Philip S. Boonstra, Thomas M. Braun, Elizabeth C. Chase
The University of Michigan Department of Biostatistics Working Paper Series
Background: As our understanding of the etiology and mechanisms of cancer becomes more sophisticated and the number of therapeutic options increases, phase I oncology trials today have multiple primary objectives. Many such designs are now 'seamless', meaning that the trial estimates both the maximum tolerated dose and the efficacy at this dose level. Sponsors often proceed with further study only with this additional efficacy evidence. However, with this increasing complexity in trial design, it becomes challenging to articulate fundamental operating characteristics of these trials, such as (i) what is the probability that the design will identify an acceptable, i.e. safe …
Unified Methods For Feature Selection In Large-Scale Genomic Studies With Censored Survival Outcomes, Lauren Spirko-Burns, Karthik Devarajan
Unified Methods For Feature Selection In Large-Scale Genomic Studies With Censored Survival Outcomes, Lauren Spirko-Burns, Karthik Devarajan
COBRA Preprint Series
One of the major goals in large-scale genomic studies is to identify genes with a prognostic impact on time-to-event outcomes which provide insight into the disease's process. With rapid developments in high-throughput genomic technologies in the past two decades, the scientific community is able to monitor the expression levels of tens of thousands of genes and proteins resulting in enormous data sets where the number of genomic features is far greater than the number of subjects. Methods based on univariate Cox regression are often used to select genomic features related to survival outcome; however, the Cox model assumes proportional hazards …
Robust Inference For The Stepped Wedge Design, James P. Hughes, Patrick J. Heagerty, Fan Xia, Yuqi Ren
Robust Inference For The Stepped Wedge Design, James P. Hughes, Patrick J. Heagerty, Fan Xia, Yuqi Ren
UW Biostatistics Working Paper Series
Based on a permutation argument, we derive a closed form expression for an estimate of the treatment effect, along with its standard error, in a stepped wedge design. We show that these estimates are robust to misspecification of both the mean and covariance structure of the underlying data-generating mechanism, thereby providing a robust approach to inference for the treatment effect in stepped wedge designs. We use simulations to evaluate the type I error and power of the proposed estimate and to compare the performance of the proposed estimate to the optimal estimate when the correct model specification is known. The …
Evaluation Of Multiple Interventions Using A Stepped Wedge Design, Vivian H. Lyons, Lingyu Li, James Hughes, Ali Rowhani-Rahbar
Evaluation Of Multiple Interventions Using A Stepped Wedge Design, Vivian H. Lyons, Lingyu Li, James Hughes, Ali Rowhani-Rahbar
UW Biostatistics Working Paper Series
Background: Stepped wedge cluster randomized trials are a class of unidirectional crossover studies that have historically been limited to evaluating a single intervention. This design is especially suitable for pragmatic trials where the study feasibility can be improved with a phased introduction of the intervention. We examined variations of stepped wedge designs that would support evaluation of multiple interventions. Methods: We propose four different design variants for implementing a stepped wedge trial with two interventions: concurrent design, supplementation, replacement, and factorial designs. Analyses were conducted comparing the precision of the estimated intervention effects for the different designs. Results: Concurrent, …
Adaptive Non-Inferiority Margins Under Observable Non-Constancy, Brett S. Hanscom, Deborah J. Donnell, Brian D. Williamson, Jim Hughes
Adaptive Non-Inferiority Margins Under Observable Non-Constancy, Brett S. Hanscom, Deborah J. Donnell, Brian D. Williamson, Jim Hughes
UW Biostatistics Working Paper Series
A central assumption in the design and conduct of non-inferiority trials is that the active-control therapy will have the same degree of effectiveness in the planned non-inferiority trial as it had in the prior placebo-controlled trials used to define the non-inferiority margin. This is referred to as the `constancy' assumption. If the constancy assumption fails, the chosen non-inferiority margin is not valid and the study runs the risk of approving an inferior product or failing to approve a beneficial product. The constancy assumption cannot be validated in a trial without a placebo arm, and it is unlikely ever to be …
Variance Prior Specification For A Basket Trial Design Using Bayesian Hierarchical Modeling, Kristen Cunanan, Alexia Iasonos, Ronglai Shen, Mithat Gonen
Variance Prior Specification For A Basket Trial Design Using Bayesian Hierarchical Modeling, Kristen Cunanan, Alexia Iasonos, Ronglai Shen, Mithat Gonen
Memorial Sloan-Kettering Cancer Center, Dept. of Epidemiology & Biostatistics Working Paper Series
