Statistical Methods in Medical Research

Papers
(The TQCC of Statistical Methods in Medical Research is 4. 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 2021-03-01 to 2025-03-01.)
ArticleCitations
A novel rare variants association test for binary traits in family-based designs via copulas91
Reference ranges: Why tolerance intervals should not be used. Comment on Liu, Bretz and Cortina-Borja, Reference range: Which statistical intervals to use? SMMR, 2021,Vol. 30(2) 523–53462
Standardization of continuous and categorical covariates in sparse penalized regressions55
Covariate adjustment in Bayesian adaptive randomized controlled trials50
Leveraging historical data to optimize the number of covariates and their explained variance in the analysis of randomized clinical trials.44
Logistic regression with correlated measurement error and misclassification in covariates32
Copula modeling for the estimation of measures of marker classification and predictiveness performance with survival outcomes29
Multivariate generalized linear mixed models for continuous bounded outcomes: Analyzing the body fat percentage data25
The staircase cluster randomised trial design: A pragmatic alternative to the stepped wedge25
Regression analysis of a future state entry time distribution conditional on a past state occupation in a progressive multistate model24
Clustering minimal inhibitory concentration data through Bayesian mixture models: An application to detect Mycobacterium tuberculosis resistance mutations23
Estimating the sample mean and standard deviation from order statistics and sample size in meta-analysis22
Unbiased and robust analysis of co-localization in super-resolution images19
Evaluating Bayesian adaptive randomization procedures with adaptive clip methods for multi-arm trials18
Optimal study designs for cluster randomised trials: An overview of methods and results17
Statistical methods for analysis of combined biomarker data from multiple nested case–control studies17
Semiparametric generalized estimating equations for repeated measurements in cross-over designs17
A general consonance principle for closure tests based on p-values17
A novel model-checking approach for dose-response relationships17
Bayesian multiple Gaussian graphical models for multilevel variables from unknown classes16
Semiparametric copula method for semi-competing risks data subject to interval censoring and left truncation: Application to disability in elderly16
Using dichotomized survival data to construct a prior distribution for a Bayesian seamless Phase II/III clinical trial15
Analysis of hospital readmissions with competing risks15
Exact interval estimation for the linear combination of binomial proportions15
Simultaneous inference procedures for the comparison of multiple characteristics of two survival functions14
Estimating target population treatment effects in meta-analysis with individual participant-level data14
Accounting for regression to the mean under the bivariate t-distribution13
Equivalence tests for ratio of means in bioequivalence studies under crossover design13
Heterogeneous treatment effect estimation for observational data using model-based forests12
Variance partitioning in spatio-temporal disease mapping models12
Point estimation following a two-stage group sequential trial12
Sample sizes required to estimate the protective efficacy of a vaccine when there is an unequal allocation of individuals across the vaccine and placebo groups11
A model-based approach for historical borrowing, with an application to neovascular age-related macular degeneration11
A unified approach to power and sample size determination for log-rank tests under proportional and nonproportional hazards11
A Bayesian beta-binomial piecewise growth mixture model for longitudinal overdispersed binomial data11
Sensitivity analysis for unmeasured confounding in estimating the difference in restricted mean survival time11
Matching ratio and sample size for optimal sequential testing with binomial data11
Time-varying coefficient additive hazards model with latent variables10
Impact of unequal censoring and insufficient follow-up on comparing survival outcomes: Applications to clinical studies10
A comparison of two frameworks for multi-state modelling, applied to outcomes after hospital admissions with COVID-1910
Variance estimation for the average treatment effects on the treated and on the controls9
Nonparametric Bayesian Q-learning for optimization of dynamic treatment regimes in the presence of partial compliance9
Joint regression analysis of clustered current status data with latent variables9
Generalized fiducial inference for the restricted mean survival time8
Partly linear single-index cure models with a nonparametric incidence link function8
A latent class linear mixed model for monotonic continuous processes measured with error8
Simulation extrapolation method for measurement error: A review8
Group lasso priors for Bayesian accelerated failure time models with left-truncated and interval-censored data8
Sample size determination for stepped wedge cluster randomized trials in pragmatic settings8
Bayesian analysis of joint quantile regression for multi-response longitudinal data with application to primary biliary cirrhosis sequential cohort study8
Editorial8
Goodness-of-fit tests for modified Poisson regression possibly producing fitted values exceeding one in binary outcome analysis8
Regression analysis of longitudinal data with random change point8
Robust integration of secondary outcomes information into primary outcome analysis in the presence of missing data7
A review of multistate modelling approaches in monitoring disease progression: Bayesian estimation using the Kolmogorov-Chapman forward equations7
Model-based optimal randomization procedure for treatment–covariate interaction tests7
Demystifying estimands in cluster-randomised trials7
A generalized epidemiological model with dynamic and asymptomatic population7
Comparison of statistical methods for the analysis of patient-reported outcomes in randomised controlled trials: A simulation study7
Instrumental variable analysis with categorical treatment7
Omnibus test for restricted mean survival time based on influence function7
Confidence estimation based on data from independent studies7
Estimating drug concentration–response relationships by applying causal inference methods for continuous point exposures and time-to-event outcomes7
Adaptive enrichment trial designs using joint modelling of longitudinal and time-to-event data7
Bivariate copula regression models for semi-competing risks6
Design and analysis of factorial clinical trials: The impact of one treatment's effectiveness on the statistical power and required sample size of the other6
Refined moderation analysis with binary outcomes in precision medicine research6
ROSIE: RObust Sparse ensemble for outlIEr detection and gene selection in cancer omics data6
A new cure model accounting for longitudinal data and flexible patterns of hazard ratios over time6
A matching-based machine learning approach to estimating optimal dynamic treatment regimes with time-to-event outcomes6
Comparison between inverse-probability weighting and multiple imputation in Cox model with missing failure subtype6
