Statistical Methods in Medical Research

Papers
(The median citation count of Statistical Methods in Medical Research is 1. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2022-08-01 to 2026-08-01.)
ArticleCitations
Exact interval estimation for the linear combination of binomial proportions264
Confidence estimation based on data from independent studies164
Joint meta-analysis of two diagnostic tests accounting for within and between studies dependence80
Investigations of sharp bounds for causal effects under selection bias56
A new cure model accounting for longitudinal data and flexible patterns of hazard ratios over time51
Covariate adjustment in Bayesian adaptive randomized controlled trials46
A Bayesian beta-binomial piecewise growth mixture model for longitudinal overdispersed binomial data46
Implementing response-adaptive designs when responses are missing: Impute or ignore?45
Semiparametric copula method for semi-competing risks data subject to interval censoring and left truncation: Application to disability in elderly41
Omnibus test for restricted mean survival time based on influence function33
Interval estimation in three-class receiver operating characteristic analysis: A fairly general approach based on the empirical likelihood29
Modeling and estimating a threshold effect: An application to improving cardiac surgery practices27
Bayesian feature selection in joint models with application to a cardiovascular disease cohort study27
A family of Bayesian prognostic and predictive covariate-adjusted response-adaptive randomization designs22
New clinical trial design borrowing information across patient subgroups based on fusion-penalized regression models20
Generalised pairwise comparisons for trend: An extension to the win ratio and win odds for dose-response and prognostic variable analysis with arbitrary statements of outcome preference19
Efficient design of partially nested randomized trials: A maximin approach18
Correlational analyses of biomarkers that are harmonized through a bridging study due to measurement errors18
A capture-recapture modeling framework emphasizing expert opinion in disease surveillance16
Analyzing heterogeneity in biomarker discriminative performance through partial time-dependent receiver operating characteristic curve modeling15
Accounting for informative observation process in transition models of binary longitudinal outcome: Application to medical record data15
Using shrinkage methods to estimate treatment effects in overlapping subgroups in randomized clinical trials with a time-to-event endpoint14
Random survival forests with multivariate longitudinal endogenous covariates14
A new information integration framework for complex models with applications to real-world data14
Multiplicative versus additive modelling of causal effects using instrumental variables for survival outcomes – a comparison14
On flexible inverse probability of treatment and intensity weighting: Informative censoring, variable selection, and weight trimming14
Simultaneous confidence intervals for an extended Koch-Röhmel design in three-arm non-inferiority trials13
Generalized Bayesian kernel machine regression13
Familywise error rate control for block response-adaptive randomization13
Quantile inference for multivariate response regression in joint modeling of longitudinal and survival data13
Competing risks models with two time scales12
A unified approach based on multidimensional scaling for calibration estimation in survey sampling with qualitative auxiliary information12
Evaluating prognostic biomarkers for survival outcomes subject to informative censoring12
Covariate selection for optimizing balance with an innovative adaptive randomization approach12
Smoothing Lexis diagrams using kernel functions: A contemporary approach11
Penalized variable selection in multi-parameter regression survival modeling11
A simple and robust parametric shared frailty model for recurrent events with the competing risk of death: An application to the Carvedilol Prospective Randomized Cumulative Survival trial11
An iterative matrix uncertainty selector for high-dimensional generalized linear models with measurement errors11
A Bayesian genomic selection approach incorporating prior feature ordering and population structures with application to coronary artery disease10
Quantifying proportion of treatment effect by surrogate endpoint under heterogeneity10
Empowering classification for multivariate functional data with simultaneous feature selection10
Bayesian order constrained adaptive design for phase II clinical trials evaluating subgroup-specific treatment effect10
Quantification of the influence of risk factors with application to cardiovascular diseases in subjects with type 1 diabetes10
The effect of estimating prevalences on the population-wise error rate9
Linearized maximum rank correlation estimation of doubly truncated data9
Hazard-based distributional regression via ordinary differential equations9
Adjusting for switches to multiple treatments: Should switches be handled separately or combined?9
Model-based adaptive randomization procedures for heteroscedasticity of treatment responses9
