Journal of Biopharmaceutical Statistics

Article	Citations
Introduction to the special issue Advances in statistical methods for the assessment of patient-centered outcomes	31
An adaptive seamless 2-in-1 design with biomarker-driven subgroup enrichment	26
A practical analysis procedure on generalizing comparative effectiveness in the randomized clinical trial to the real-world trial-eligible population	21
Quasi-Empirical Bayes methods for parameter estimation involving many small samples	20
Comparison of profile-likelihood-based confidence intervals with other rank-based methods for the two-sample problem in ordered categorical data	16
Different view of the diagnostics test accuracy measures and optimal cut-off point selection procedure under tree or umbrella ordering	12
In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f 2	12
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	12
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	10
Correction	10
A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions	10
A simulation study comparing backfill and randomized expansion in oncology dose optimization	10
A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	9
Estimand in benefit-risk assessment	9
Whole-cage randomization for animal studies with unequal cage or group sizes	9
A survey of parallelism testing methods for bioassays	8
Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects	8
Industrialization of Bayesian decision-making for proof-of-commercial-concept study designs	8
Optimization of EWOC principle in BLRM design for phase 1 oncology trials	8
Enhancing dose selection in phase I cancer trials: Extending the Bayesian Logistic Regression Model with non-DLT adverse events integration	8
Non-constant mean relative potency for antibody-dependent cellular cytotoxicity assays	8
Statistical innovation for next generation pharmaceutical development	8
Machine learning approach for detection of MACE events within clinical trial data	7
Evaluating bias in the anchor method for the minimal clinically important difference: a simulation approach	7
Implementation of statistical features of a Bayesian two-armed responsive adaptive randomization trial with post hoc analysis of time trend drift	7

Analysis of innovative two-stage seamless adaptive design with different endpoints and population shift	7
Case study using RWD in the context of a pivotal trial for regulatory approval in a rare disease	7
An enriched approach to combining high-dimensional genomic and low-dimensional phenotypic data	7
MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design	6
Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect	6
Defining optimal cut-off points for multiple class ROC analysis: generalization of the Index of Union method	6
Strategies for successful dose optimization in oncology drug development: a practical guide	6
Comparison of continuous, binary, and ordinal endpoints	6
Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect	5
genRCT: a statistical analysis framework for generalizing RCT findings to real-world population	5
Linear regression models for analyzing the covariate-adjusted Youden index and associated cut-off points in three diagnostic groups	5
Double machine learning methods for estimating average treatment effects: a comparative study	5
Adjusted win ratio using the inverse probability of treatment weighting	5
Analysis of continuous monitoring device data	5
FDA experiences with a centralized statistical monitoring tool	5
Guest editors’ note on special issue on application of estimand	5
Balancing sample size and accuracy of dose selection in phase 1 oncology trials – designs tiered by cohort size and maximum number of patients treated per dose	5
Special issue on dose optimization	5
Bayesian analysis of the causal reference-based model for missing data in clinical trials	5
Estimation of median survival time and its 95% confidence interval using SAS PROC LIFETEST	5
Recovery of overall survival information from treatment switching in oncology trials using multiple imputation	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
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
Evaluating bias reduction methods in binary Emax model for reliable dose-response estimation	4
Investigating pharmacokinetic profiles of Centella asiatica using machine learning and PBPK modelling	4
Combination MCP-Mod for two-drug combination dose-ranging studies	4
Clustering plasma concentration-time curves: applications ofunsupervised learning in pharmacogenomics	4
The use of real-world data for clinical investigation of effectiveness in drug development	4
Sample size determination for a study with variable follow-up time	4
Nonparametric testing methods based on relative effect in non-inferiority clinical trial with multiple experimental drugs	4
Change-point detection in Weibull-accelerated failure time models via narrowest significance pursuit	4
Bayesian method for comparing F1 scores in the absence of a gold standard	4
Assessing the use of GEE methods for analyzing binary outcomes in family studies: the Strong Heart Family Study	4
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
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
P-values and confidence intervals for weighted log-rank tests under truncated binomial design based on clustered medical data	4
Common risk difference and corresponding confidence interval adjusted for multiple factors – comparison of seven different methods	4
Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease	4
BOP2-FE: Bayesian optimal phase II design with futility and efficacy-stopping boundaries	3
Assessment of nonlinear dose–response relationships via nonparametric regression	3
Enhanced doubly robust estimation in clinical trials accounting for intercurrent events	3
Random intercept hierarchical linear model for multi-regional clinical trials	3
Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	3
Optimum designs for clinical trials in personalized medicine when response variance depends on treatment	3
Identifying the clinical relative importance of each correlated outcome variables in multivariate approach: an exploration using ACCORD trial data	3
Adaptive two-stage seamless sequential design for clinical trials	3
A systematic approach to adaptive sequential design for clinical trials: using simulations to select a design with desired operating characteristics	3
