Journal of Biopharmaceutical Statistics

Article	Citations
Heterogeneous logistic regression for estimation of subgroup effects on hypertension	27
Introduction to the special issue Advances in statistical methods for the assessment of patient-centered outcomes	21
An adaptive seamless 2-in-1 design with biomarker-driven subgroup enrichment	18
A practical analysis procedure on generalizing comparative effectiveness in the randomized clinical trial to the real-world trial-eligible population	17
Quasi-Empirical Bayes methods for parameter estimation involving many small samples	14
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	12
Comparison of profile-likelihood-based confidence intervals with other rank-based methods for the two-sample problem in ordered categorical data	12
In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f 2	11
A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions	10
Whole-cage randomization for animal studies with unequal cage or group sizes	10
Different view of the diagnostics test accuracy measures and optimal cut-off point selection procedure under tree or umbrella ordering	10
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	10
Optimal sample size determination for single-arm trials in pediatric and rare populations with Bayesian borrowing	9
A simulation study comparing backfill and randomized expansion in oncology dose optimization	9
Using surrogate information to improve confirmatory platform trial with sample size re-estimation	9
Statistical innovation for next generation pharmaceutical development	8
Non-constant mean relative potency for antibody-dependent cellular cytotoxicity assays	8
A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	8
Analysis of innovative two-stage seamless adaptive design with different endpoints and population shift	8
Estimand in benefit-risk assessment	8
An enriched approach to combining high-dimensional genomic and low-dimensional phenotypic data	8
Propensity score-integrated approach to survival analysis: leveraging external evidence in single-arm studies	8
Optimization of EWOC principle in BLRM design for phase 1 oncology trials	7
Enhancing dose selection in phase I cancer trials: Extending the Bayesian Logistic Regression Model with non-DLT adverse events integration	7
Strategies for successful dose optimization in oncology drug development: a practical guide	7

Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects	7
Machine learning approach for detection of MACE events within clinical trial data	7
Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect	7
Adjusted win ratio using the inverse probability of treatment weighting	7
Evaluating bias in the anchor method for the minimal clinically important difference: a simulation approach	7
Case study using RWD in the context of a pivotal trial for regulatory approval in a rare disease	7
Defining optimal cut-off points for multiple class ROC analysis: generalization of the Index of Union method	7
Implementation of statistical features of a Bayesian two-armed responsive adaptive randomization trial with post hoc analysis of time trend drift	7
PMED: Optimal Bayesian Platform Trial Design with Multiple Endpoints	6
Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect	6
MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design	6
Comparison of continuous, binary, and ordinal endpoints	6
Optimal designs for phase II clinical trials with heterogeneous patient populations	6
Special issue on dose optimization	5
Survival stacking with multiple data types using pseudo-observation-based-AUC loss	5
Linear regression models for analyzing the covariate-adjusted Youden index and associated cut-off points in three diagnostic groups	5
Sample size determination for a study with variable follow-up time	5
Bayesian analysis of the causal reference-based model for missing data in clinical trials	5
A structured framework for adaptively incorporating external evidence in sequentially monitored clinical trials	5
FDA experiences with a centralized statistical monitoring tool	5
Double machine learning methods for estimating average treatment effects: a comparative study	5
Joint comparison of the predictive values of multiple binary diagnostic tests: an extension of McNemar’s test	5
Estimation of median survival time and its 95% confidence interval using SAS PROC LIFETEST	5
Analysis of continuous monitoring device data	5
genRCT: a statistical analysis framework for generalizing RCT findings to real-world population	5
Bayesian screening for feature selection	5
Guest editors’ note on special issue on application of estimand	5
Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease	5
Using Bayesian hierarchical models for controlled post hoc subgroup analysis of clinical trials: application to smoking cessation treatment in American Indians and Alaska Natives	4
Conditional power in vaccine trials with seasonal variations	4
Clustering plasma concentration-time curves: applications ofunsupervised learning in pharmacogenomics	4
DOD-Combo: Bayesian dose finding design in combination trials with meta-analytic-predictive prior	4