Modified Brier score for evaluating prediction accuracy for binary outcomes9
Complete effect decomposition for an arbitrary number of multiple ordered mediators with time-varying confounders: A method for generalized causal multi-mediation analysis8
Review of sample size determination methods for the intraclass correlation coefficient in the one-way analysis of variance model8
A generalization of moderated statistics to data adaptive semiparametric estimation in high-dimensional biology8
Improved survival analysis with shrinkage Kibria–Lukman estimators in the Cox model: Application to lung cancer data8
Sample size calculation for multi-arm parallel design with restricted mean survival time8
Change plane model averaging for subgroup identification8
Testing for marginal covariate effect when the subgroup size induced by the covariate is informative8
More efficient estimators of the area under the receiver operating characteristic curve in paired ranked set sampling8
Addressing nonignorable missing data and heterogeneity in prognostic biomarker assessment8
Adaptive sample size re-estimation designs for a two-stage randomized trial with binary outcome8
A tight fit of the SIR dynamic epidemic model to daily cases of COVID-19 reported during the 2021–2022 Omicron surge in New York City: A novel approach8
qTPI: A quasi-toxicity probability interval design for phase I trials with multiple-grade toxicities8
Estimand-based inference in the presence of long-term survivors7
Cause-specific hazard Cox models with partly interval censoring – Penalized likelihood estimation using Gaussian quadrature7
Fixed and random effect selections in generalized linear mixed models7
Combining multiple biomarkers linearly to minimize the Euclidean distance of the closest point on the receiver operating characteristic surface to the perfection corner in trichotomous settings7
Discrimination performance in illness-death models with interval-censored disease data7
Estimation and inference on the partial volume under the receiver operating characteristic surface7
Test sensitivity in a prospective cancer screening program: A critique of a common proxy measure7
Estimation in discrete time coarsened multivariate longitudinal models7
BOIN-ETC: A Bayesian optimal interval design considering efficacy and toxicity to identify the optimal dose combinations7
A practical review of response-adaptive randomization: Under-explored challenges and potential directions7
Statistical methods for clinical trials interrupted by the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic: A review7
An effective method for modeling highly correlated interaction models with applications in Alzheimer’s disease analysis7
Regression analysis of multivariate recurrent event data allowing time-varying dependence with application to stroke registry data7
Generalized fiducial inference for the restricted mean survival time6
A new framework for semi-Markovian parametric multi-state models with interval censoring6
A Bayesian latent class approach to causal inference with longitudinal data6
Interval estimation for the Youden index of a continuous diagnostic test with verification biased data6
Multiple imputation approaches for epoch-level accelerometer data in trials6
Editorial6
A generalized epidemiological model with dynamic and asymptomatic population6
Using short-term endpoints to improve interim decision making and trial duration in two-stage phase II trials with nested binary endpoints6
Local linear approximation with Laplacian smoothing penalty and application in biology6
Using circulating tumor DNA as a novel biomarker of efficacy for dose-finding designs in oncology6
Improved and computationally stable estimation of relative risk regression with one binary exposure6
Using Bayesian evidence synthesis to quantify uncertainty in population trends in smoking behaviour6
Handling missing data, skewness, and outliers in medical research: A robust factor analysis approach using the canonical fundamental skew-t distribution6
A Weibull mixture cure frailty model for high-dimensional covariates6
Time-dependent receiver operating characteristic curve estimator for correlated right-censored time-to-event data6
A hybrid prior Bayesian method for combining domestic real-world data and overseas data in global drug development5
Sample sizes required to estimate the protective efficacy of a vaccine when there is an unequal allocation of individuals across the vaccine and placebo groups5
Group lasso priors for Bayesian accelerated failure time models with left-truncated and interval-censored data5
Heterogeneous treatment effect estimation for observational data using model-based forests5
Additive hazards model with time-varying coefficients and imaging predictors5
Distribution-free control charts for mixed-type data based on rank of interpoint distances5
An overview of optimal designs under a given budget in cluster randomized trials with a binary outcome5
LASSO-type instrumental variable selection methods with an application to Mendelian randomization5