Background: In the era of targeted therapies, clinical trials in oncology are rapidly evolving, wherein patients from multiple diseases are now enrolled and treated according to their genomic mutation(s). In such trials, known as basket trials, the different disease cohorts form the different baskets for inference. Several approaches have been proposed in the literature to efficiently use information from all baskets while simultaneously screening to find individual baskets where the drug works. Most proposed methods are developed in a Bayesian paradigm that requires specifying a prior distribution for a variance parameter, which controls the degree to which information is shared …
Studying The Optimal Scheduling For Controlling Prostate Cancer Under Intermittent Androgen Suppression, Sunil K. Dhar, Hans R. Chaudhry, Bruce G. Bukiet, Zhiming Ji, Nan Gao, Thomas W. Findley
Studying The Optimal Scheduling For Controlling Prostate Cancer Under Intermittent Androgen Suppression, Sunil K. Dhar, Hans R. Chaudhry, Bruce G. Bukiet, Zhiming Ji, Nan Gao, Thomas W. Findley
Harvard University Biostatistics Working Paper Series
This retrospective study shows that the majority of patients’ correlations between PSA and Testosterone during the on-treatment period is at least 0.90. Model-based duration calculations to control PSA levels during off-treatment are provided. There are two pairs of models. In one pair, the Generalized Linear Model and Mixed Model are both used to analyze the variability of PSA at the individual patient level by using the variable “Patient ID” as a repeated measure. In the second pair, Patient ID is not used as a repeated measure but additional baseline variables are included to analyze the variability of PSA.
Models For Hsv Shedding Must Account For Two Levels Of Overdispersion, Amalia Magaret
Models For Hsv Shedding Must Account For Two Levels Of Overdispersion, Amalia Magaret
UW Biostatistics Working Paper Series
We have frequently implemented crossover studies to evaluate new therapeutic interventions for genital herpes simplex virus infection. The outcome measured to assess the efficacy of interventions on herpes disease severity is the viral shedding rate, defined as the frequency of detection of HSV on the genital skin and mucosa. We performed a simulation study to ascertain whether our standard model, which we have used previously, was appropriately considering all the necessary features of the shedding data to provide correct inference. We simulated shedding data under our standard, validated assumptions and assessed the ability of 5 different models to reproduce the …
The Myth Of Making Inferences For An Overall Treatment Efficacy With Data From Multiple Comparative Studies Via Meta-Analysis, Takahiro Hasegawa, Brian Claggett, Lu Tian, Scott D. Solomon, Marc A. Pfeffer, Lee-Jen Wei
The Myth Of Making Inferences For An Overall Treatment Efficacy With Data From Multiple Comparative Studies Via Meta-Analysis, Takahiro Hasegawa, Brian Claggett, Lu Tian, Scott D. Solomon, Marc A. Pfeffer, Lee-Jen Wei
Harvard University Biostatistics Working Paper Series
Meta analysis techniques, if applied appropriately, can provide a summary of the totality of evidence regarding an overall difference between a new treatment and a control group using data from multiple comparative clinical studies. The standard meta analysis procedures, however, may not give a meaningful between-group difference summary measure or identify a meaningful patient population of interest, especially when the fixed effect model assumption is not met. Moreover, a single between-group comparison measure without a reference value obtained from patients in the control arm would likely not be informative enough for clinical decision making. In this paper, we propose a …
C-Learning: A New Classification Framework To Estimate Optimal Dynamic Treatment Regimes, Baqun Zhang, Min Zhang
C-Learning: A New Classification Framework To Estimate Optimal Dynamic Treatment Regimes, Baqun Zhang, Min Zhang
The University of Michigan Department of Biostatistics Working Paper Series
Personalizing treatment to accommodate patient heterogeneity and the evolving nature of a disease over time has received considerable attention lately. A dynamic treatment regime is a set of decision rules, each corresponding to a decision point, that determine that next treatment based on each individual’s own available characteristics and treatment history up to that point. We show that identifying the optimal dynamic treatment regime can be recast as a sequential classification problem and is equivalent to sequentially minimizing a weighted expected misclassification error. This general classification perspective targets the exact goal of optimally individualizing treatments and is new and fundamentally …
Hypothesis Testing For An Extended Cox Model With Time-Varying Coefficients, Takumi Saegusa, Chongzhi Di, Ying Qing Chen
Hypothesis Testing For An Extended Cox Model With Time-Varying Coefficients, Takumi Saegusa, Chongzhi Di, Ying Qing Chen
UW Biostatistics Working Paper Series
The log-rank test has been widely used to test a treatment effect under the Cox model for censored time-to-event outcomes, though it may lose power substantially when the model's proportional hazards assumption does not hold. In this paper, we consider an extended Cox model that uses B-splines or smoothing splines to model a time-varying treatment effect and propose score test statistics for the treatment effect. Our proposed new tests combine statistical evidence from both the magnitude and the shape of the time-varying hazard ratio function, and thus are omnibus and powerful against various types of alternatives. In addition, the new …