Isotonic design for phase I cancer clinical trials with late-onset toxicities	4
The use of real-world data for clinical investigation of effectiveness in drug development	4
The impact of different data handling strategies in exploratory and confirmatory factor analysis of diary measures: an evaluation using simulated and real-world asthma nighttime symptoms diary data	4
Assessing the use of GEE methods for analyzing binary outcomes in family studies: the Strong Heart Family Study	4
Assessing the incidence and severity of drug adverse events: a Bayesian hierarchical cumulative logit model	4
Retrieved-Dropout-Based multiple imputation for time-to-event data in cardiovascular outcome trials	4
P-values and confidence intervals for weighted log-rank tests under truncated binomial design based on clustered medical data	4
Combination MCP-Mod for two-drug combination dose-ranging studies	4
Recovery of overall survival information from treatment switching in oncology trials using multiple imputation	4
Bayesian method for comparing F1 scores in the absence of a gold standard	4
Investigating pharmacokinetic profiles of Centella asiatica using machine learning and PBPK modelling	4
Lessons learned in the Apple Heart Study and implications for the data management of future digital clinical trials	4
Robust time selection for interim analysis in the Bayesian phase 2 exploratory clinical trial	3
Evaluating bias reduction methods in binary Emax model for reliable dose-response estimation	3
A win ratio approach for comparing crossing survival curves in clinical trials	3
Adaptive two-stage seamless sequential design for clinical trials	3
Guest editors’ note on the special issue International Chinese Statistical Association (ICSA) Applied Statistics Symposium 2021	3
A systematic approach to adaptive sequential design for clinical trials: using simulations to select a design with desired operating characteristics	3

Establishment of RWS guidance reflecting contributions of China to regulatory science	3
Assessment of nonlinear dose–response relationships via nonparametric regression	3
Emerging clinical initiatives in pharmaceutical development: methodology and regulatory perspectives	3
Common risk difference and corresponding confidence interval adjusted for multiple factors – comparison of seven different methods	3
BOP2-FE: Bayesian optimal phase II design with futility and efficacy-stopping boundaries	3
Real world data (RWD) in pediatrics	3
Step-down multiple testing procedures for the overall population and partitioning subgroups	3
Emerging insights and commentaries – MMRM vs LOCF	3
Dynamic incorporation of real world evidence within the framework of adaptive design	3
Applications of correlative analysis for chemistry, manufacturing and controls in cell and gene therapy	3
Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	3
Enhanced doubly robust estimation in clinical trials accounting for intercurrent events	3
Change-point detection in Weibull-accelerated failure time models via narrowest significance pursuit	3
Group sequential multi-arm multi-stage survival trial design with treatment selection	3
Reflections on estimands for patient-reported outcomes in cancer clinical trials	3
On F β -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy	3
Random intercept hierarchical linear model for multi-regional clinical trials	3
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials	2
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study	2
A randomized Bayesian optimal phase II design with binary endpoint	2
Correction	2
Statistical considerations for using tolerance interval to set product specification for normally distributed attribute	2
Assessing model accuracy using random data split: a simulation study	2
Random effect restricted mean survival time model	2
Informative event rate in study determination, study design, and interim analysis monitoring with non-proportional hazards	2
Bayesian analyses of multiple random change points in survival models with applications to clinical trials	2
Identifying the clinical relative importance of each correlated outcome variables in multivariate approach: an exploration using ACCORD trial data	2
Comparing diagnostic tests and biomarkers based on benefit-risk under tree orderings of disease classes	2
Correction	2
Group sequential designs for cancer immunotherapy trial with delayed treatment effect	2
An enhanced exact permutation rank-based inferential seamless phase 2/3 design	2
Meta-analysis application to hERG safety evaluation in clinical trials	2
Inverse probability weighted Bayesian dynamic borrowing for estimation of marginal treatment effects with application to hybrid control arm oncology studies	2
Incorporating patient-reported outcomes in dose-finding clinical trials with continuous patient enrollment	2
Sample size reestimation and Bayesian predictive probability for single-arm clinical trials with a time-to-event endpoint using Weibull distribution with unknown shape parameter	2
An improved biomarker-guided adaptive patient enrichment design for oncology trials	2
Generalized triple outcome decision-making in basket trials	2
