Analyzing incomplete discrete longitudinal clinical trial data
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Abstract: Commonly used methods to analyze incomplete longitudinal clinical trial data include complete case analysis (CC) and last observation carried forward (LOCF). However, such methods rest on strong assumptions, including missing completely at random (MCAR) for CC and unchanging profile after dropout for LOCF. Such assumptions are too strong to generally hold. Over the last decades, a number of full longitudinal data analysis methods have become available, such as the linear mixed model for Gaussian outcomes, that are valid under the much weaker missing at random (MAR) assumption. Such a method is useful, even if the scientific question is in terms of a single time point, for example, the last planned measurement occasion, and it is generally consistent with the intention-to-treat principle. The validity of such a method rests on the use of maximum likelihood, under which the missing data mechanism is ignorable as soon as it is MAR. In this paper, we will focus on non-Gaussian outcomes, such as binary, categorical or count data. This setting is less straightforward since there is no unambiguous counterpart to the linear mixed model. We first provide an overview of the various modeling frameworks for non-Gaussian longitudinal data, and subsequently focus on generalized linear mixed-effects models, on the one hand, of which the parameters can be estimated using full likelihood, and on generalized estimating equations, on the other hand, which is a nonlikelihood method and hence requires a modification to be valid under MAR. We briefly comment on the position of models that assume missingness not at random and argue they are most useful to perform sensitivity analysis. Our developments are underscored using data from two studies. While the case studies feature binary outcomes, the methodology applies equally well to other discrete-data settings, hence the qualifier ``discrete in the title.
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Cites work
- A comparison of the random-effects pattern mixture model with last-observation-carried-forward(locf) analysis in longitudinal clinical trials with dropouts
- A local influence approach applied to binary data from a psychiatric study
- A note on the comparison of pseudo-likelihood and generalized estimating equations for marginally specified odds ratio models with exchangeable association structure
- A Saturated Model for Analyzing Exchangeable Binary Data: Applications to Clinical and Developmental Toxicity Studies
- Analysis of Semiparametric Regression Models for Repeated Outcomes in the Presence of Missing Data
- Analyzing incomplete longitudinal clinical trial data
- Approximate Inference in Generalized Linear Mixed Models
- Assessing Response Profiles from Incomplete Longitudinal Clinical Trial Data Under Regulatory Considerations
- Conditional Log-Linear Models for Analyzing Categorical Panel Data
- (SOFTWARE) Fahrmeir
- Frequentist Model Average Estimators
- Generalized linear mixed models a pseudo-likelihood approach
- scientific article; zbMATH DE number 3169076 (Why is no real title available?)
- scientific article; zbMATH DE number 3945130 (Why is no real title available?)
- scientific article; zbMATH DE number 48904 (Why is no real title available?)
- scientific article; zbMATH DE number 3567782 (Why is no real title available?)
- scientific article; zbMATH DE number 1990012 (Why is no real title available?)
- scientific article; zbMATH DE number 2140075 (Why is no real title available?)
- scientific article; zbMATH DE number 1834445 (Why is no real title available?)
- scientific article; zbMATH DE number 1865743 (Why is no real title available?)
- scientific article; zbMATH DE number 1873785 (Why is no real title available?)
- scientific article; zbMATH DE number 795293 (Why is no real title available?)
- Inference and missing data
- Likelihood based frequentist inference when data are missing at random
- Linear mixed models for longitudinal data
- Logistic Regression for Correlated Binary Data
- Longitudinal data analysis using generalized linear models
- Marginal Modeling of Correlated Ordinal Data Using a Multivariate Plackett Distribution
- Model-based Inference for Categorical Survey Data Subject to Non-ignorable Non-response
- Models for discrete longitudinal data.
- Multivariate statistical modelling based on generalized linear models.
- NOTE ON AN EXACT TREATMENT OF CONTINGENCY, GOODNESS OF FIT AND OTHER PROBLEMS OF SIGNIFICANCE
- Semiparametric Regression for Repeated Outcomes with Nonignorable Nonresponse
- Sensitivity analysis for incomplete categorical data
- Simultaneously Modeling Joint and Marginal Distributions of Multivariate Categorical Responses
- Unified methods for censored longitudinal data and causality
Cited in
(26)- A simulation study comparing weighted estimating equations with multiple imputation based estimating equations for longitudinal binary data
- Different methods for handling incomplete longitudinal binary outcome due to missing at random dropout
- Longitudinal and incomplete clinical studies
- Handling non-ignorable dropouts in longitudinal data: a conditional model based on a latent Markov heterogeneity structure
- Bayesian nonparametrics for missing data in longitudinal clinical trials
- Analysis of incomplete longitudinal binary data: a combined Markov's transition and logistic model for non-ignorable missingness
- A dynamic approach for reconstructing missing longitudinal data using the linear increments model
- A Bayesian transition model for missing longitudinal binary outcomes and an application to a smoking cessation study
- Marginal Analysis of Incomplete Longitudinal Binary Data: A Cautionary Note on LOCF Imputation
- Analysis of incomplete data from a clinical trial with repeated measurements
- Analysis of Longitudinal Data with Non-Ignorable Non-Monotone Missing Values
- A Bayesian Framework for Intent-to-Treat Analysis with Missing Data
- Assessing Response Profiles from Incomplete Longitudinal Clinical Trial Data Under Regulatory Considerations
- Evaluation of statistical methods for analysis of small-sample longitudinal clinical trials with dropouts
- Longitudinal and incomplete clinical studies
- Strategies for handling missing data in longitudinal studies with questionnaires
- Longitudinal conditional models with intermittent missingness: SAS code and applications
- A Bayesian shrinkage model for incomplete longitudinal binary data with application to the breast cancer prevention trial
- A Latent‐Class Mixture Model for Incomplete Longitudinal Gaussian Data
- Analyzing incomplete longitudinal clinical trial data
- Missing data methods in longitudinal studies: a review
- Towards using a full spectrum of early clinical trial data: a retrospective analysis to compare potential longitudinal categorical models for molecular targeted therapies in oncology
- Formal and informal model selection with incomplete data
- Likelihood inference for models with unobservables: another view
- The nature of sensitivity in monotone missing not at random models
- Repeated measures proportional odds logistic regression analysis of ordinal score data in the statistical software package R
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