Applications of correlative analysis for chemistry, manufacturing and controls in cell and gene therapy	3

Sample sizes for randomized controlled trials utilizing Bayesian response adaptive randomization for continuous outcomes	3
Robust time selection for interim analysis in the Bayesian phase 2 exploratory clinical trial	3
A win ratio approach for comparing crossing survival curves in clinical trials	3
Step-down multiple testing procedures for the overall population and partitioning subgroups	3
Mind the gap: Bayesian equipoise calibration of clinical trial designs	3
Bayesian analyses of multiple random change points in survival models with applications to clinical trials	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
Real world data (RWD) in pediatrics	3
Order-of-addition in mixture and component-amount designs in orthogonal blocks	3
Establishment of RWS guidance reflecting contributions of China to regulatory science	3
Impacts of reducing the number of hERG assay concentration levels on IC50 estimation	3
Emerging insights and commentaries – MMRM vs LOCF	3
Estimation of treatment effects in early-phase randomized clinical trials involving external control data	3
Why the minimum effective dose is unidentifiable – and how a DOR→OOD standard delivers label-ready dosing in oncology	3
Group sequential multi-arm multi-stage survival trial design with treatment selection	3
Special issue: cell and gene causal inference in the design and analysis of gene therapy clinical trials	2
Use of alternative and confirmatory data in support of rare disease drug development	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
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials	2
Developing large language models to detect adverse drug events in posts on x	2
Bayesian design of clinical trials using the scale transformed power prior	2
Robust CATE estimation using novel ensemble methods	2
Correction	2
A multivariate equivalence test based on Mahalanobis distance with a data-driven margin	2
Comparison of in-vitro release rates for vaginal systems	2
Meta-analysis application to hERG safety evaluation in clinical trials	2
Comparing diagnostic tests and biomarkers based on benefit-risk under tree orderings of disease classes	2
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing	2
Reimagining optimization of clinical trials efficiency through use of statistical innovation, technology and non-standard data sources	2
Random effect restricted mean survival time model	2
Estimation of the selected treatment mean in two stage drop-the-losers design	2
A basket trial design based on constrained hierarchical Bayesian model for latent subgroups	2
Novel machine learning approach to differential cell flow cytometry analysis based on projection pursuit	2
Informative event rate in study determination, study design, and interim analysis monitoring with non-proportional hazards	2
On stepwise MTPs between Holm’s step-down MTP and the max- p -value MTP for co-primary endpoints: A further extension for free	2
Estimation of treatment effects in early phase randomized clinical trials involving multiple data sources for external control	2
Statistical approaches for shelf-life determination of a drug product	2
Non-inferiority testing for qualitative microbiological methods:Assessing and improving the approach in USP <1223>	2
Generalized triple outcome decision-making in basket trials	2
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study	2
The value of a two-armed Bayesian response adaptive randomization trial	2
An improved biomarker-guided adaptive patient enrichment design for oncology trials	2
Incorporating patient-reported outcomes in dose-finding clinical trials with continuous patient enrollment	2
Response to Comment on “Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	2
A semi-parametric approach for time-dependent ROC curves with nonignorable missing biomarker	2
Correction	1
Statistical considerations for using tolerance interval to set product specification for normally distributed attribute	1
Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time	1
DOD-BART: machine learning-based dose optimization design incorporating patient-level prognostic factors via Bayesian additive regression trees	1
The role of regulatory flexibility in the review and approval process of rare disease drug development	1
Dissecting the restricted mean time in favor of treatment	1
Non-inferiority testing for the odds ratio in two independent binomial proportions using the method of variance estimates recovery	1
Analyze paired case–control data prospectively in comparing two predictive values	1
The SMART-of-1 approach to personalized treatment trials	1
Two-stage response adaptive randomization designs for multi-arm trials with binary outcome	1
TITE-STEIN: Time-to-event simple toxicity and efficacy interval design to accelerate phase I/II trials	1
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US	1
Biomarker-driven basket trial designs: origins and new methodological developments	1
Response to comment on “Transporting survival of an HIV clinical trial to the external target populations by Lee et al. (2024)”	1
Drug safety assessment by machine learning models	1
A systematic analysis of the inherent dose uncertainty in autologous CAR T-cell products	1
Bayesian meta-analysis for rare outcomes	1
A composite semiparametric homogeneity test for the distributions of multigroup interval-bounded longitudinal data	1
Augmented match weighted estimators: new methods for estimating average treatment effects under extreme propensity scores	1
Comments on ”Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	1
A multiple imputation approach in enhancing causal inference for overall survival in randomized controlled trials with crossover	1
Exploring Bayesian adaptive designs in multi-arm randomized controlled trials with a patient preference arm	1
Optimal two-phase sampling for comparing correlated areas under the ROC curves of two screening tests in the presence of verification bias	1
Estimation of exponential rates in non-homogeneous populations using the EAIR estimator	1
Win statistics (win ratio, win odds, and net benefit): Noncollapsibility and standardization for randomized clinical trials	1