Novel machine learning approach to differential cell flow cytometry analysis based on projection pursuit	2
Reimagining optimization of clinical trials efficiency through use of statistical innovation, technology and non-standard data sources	2
Response to Comment on “Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	2
Optimum designs for clinical trials in personalized medicine when response variance depends on treatment	2
Estimation of the selected treatment mean in two stage drop-the-losers design	2
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing	2
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US	2
Statistical methods of indirect comparison with real-world data for survival endpoint under non-proportional hazards	2
The role of regulatory flexibility in the review and approval process of rare disease drug development	2
The value of a two-armed Bayesian response adaptive randomization trial	2
Non-inferiority testing for qualitative microbiological methods:Assessing and improving the approach in USP <1223>	2
A basket trial design based on constrained hierarchical Bayesian model for latent subgroups	2
Investigating the impact of data monitoring committee recommendations on the probability of trial success	2
Use of alternative and confirmatory data in support of rare disease drug development	2
Bayesian design of clinical trials using the scale transformed power prior	2
A semi-parametric approach for time-dependent ROC curves with nonignorable missing biomarker	2
Mind the gap: Bayesian equipoise calibration of clinical trial designs	2
Estimation of treatment effects in early-phase randomized clinical trials involving external control data	2
Estimation of treatment effects in early phase randomized clinical trials involving multiple data sources for external control	2
Developing large language models to detect adverse drug events in posts on x	2
Rectangular multivariate normal prediction regions for setting reference regions in laboratory medicine	2
Confidence intervals of mean residual life function in length-biased sampling based on modified empirical likelihood	2
Bayesian Hierarchy model for population pharmacokinetics of amikacin in Japanese clinical population	1
Multi-armed bandits backfill Bayesian optimal interval design	1
Response to comment on “Transporting survival of an HIV clinical trial to the external target populations by Lee et al. (2024)”	1
A multiple imputation approach in enhancing causal inference for overall survival in randomized controlled trials with crossover	1
A bias correction method for hazard ratio estimation and its inference in a multiple-arm clinical trial	1
Statistical operating characteristics of current early phase dose finding designs with toxicity and efficacy in oncology	1
Exploring Bayesian adaptive designs in multi-arm randomized controlled trials with a patient preference arm	1
Two-stage response adaptive randomization designs for multi-arm trials with binary outcome	1
Response to Comment on ‘Statistical Consideration and Challenges in Bridging Study of Personalized Medicine’: A modified variance for sensitivity	1
Covariate adjusted dose–response curves with applications to vaccine clinical trials	1
Medical diagnostic accuracy measures: an innovative approach based on the area under predictive values curves	1
Novel 3-arm wait-list controlled trial designs together with mixed-effects analysis improve precision of treatment effect estimators	1
Win statistics (win ratio, win odds, and net benefit): Noncollapsibility and standardization for randomized clinical trials	1
Saddlepoint approximation for weighted log-rank tests based on block truncated binomial design	1
Improving power in adaptive expansion of biomarker populations in phase 3 clinical trials	1
On a Holm-related MTP for rejecting at least k hypotheses: general validity, optimality property, confidence regions, and applications	1
DOD-BART: machine learning-based dose optimization design incorporating patient-level prognostic factors via Bayesian additive regression trees	1
Bayesian model averaging of longitudinal dose-response models	1
Simultaneous comparison of the predictive values of two binary diagnostic tests in the presence of categorical covariates	1
Adaptive promising zone design for cancer immunotherapy with heterogeneous delayed treatment effect	1
Assessing predictive probability of success for future clinical trials	1

Statistical learning in preclinical drug proarrhythmic assessment	1
Multivariate equivalence and component-wise superiority tests for paired samples	1
Dissecting the restricted mean time in favor of treatment	1
Applying latent profile analysis to identify adolescents and young adults with chronic conditions at risk for poor health-related quality of life	1
A comparison of Bayesian and score methods for interval estimates of positive/negative likelihood ratios in support of diagnostic device performance evaluation	1
Direct estimation of volume under the ROC surface with verification bias	1
Statistical approaches to evaluate the positive control drug using the hERG assay	1