Comparison of statistical methods for the analysis of patient-reported outcomes in randomised controlled trials: A simulation study5
Adaptive enrichment trial designs using joint modelling of longitudinal and time-to-event data5
Semiparametric generalized estimating equations for repeated measurements in cross-over designs5
Bayesian analysis of joint quantile regression for multi-response longitudinal data with application to primary biliary cirrhosis sequential cohort study5
Use of clinical tolerance limits for assessing agreement5
Data-driven clustering of infectious disease incidence into age groups5
Covariate hypothesis tests for the cure rate in mixture cure models based on martingale difference correlation5
Confidence intervals and point estimates for treatment effects in adaptive enrichment designs5
Penalized estimation of general frailty Poisson models for recurrent count events5
Augmented two-stage estimation for treatment switching in oncology trials: Leveraging external data for improved precision5
Health utility adjusted survival: A composite endpoint for clinical trial designs5
Bivariate copula regression models for semi-competing risks5
The “Why” behind including “Y” in your imputation model5
Group sequential analysis of marked point processes: Plasma donation trials5
Joint estimation of multiple graphical models for an fMRI study of brain connectivity networks5
Sample size determination for adaptive crossover trial in detecting gene-drug interactions4
Efficient algorithms for survival data with multiple outcomes using the frailty model4
Comparisons of various estimates of the I2 statistic for quantifying between-study heterogeneity in meta-analysis4
Quantile outcome adaptive lasso: Covariate selection for inverse probability weighting estimator of quantile treatment effects4
Divided-and-combined omnibus test for genetic association analysis with high-dimensional data4
Causal rule ensemble method for estimating heterogeneous treatment effect with consideration of prognostic effects4
Strategies to boost statistical efficiency in randomized oncology trials with primary time-to-event endpoints4
Graphical methods to illustrate the nature of the relation between a continuous variable and the outcome when using restricted cubic splines with a Cox proportional hazards model4
Measuring the individualization potential of treatment individualization rules: Application to rules built with a new parametric interaction model for parallel-group clinical trials4
Bivariate joint models for survival and change of cognitive function4
Methods for comparative effectiveness based on time to confirmed disability progression with irregular observations in multiple sclerosis4
Regularization approaches in clinical biostatistics: A review of methods and their applications4
Vaccine efficacy estimands and power considerations4
Covariate-adjusted inference for doubly adaptive biased coin design4
Average treatment effect on the treated, under lack of positivity4
Estimating dynamic treatment regimes for ordinal outcomes with household interference: Application in household smoking cessation4
Quantifying the effects of air pollution on respiratory ill health treated in primary care when the locations of the populations at risk are partially unknown4
Simulating time-to-event data subject to competing risks and clustering: A review and synthesis4
Diagnostic accuracy analysis for multiple raters using probit hierarchical model for ordinal ratings4
Extended excess hazard models for spatially dependent survival data4
Factorial survival analysis for treatment effects under dependent censoring4
Joint model with latent disease age: Overcoming the need for reference time4
A Bayesian transformation model for informative partly interval-censored data with covariates subject to measurement error4
Biomarker-driven optimal designs for patient enrollment restriction4
Using inverse probability of censoring weighting to estimate hypothetical estimands in clinical trials: Should we implement stabilisation, and if so how?4
Restricted mean survival time in cluster randomized trials with a small number of clusters: Improving variance estimation of the intervention effect from the pseudo-values regression4
Variable selection for mixed panel count data under the proportional mean model3
Jointly assessing multiple endpoints in pilot and feasibility studies3
Improving estimation efficiency of case-cohort studies with interval-censored failure time data3
Modeling treatment effects on absorbing outcomes in clinical trials: Leveraging longitudinal and ordinal data for efficiency gains3
A natural history and copula-based joint model for regional and distant breast cancer metastasis3
Uniformization and bounded Taylor series in Newton–Raphson method improves computational performance for a multistate transition model estimation and inference3
Efficient estimation of the marginal mean of recurrent events in randomized controlled trials3
Comparison of random forest methods for conditional average treatment effect estimation with a continuous treatment3
Online control of the False Discovery Rate in group-sequential platform trials3