An Evaluation Of Inferential Procedures For Adaptive Clinical Trial Designs With Pre-Specified Rules For Modifying The Sample Size, Greg P. Levin, Sarah C. Emerson, Scott S. Emerson
An Evaluation Of Inferential Procedures For Adaptive Clinical Trial Designs With Pre-Specified Rules For Modifying The Sample Size, Greg P. Levin, Sarah C. Emerson, Scott S. Emerson
UW Biostatistics Working Paper Series
Many papers have introduced adaptive clinical trial methods that allow modifications to the sample size based on interim estimates of treatment effect. There has been extensive commentary on type I error control and efficiency considerations, but little research on estimation after an adaptive hypothesis test. We evaluate the reliability and precision of different inferential procedures in the presence of an adaptive design with pre-specified rules for modifying the sampling plan. We extend group sequential orderings of the outcome space based on the stage at stopping, likelihood ratio test statistic, and sample mean to the adaptive setting in order to compute …
Effectively Selecting A Target Population For A Future Comparative Study, Lihui Zhao, Lu Tian, Tianxi Cai, Brian Claggett, L. J. Wei
Effectively Selecting A Target Population For A Future Comparative Study, Lihui Zhao, Lu Tian, Tianxi Cai, Brian Claggett, L. J. Wei
Harvard University Biostatistics Working Paper Series
When comparing a new treatment with a control in a randomized clinical study, the treatment effect is generally assessed by evaluating a summary measure over a specific study population. The success of the trial heavily depends on the choice of such a population. In this paper, we show a systematic, effective way to identify a promising population, for which the new treatment is expected to have a desired benefit, using the data from a current study involving similar comparator treatments. Specifically, with the existing data we first create a parametric scoring system using multiple covariates to estimate subject-specific treatment differences. …
On The Covariate-Adjusted Estimation For An Overall Treatment Difference With Data From A Randomized Comparative Clinical Trial, Lu Tian, Tianxi Cai, Lihui Zhao, L. J. Wei
On The Covariate-Adjusted Estimation For An Overall Treatment Difference With Data From A Randomized Comparative Clinical Trial, Lu Tian, Tianxi Cai, Lihui Zhao, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Adaptive Clinical Trial Designs With Pre-Specified Rules For Modifying The Sample Size: Understanding Efficient Types Of Adaptation, Gregory P. Levin, Sarah C. Emerson, Scott S. Emerson
Adaptive Clinical Trial Designs With Pre-Specified Rules For Modifying The Sample Size: Understanding Efficient Types Of Adaptation, Gregory P. Levin, Sarah C. Emerson, Scott S. Emerson
UW Biostatistics Working Paper Series
Methods allowing unplanned adaptations to the sample size based on the interim estimate of treatment effect do not base inference on the minimal sufficient statistic and suffer losses in efficiency when compared to group sequential designs [1, 2, 3]. However, when adaptive sampling plans are completely pre-specified at the design stage of the trial, investigators can proceed with frequentist inference based on the minimal sufficient statistic at the analysis stage. In the context of two general settings where different optimality criteria govern the choice of clinical trial design, we quantify the relative costs and benefits of a variety of fixed …
Estimating Subject-Specific Treatment Differences For Risk-Benefit Assessment With Competing Risk Event-Time Data, Brian Claggett, Lihui Zhao, Lu Tian, Davide Castagno, L. J. Wei
Estimating Subject-Specific Treatment Differences For Risk-Benefit Assessment With Competing Risk Event-Time Data, Brian Claggett, Lihui Zhao, Lu Tian, Davide Castagno, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Stratifying Subjects For Treatment Selection With Censored Event Time Data From A Comparative Study, Lihui Zhao, Tianxi Cai, Lu Tian, Hajime Uno, Scott D. Solomon, L. J. Wei
Stratifying Subjects For Treatment Selection With Censored Event Time Data From A Comparative Study, Lihui Zhao, Tianxi Cai, Lu Tian, Hajime Uno, Scott D. Solomon, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Improving Statistical Analysis Of Prospective Clinical Trials In Stem Cell Transplantation. An Inventory Of New Approaches In Survival Analysis, Aurelien Latouche
Improving Statistical Analysis Of Prospective Clinical Trials In Stem Cell Transplantation. An Inventory Of New Approaches In Survival Analysis, Aurelien Latouche
COBRA Preprint Series