Multi-arm multi-stage survival trial design with arm-specific stopping rule	1
On a Holm-related MTP for rejecting at least k hypotheses: general validity, optimality property, confidence regions, and applications	1
Inverse probability weighted Bayesian dynamic borrowing for estimation of marginal treatment effects with application to hybrid control arm oncology studies	1
Covariate adjusted dose–response curves with applications to vaccine clinical trials	1
Statistical methodologies for absolute and relative efficacy assessment based on single-arm trials: a scoping review	1
Inferential properties with a novel two parameter Poisson generalized Lindley distribution with regression and application to INAR(1) process	1
Covariate-adjusted value-guided subgroup identification via boosting	1
Statistical operating characteristics of current early phase dose finding designs with toxicity and efficacy in oncology	1
Bayesian optimal Phase II survival trial design with event-driven approach	1

Investigating the impact of data monitoring committee recommendations on the probability of trial success	1
Correction	1
Power priors and type I error control: constrained borrowing of external control data	1
Group sequential designs for cancer immunotherapy trial with delayed treatment effect	1
Improving power in adaptive expansion of biomarker populations in phase 3 clinical trials	1
Bayesian model averaging of longitudinal dose-response models	1
Rectangular multivariate normal prediction regions for setting reference regions in laboratory medicine	1
Saddlepoint approximation for weighted log-rank tests based on block truncated binomial design	1
Mitigating propensity score model misspecification with multiply robust weights when leveraging external data	1
Multi-armed bandits backfill Bayesian optimal interval design	1
Demystifying stabilization in inverse probability of treatment weighting	1
Bayesian Hierarchy model for population pharmacokinetics of amikacin in Japanese clinical population	1
Bayesian design of clinical trials with multiple time-to-event outcomes subject to functional cure	1
Nonparametric estimation of a biometric function using neoteric ranked set sampling with application to breast cancer data	1
The proportional treatment effect: A metric that empowers and connects	1
Defective regression models for cure rate data with competing risks	1
Novel 3-arm wait-list controlled trial designs together with mixed-effects analysis improve precision of treatment effect estimators	1
Medical diagnostic accuracy measures: an innovative approach based on the area under predictive values curves	1
AI-powered digital innovations in pharmaceuticals research & development: Current landscape and case examples	1
Joint model for repeated measurements and competing risks data using flexible shared random effects	1
Overview of real-world applications of federated learning with NVIDIA FLARE	1
Estimation and inference of the win ratio for two hierarchical endpoints subject to censoring and missing data	1
A bias correction method for hazard ratio estimation and its inference in a multiple-arm clinical trial	1
Advancing cell and gene therapy: Application of AI/ML in clinical development and patient management	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
Applying latent profile analysis to identify adolescents and young adults with chronic conditions at risk for poor health-related quality of life	1
Bayesian hierarchical model for dose-finding trial incorporating historical data	1
Statistical approaches to evaluate the positive control drug using the hERG assay	1
Adaptive promising zone design for cancer immunotherapy with heterogeneous delayed treatment effect	1
Propensity score stratified MAP prior and posterior inference for incorporating information across multiple potentially heterogeneous data sources	1
Overview of dose-finding designs and trials in cell and gene therapies	1
Miettinen and Nurminen score statistics revisited	1
Using principal progression rate to quantify and compare disease progression in comparative studies	1
Interval estimation of relative risks for combined unilateral and bilateral correlated data	1
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	1
Determining the late effect parameter in the Fleming-Harrington test using asymptotic relative efficiency in cancer immunotherapy clinical trials	1
Issues in cox proportional hazards model with unequal randomization	1
Direct estimation of volume under the ROC surface with verification bias	1
Joint linear and nonlinear mixed effects model with random change points for left-censored longitudinal data: application to HIV surveillance	1
A comparison of Bayesian and score methods for interval estimates of positive/negative likelihood ratios in support of diagnostic device performance evaluation	1
Simultaneous comparison of the predictive values of two binary diagnostic tests in the presence of categorical covariates	1
Assessing predictive probability of success for future clinical trials	1
Epistemic uncertainty in Bayesian predictive probabilities	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
A geometrically intuitive framework for the synthesis method in non-inferiority trials	1
Multivariate equivalence and component-wise superiority tests for paired samples	1
Transporting survival of an HIV clinical trial to the external target populations	1
Large-scale dependent multiple testing via higher-order hidden Markov models	1
BPED: A Bayesian basket design for pediatric trials with external data	1
Handing missing data using multiple imputation in hybrid control clinical trials with modified power prior	1
Pivotal trial design considerations for new and next generation cell and gene therapies	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
RESTART trial design: two-stage seamless transition design with operational considerations	1
A promising subgroup identification method based on a genetic algorithm for censored survival data	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
A phase II, seamless single-arm to two-arm Bayesian design for a time-to-event endpoint	1
Approximate Bayesian estimation of time to clinical benefit using Frequentist approaches: an application to an intensive blood pressure control trial	1
An enhanced exact permutation rank-based inferential seamless phase 2/3 design	1