Commentary on the use of the reproduction number R during the COVID-19 pandemic6
The “Why” behind including “Y” in your imputation model6
Frequentist rules for regulatory approval of subgroups in phase III trials: A fresh look at an old problem6
Solutions to problems of nonexistence of parameter estimates and sparse data bias in Poisson regression6
Interval estimation in three-class receiver operating characteristic analysis: A fairly general approach based on the empirical likelihood6
Bayesian blockwise inference for joint models of longitudinal and multistate data with application to longitudinal multimorbidity analysis6
Non-parametric treatment time-lag effect estimation6
Weighted reverse counting process (WRCP): A novel approach to quantify the overall treatment effect with multiple time-to-event outcomes by adaptive weighting6
Estimating latent, dynamic processes of breast cancer tumour growth and distant metastatic spread from mammography screening data6
Design and analysis of partially randomized preference trials with propensity score stratification6
Combining cox regressions across a heterogeneous distributed research network facing small and zero counts6
l2-Penalized temporal logit-mixed models for the estimation of regional obesity prevalence over time5
A state-space approach for longitudinal outcomes: An application to neuropsychological outcomes5
Joint meta-analysis of two diagnostic tests accounting for within and between studies dependence5
Dynamic survival analysis: Modelling the hazard function via ordinary differential equations5
Multiple imputation with missing data indicators5
Approximate Bayesian computation design for phase I clinical trials5
Model-free screening for variables with treatment interaction5
A Bayesian quasi-likelihood design for identifying the minimum effective dose and maximum utility dose in dose-ranging studies5
The change in estimate method for selecting confounders: A simulation study5
Multiple imputation for cause-specific Cox models: Assessing methods for estimation and prediction5
Random survival forests with multivariate longitudinal endogenous covariates5
A capture-recapture modeling framework emphasizing expert opinion in disease surveillance5
Nonparametric kernel estimation of the probability of cure in a mixture cure model when the cure status is partially observed5
Simulation models for aggregated data meta-analysis: Evaluation of pooling effect sizes and publication biases5
Optimal sampling allocation for outcome-dependent designs in cluster-correlated data settings5
A sequential test to compare the real-time fatality rates of a disease among multiple groups with an application to COVID-19 data5
A competing risks model with binary time varying covariates for estimation of breast cancer risks in BRCA1 families5
Glucodensities: A new representation of glucose profiles using distributional data analysis5
A functional proportional hazard cure rate model for interval-censored data5
Detecting the skewness of data from the five-number summary and its application in meta-analysis5
Goodness-of-fit tests for a logistic regression model with missing covariates5
Promising zone two-stage design for a single-arm study with binary outcome5
Weighted functional linear Cox regression model5
Regularization approaches in clinical biostatistics: A review of methods and their applications5
Use of clinical tolerance limits for assessing agreement5
Mediation effects that emulate a target randomised trial: Simulation-based evaluation of ill-defined interventions on multiple mediators5
Improved semi-parametric inference for a mixture model of responses from a control versus treatment group trial5
A new measure to quantify sedentary behavior using accelerometer data: Application to the Hispanic Community Health Study/Study of Latinos5
Joint modeling of zero-inflated longitudinal measurements and time-to-event outcomes with applications to dynamic prediction5
Improving transportability of randomized controlled trial inference using robust prediction methods5
Correlational analyses of biomarkers that are harmonized through a bridging study due to measurement errors5
Challenges of modelling approaches for network meta-analysis of time-to-event outcomes in the presence of non-proportional hazards to aid decision making: Application to a melanoma network5
Imputation and missing indicators for handling missing data in the development and deployment of clinical prediction models: A simulation study5
A multi-state Markov model using notification data to estimate HIV incidence, number of undiagnosed individuals living with HIV, and delay between infection and diagnosis: Illustration in France, 20084
Data-driven clustering of infectious disease incidence into age groups4
Risk difference tests for stratified binary data under Dallal’s model4
New clinical trial design borrowing information across patient subgroups based on fusion-penalized regression models4
Quantile outcome adaptive lasso: Covariate selection for inverse probability weighting estimator of quantile treatment effects4
An overview of optimal designs under a given budget in cluster randomized trials with a binary outcome4
Additive hazards model with time-varying coefficients and imaging predictors4
Adaptive group sequential survival comparisons based on log-rank and pointwise test statistics4
Reducing the false discovery rate of preclinical animal research with Bayesian statistical decision criteria4
Nonparametric analysis of doubly truncated and interval-censored data4
Modeling and estimating a threshold effect: An application to improving cardiac surgery practices4
Long-term Dagum-power variance function frailty regression model: Application in health studies4
Repeated measures discriminant analysis using multivariate generalized estimation equations4
Using joint models for longitudinal and time-to-event data to investigate the causal effect of salvage therapy after prostatectomy4
Estimation of covariate effects on net survivals in the relative survival progressive illness-death model4
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 preference4
Role of calibration in uncertainty-based referral for deep learning4
Weighted mean difference statistics for paired data in the presence of missing values4
Using a centered general linear model for detection of interactions among biomarkers4
Ranking procedures for repeated measures designs with missing data: Estimation, testing and asymptotic theory4
Distribution-free control charts for mixed-type data based on rank of interpoint distances4
Comparisons of various estimates of the I2 statistic for quantifying between-study heterogeneity in meta-analysis4
MEGH: A parametric class of general hazard models for clustered survival data4
An additive-multiplicative model for longitudinal data with informative observation times4
A Bayesian hierarchical model for disease mapping that accounts for scaling and heavy-tailed latent effects4
A structured iterative division approach for non-sparse regression models and applications in biological data analysis4
Average treatment effect on the treated, under lack of positivity4
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