The CLINT project is an European Union funded project, run as a specific support action, under the sixth framework programme. It is a 2 year project aimed at supporting the European Group for Blood and Marrow Transplantation (EBMT) to develop its infrastructure for the conduct of trans-European clinical trials in accordance with the EU Clinical Trials Directive, and to facilitate International prospective clinical trials in stem cell transplantation. The initial task is to create an inventory of the existing biostatistical literature on new approaches to survival analyses that are not currently widely utilised. The estimation of survival endpoints is introduced, …
The Use Of Propensity Scores To Assess The Generalizability Of Results From Randomized Trials, Elizabeth A. Stuart, Stephen R. Cole, Catherine P. Bradshaw, Philip J. Leaf
The Use Of Propensity Scores To Assess The Generalizability Of Results From Randomized Trials, Elizabeth A. Stuart, Stephen R. Cole, Catherine P. Bradshaw, Philip J. Leaf
Johns Hopkins University, Dept. of Biostatistics Working Papers
Randomized trials remain the most accepted design for estimating the effects of interventions, but they do not necessarily answer a question of primary interest: Will the program be effective in a target population in which it may be implemented? In other words,are the results generalizable? There has been very little statistical research on how to assess the generalizability, or "external validity," of randomized trials. We propose the use of propensity-score-based metrics to quantify the similarity of the participants in a randomized trial and a target population. In this setting the propensity score model predicts participation in the randomized trial, given …
Estimating Causal Effects In Trials Involving Multi-Treatment Arms Subject To Non-Compliance: A Bayesian Frame-Work, Qi Long, Roderick J. Little, Xihong Lin
Estimating Causal Effects In Trials Involving Multi-Treatment Arms Subject To Non-Compliance: A Bayesian Frame-Work, Qi Long, Roderick J. Little, Xihong Lin
Harvard University Biostatistics Working Paper Series
No abstract provided.
Assessing Noninferiority In A Three-Arm Trial Using The Bayesian Approach, Pulak Ghosh, Farouk S. Nathoo, Mithat Gonen, Ram C. Tiwari
Assessing Noninferiority In A Three-Arm Trial Using The Bayesian Approach, Pulak Ghosh, Farouk S. Nathoo, Mithat Gonen, Ram C. Tiwari
Memorial Sloan-Kettering Cancer Center, Dept. of Epidemiology & Biostatistics Working Paper Series
Non-inferiority trials, which aim to demonstrate that a test product is not worse than a competitor by more than a pre-specified small amount, are of great importance to the pharmaceutical community. As a result, methodology for designing and analyzing such trials is required, and developing new methods for such analysis is an important area of statistical research. The three-arm clinical trial is usually recommended for non-inferiority trials by the Food and Drug Administration (FDA). The three-arm trial consists of a placebo, a reference, and an experimental treatment, and simultaneously tests the superiority of the reference over the placebo along with …
Nonparametric Regression With Missing Outcomes Using Weighted Kernel Estimating Equations, Lu Wang, Andrea Rotnitzky, Xihong Lin
Nonparametric Regression With Missing Outcomes Using Weighted Kernel Estimating Equations, Lu Wang, Andrea Rotnitzky, Xihong Lin
Harvard University Biostatistics Working Paper Series
No abstract provided.
Utilizing The Integrated Difference Of Two Survival Functions To Quantify The Treatment Contrast For Designing, Monitoring And Analyzing A Comparative Clinical Study, Lihui Zhao, Lu Tian, Hajime Uno, Scott D. Solomon, Marc A. Pfeffer, J. S. Schindler, L. J. Wei
Utilizing The Integrated Difference Of Two Survival Functions To Quantify The Treatment Contrast For Designing, Monitoring And Analyzing A Comparative Clinical Study, Lihui Zhao, Lu Tian, Hajime Uno, Scott D. Solomon, Marc A. Pfeffer, J. S. Schindler, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Efficient Design And Inference For Multi-Stage Randomized Trials Of Individualized Treatment Policies, Ree Dawson, Philip W. Lavori
Efficient Design And Inference For Multi-Stage Randomized Trials Of Individualized Treatment Policies, Ree Dawson, Philip W. Lavori
COBRA Preprint Series
Increased clinical interest in individualized ‘adaptive’ treatment policies has shifted the methodological focus for their development from the analysis of naturalistically observed strategies to experimental evaluation of a pre-selected set of strategies via multi-stage designs. Because multi-stage studies often avoid the ‘curse of dimensionality’ inherent in uncontrolled studies, and hence the need to parametrically smooth trial data, it is not surprising in this context to find direct connections among different methodological approaches. We show by asymptotic and algebraic proof that the maximum likelihood (ML) and optimal semi-parametric estimators of the mean of a treatment policy and its standard error are …
Graphical Procedures For Evaluating Overall And Subject-Specific Incremental Values From New Predictors With Censored Event Time Data, Hajime Uno, Tianxi Cai, Lu Tian, L. J. Wei
Graphical Procedures For Evaluating Overall And Subject-Specific Incremental Values From New Predictors With Censored Event Time Data, Hajime Uno, Tianxi Cai, Lu Tian, L. J. Wei
Harvard University Biostatistics Working Paper Series
No abstract provided.