Multivariate contaminated normal linear mixed models applied to Alzheimer’s disease study with censored and missing data3
Adaptive aggregation for longitudinal quantile regression based on censored history process3
Response adaptive randomisation in clinical trials: Current practice, gaps and future directions3
On identification and estimation for sufficient cause interaction through a quasi-instrumental variable3
Wilcoxon rank-sum tests to detect one-sided mixture alternatives in group sequential clinical trials3
Youden index estimation based on group-tested data3
A robust Bayesian dose optimization design with backfill and randomization for phase I/II clinical trials3
Multicategory matched learning for estimating optimal individualized treatment rules in observational studies with application to a hepatocellular carcinoma study3
Cross-validation approaches for penalized Cox regression3
Informative simultaneous confidence intervals for graphical test procedures3
Paired count regressions for modeling the number of doctor consultations and non-prescribed drugs intake3
Assessing spillover effects: Handling missing outcomes in network-based studies3
Isotonic design for single-arm biomarker stratified trials3
A Bayesian hierarchical model for the analysis of visual analogue scaling tasks3
An overview of propensity score matching methods for clustered data3
An efficient approach for optimizing the cost-effective individualized treatment rule using conditional random forest3
A distribution-free smoothed combination method to improve discrimination accuracy in multi-category classification3
Hierarchical Bayesian bivariate spatial modeling of small area proportions with application to health survey data3
Nonparametric Bayesian functional selection in 1-M matched case-crossover studies3
Bayesian semiparametric joint modeling of a count outcome and inconveniently timed longitudinal predictors3
Intervention treatment distributions that depend on the observed treatment process and model double robustness in causal survival analysis3
Optimising error rates in programmes of pilot and definitive trials using Bayesian statistical decision theory3
Modified interactive Q-learning for attenuating the impact of model misspecification with treatment effect heterogeneity3
Model detection for semiparametric accelerated failure additive model with right-censored data3
Semi-parametric testing for ordinal treatment effects in time-to-event data via dynamic Dirichlet process mixtures of the inverse-Gaussian distribution3
Weighting methods for truncation by death in cluster-randomized trials3
Using information criteria to select smoothing parameters when analyzing survival data with time-varying coefficient hazard models3
Does it decay? Obtaining decaying correlation parameter values from previously analysed cluster randomised trials3
On the estimation of population size—A comparison of capture-recapture and multiplier-benchmark methods3
A jackknife approach to estimate the prediction uncertainty from binary classifiers under right-censoring3
Nonparametric change-point control charts for joint monitoring of mean and covariance with application to medical imaging data2
Maintaining the validity of inference from linear mixed models in stepped-wedge cluster randomized trials under misspecified random-effects structures2
Evaluating and testing for actionable treatment effect heterogeneity2
Flexible modeling of multiple nonlinear longitudinal trajectories with censored and non-ignorable missing outcomes2
Joint regression analysis of clustered current status data with latent variables2
Sample sizes for estimating the sensitivity of a monitoring system that generates repeated binary outcomes with autocorrelation2
Nonparametric Bayesian Q-learning for optimization of dynamic treatment regimes in the presence of partial compliance2
Bayesian sample size determination using robust commensurate priors with interpretable discrepancy weights2
Logistic regression with correlated measurement error and misclassification in covariates2
A Bayesian likely responder approach for the analysis of randomized controlled trials2
Determination of correlations in multivariate count data with informative observation times2
Dynamic prediction by landmarking with data from cohort subsampling designs2
A novel power prior approach for borrowing historical control data in clinical trials2
A note on response-adaptive randomization from a Bayesian prediction viewpoint2
Modeling the effect of longitudinal markers on left-truncated time-to-event outcomes in twin studies2
Permutation-based global rank test with adaptive weights for multiple primary endpoints2
Compositional functional regression and isotemporal substitution analysis: Methods and application in time-use epidemiology2
Incorporation of missing indicator with multiple imputation in propensity score analysis with partially observed covariates: A simulation study2
Estimation of the average treatment effect with variable selection and measurement error simultaneously addressed for potential confounders2
Cure models with adaptive activation for modeling cancer survival2