Application of marginal structural models for causal inference on the treatment effect for overall survival in randomized controlled trials with control arm patients switching to active intervention a	1
The proportional treatment effect: A metric that empowers and connects	1
Advancing cell and gene therapy: Application of AI/ML in clinical development and patient management	1
Statistical methodologies for absolute and relative efficacy assessment based on single-arm trials: a scoping review	1
A composite semiparametric homogeneity test for the distributions of multigroup interval-bounded longitudinal data	1
Joint model for repeated measurements and competing risks data using flexible shared random effects	1
Transporting survival of an HIV clinical trial to the external target populations	1
Comments on ”Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	1
RESTART trial design: two-stage seamless transition design with operational considerations	1
Interval estimation of relative risks for combined unilateral and bilateral correlated data	1
Defective regression models for cure rate data with competing risks	1
Overview of real-world applications of federated learning with NVIDIA FLARE	1
Large-scale dependent multiple testing via higher-order hidden Markov models	1
Drug safety assessment by machine learning models	1
Mitigating propensity score model misspecification with multiply robust weights when leveraging external data	1
Bayesian optimal Phase II survival trial design with event-driven approach	1
Correction	1
Demystifying stabilization in inverse probability of treatment weighting	1
Issues in cox proportional hazards model with unequal randomization	1
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	1
Epistemic uncertainty in Bayesian predictive probabilities	1
A Bayesian three-tier quantitative decision-making framework for single arm studies in early phase oncology	1
Approximate Bayesian estimation of time to clinical benefit using Frequentist approaches: an application to an intensive blood pressure control trial	1
Inferential properties with a novel two parameter Poisson generalized Lindley distribution with regression and application to INAR(1) process	1
Biomarker-driven basket trial designs: origins and new methodological developments	1
Reweighting estimators to extend the external validity of clinical trials: methodological considerations	1
Optimal dose selection in phase I/II dose finding trial with contextual bandits: a case study and practical recommendations	1
Bayesian meta-analysis for rare outcomes	1
A promising subgroup identification method based on a genetic algorithm for censored survival data	1
Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time	1
BPED: A Bayesian basket design for pediatric trials with external data	1
Defective 3-parameter Gompertz model with frailty term for estimating cure fraction in survival data	1
Applied Meta-Analysis with R and Stata, 2nd Edition	1
Implementation of the ICH E9 (R1) addendum in vaccine efficacy studies: the hypothetical and principal stratum strategies	1
Reverse graphical approaches for multiple test procedures	1
Bayesian design of clinical trials with multiple time-to-event outcomes subject to functional cure	1
Improved confidence estimation for the binomial proportion with applications to clinical studies	1
Covariate-adjusted value-guided subgroup identification via boosting	1
Consideration of the adaptive randomization allocation ratio in the presence of treatment group heteroscedasticity in clinical trials	1
Multi-arm multi-stage survival trial design with arm-specific stopping rule	1
Propensity score stratified MAP prior and posterior inference for incorporating information across multiple potentially heterogeneous data sources	1
Miettinen and Nurminen score statistics revisited	1
Power priors and type I error control: constrained borrowing of external control data	1
The generalized order statistics arising from three populations with the lower truncated proportional hazard rate models and its application to the sensitivity to the early disease stage	1
A geometrically intuitive framework for the synthesis method in non-inferiority trials	1
TITE-STEIN: Time-to-event simple toxicity and efficacy interval design to accelerate phase I/II trials	1
Optimal two-phase sampling for comparing correlated areas under the ROC curves of two screening tests in the presence of verification bias	1
Bias and Type I error Control in Correcting Treatment Effect for Treatment Switching Using Marginal Structural Models in Phase III Oncology Trials	1
Robust estimates of regional treatment effects in multiregional randomized clinical trials with ordinal responses	1
Determining the late effect parameter in the Fleming-Harrington test using asymptotic relative efficiency in cancer immunotherapy clinical trials	1
Multi-stage dose expansion cohort (MSDEC) design with Bayesian stopping rule	1
Bayesian hierarchical model for dose-finding trial incorporating historical data	1
Joint linear and nonlinear mixed effects model with random change points for left-censored longitudinal data: application to HIV surveillance	1