Exploring The Benefits Of Adaptive Sequential Designs In Time-To-Event Endpoint Settings, Sarah C. Emerson, Kyle Rudser, Scott S. Emerson
Exploring The Benefits Of Adaptive Sequential Designs In Time-To-Event Endpoint Settings, Sarah C. Emerson, Kyle Rudser, Scott S. Emerson
UW Biostatistics Working Paper Series
Sequential analysis is frequently employed to address ethical and financial issues in clinical trials. Sequential analysis may be performed using standard group sequential designs, or, more recently, with adaptive designs that use estimates of treatment effect to modify the maximal statistical information to be collected. In the general setting in which statistical information and clinical trial costs are functions of the number of subjects used, it has yet to be established whether there is any major efficiency advantage to adaptive designs over traditional group sequential designs. In survival analysis, however, statistical information (and hence efficiency) is most closely related to …
A Bayesian Shrinkage Model For Incomplete Longitudinal Binary Data With Application To The Breast Cancer Prevention Trial, C. Wang, M.J. Daniels, Daniel O. Scharfstein, S. Land
A Bayesian Shrinkage Model For Incomplete Longitudinal Binary Data With Application To The Breast Cancer Prevention Trial, C. Wang, M.J. Daniels, Daniel O. Scharfstein, S. Land
Johns Hopkins University, Dept. of Biostatistics Working Papers
We consider inference in randomized studies, in which repeatedly measured outcomes may be informatively missing due to drop out. In this setting, it is well known that full data estimands are not identified unless unverified assumptions are imposed. We assume a non-future dependence model for the drop-out mechanism and posit an exponential tilt model that links non-identifiable and identifiable distributions. This model is indexed by non-identified parameters, which are assumed to have an informative prior distribution, elicited from subject-matter experts. Under this model, full data estimands are shown to be expressed as functionals of the distribution of the observed data. …
Application Of Time-To-Event Methods In The Assessment Of Safety In Clinical Trials, Kelly L. Moore, Mark J. Van Der Laan
Application Of Time-To-Event Methods In The Assessment Of Safety In Clinical Trials, Kelly L. Moore, Mark J. Van Der Laan
U.C. Berkeley Division of Biostatistics Working Paper Series
Since randomized controlled trials (RCT) are typically designed and powered for efficacy rather than safety, power is an important concern in the analysis of the effect of treatment on the occurrence of adverse events (AE). These outcomes are often time-to-event outcomes which will naturally be subject to right-censoring due to early patient withdrawals. In the analysis of the treatment effect on such an outcome, gains in efficiency, and thus power, can be achieved by exploiting covariate information. We apply the targeted maximum likelihood methodology to the estimation of treatment specific survival at a fixed end point for right-censored survival outcomes. …
Relaxing Latent Ignorability In The Itt Analysis Of Randomized Studies With Missing Data And Noncompliance, L Taylor, Xiao-Hua Zhou
Relaxing Latent Ignorability In The Itt Analysis Of Randomized Studies With Missing Data And Noncompliance, L Taylor, Xiao-Hua Zhou
UW Biostatistics Working Paper Series
Abstract: In this paper we consider the problem in causal inference of estimating the local complier average causal effect (CACE) parameter in the setting of a randomized clinical trial with a binary outcome, cross-over noncompliance, and unintentional missing data on the responses. We focus on the development of a moment estimator that relaxes the assumption of latent ignorability and incorporates sensitivity parameters that represent the relationship between potential outcomes and associated potential response indicators. If conclusions are insensitive over a range of logically possible values of the sensitivity parameters, then the number of interpretations of the data is reduced, and …