Design of egocentric network-based studies to estimate causal effects under interference2
Estimating transformations for evaluating diagnostic tests with covariate adjustment2
Weight calibration in the joint modelling of medical cost and mortality2
Efficient semi-supervised estimation of optimal individualized treatment regimes with survival outcome2
Revisiting sample size planning for receiver operating characteristic studies: A confidence interval approach with precision and assurance2
Sample size estimation for comparing dynamic treatment regimens in a SMART: A Monte Carlo-based approach and case study with longitudinal overdispersed count outcomes2
Application of marginalized zero-inflated models when mediators have excess zeroes2
A statistical framework for planning and analysing test–retest studies of repeatability2
Proportion of treatment effect explained: An overview of interpretations2
A novel rare variants association test for binary traits in family-based designs via copulas2
Permutation tests for detecting treatment effect heterogeneity in cluster randomized trials2
Instrumental variable analysis with categorical treatment2
Joint modelling of longitudinal ordinal and multi-state data2
A model-based approach for historical borrowing, with an application to neovascular age-related macular degeneration2
Saddlepoint approximation p-values of weighted log-rank tests based on censored clustered data under block Efron’s biased-coin design2
Penalized estimation for varying coefficient additive hazards models2
Smoothed simulated pseudo-maximum likelihood estimation for nonlinear mixed effects models with censored responses2
Bayesian tensor logistic regression with applications to neuroimaging data analysis of Alzheimer’s disease2
Point estimation of the 100p percent lethal dose using a novel penalised likelihood approach2
A fast integrative clustering and feature selection approach for high-dimensional multiview data2
Multiple imputation for non-monotone missing not at random data using the no self-censoring model2
Outcome adaptive propensity score methods for handling censoring and high-dimensionality: Application to insurance claims2
Propensity score weighting methods for causal subgroup analysis with time-to-event outcomes2
Misclassification simulation extrapolation method for a Weibull accelerated failure time model2
Survival analysis with a random change-point2
The performance of marginal structural models for estimating risk differences and relative risks using weighted univariate generalized linear models2
Group sequential methods based on supremum logrank statistics under proportional and nonproportional hazards2
On prior smoothing with discrete spatial data in the context of disease mapping2
Two-stage subsampling variable selection for sparse high-dimensional generalized linear models2
Estimating an adjusted risk difference in a cluster randomized trial with individual-level analyses2
Variable selection using inverse probability of censoring weighting2
Additive hazard causal model with a binary instrumental variable2
Refined moderation analysis with binary outcomes in precision medicine research2
The augmented synthetic control method in public health and biomedical research2
Methods for non-proportional hazards in clinical trials: A systematic review2
Estimation of global average treatment effect in National Heart, Lung, and Blood Institute (NHLBI) Growth and Health Study2
A matching-based machine learning approach to estimating optimal dynamic treatment regimes with time-to-event outcomes2
Predictive abilities comparison from multiple dynamic prediction models2
Partly linear single-index cure models with a nonparametric incidence link function2
Dynamic prediction of interval-censored failure time data with longitudinal marker2
Matching ratio and sample size for optimal sequential testing with binomial data2
Sensitivity bounds for bias in hazard ratios: A causal hazard perspective2
Monitoring time to event in registry data using CUSUMs based on relative survival models2
Two-stage Bayesian network meta-analysis of individualized treatment rules for multiple treatments with siloed data2
Data-dependent early completion of dose-finding trials for drug-combination2
Sensitivity analyses in longitudinal clinical trials via distributional imputation2
Statistical assessment of biomarker replicability using MAJAR method2
Model-based approach for two-stage group sequential or adaptive designs in bioequivalence studies using parallel and crossover designs2
A burn-in(g) question: How long should an initial equal randomization stage be before Bayesian response-adaptive randomization?2
Dirichlet process mixture models for regression discontinuity designs2
Cluster analysis for longitudinal data and its application in the detection of adiposity trajectories2
Boosting distributional copula regression for bivariate binary, discrete and mixed responses2
An augmented likelihood approach for the Cox proportional hazards model with interval-censored auxiliary and validated outcome data—with application to the Hispanic Community Health Study/Study of Lat2
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