Journal of Biopharmaceutical Statistics

Article	Citations
Heterogeneous logistic regression for estimation of subgroup effects on hypertension	20
An adaptive seamless 2-in-1 design with biomarker-driven subgroup enrichment	19
Different view of the diagnostics test accuracy measures and optimal cut-off point selection procedure under tree or umbrella ordering	19
In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f 2	17
Quasi-Empirical Bayes methods for parameter estimation involving many small samples	13
Introduction to the special issue Advances in statistical methods for the assessment of patient-centered outcomes	12
A practical analysis procedure on generalizing comparative effectiveness in the randomized clinical trial to the real-world trial-eligible population	12
Estimands, Handling of Missing Data and Impact on Assumed Effect Size and Power in Pivotal COVID-19 Treatment Trials	10
Optimised point estimators for multi-stage single-arm phase II oncology trials	9
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	9
A stochastically curtailed single‐arm phase II trial design for binary outcomes	9
Comparison of profile-likelihood-based confidence intervals with other rank-based methods for the two-sample problem in ordered categorical data	9
Propensity score-integrated Bayesian prior approaches for augmented control designs: a simulation study	9
Estimand in benefit-risk assessment	8
Power and sample size calculation for the win odds test: application to an ordinal endpoint in COVID-19 trials	8
A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions	8
[Special issue PRO] A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	7
A shrinkage estimator for subgroup analysis without the exchangeability assumption	7
Whole-cage randomization for animal studies with unequal cage or group sizes	7
Non-constant mean relative potency for antibody-dependent cellular cytotoxicity assays	7
Testing the homogeneity of proportions for combined unilateral and bilateral data	7
Using surrogate information to improve confirmatory platform trial with sample size re-estimation	7
An enriched approach to combining high-dimensional genomic and low-dimensional phenotypic data	7
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	7
Propensity score-integrated approach to survival analysis: leveraging external evidence in single-arm studies	7

Optimal sample size determination for single-arm trials in pediatric and rare populations with Bayesian borrowing	7
Statistical innovation for next generation pharmaceutical development	7
Optimal designs for phase II clinical trials with heterogeneous patient populations	6
Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects	6
Analysis of innovative two-stage seamless adaptive design with different endpoints and population shift	6
Optimization of EWOC principle in BLRM design for phase 1 oncology trials	6
Implementation of statistical features of a Bayesian two-armed responsive adaptive randomization trial with post hoc analysis of time trend drift	6
Propensity score-integrated power prior approach for augmenting the control arm of a randomized controlled trial by incorporating multiple external data sources	6
Evaluating bias control strategies in observational studies using frequentist model averaging	6
Case study using RWD in the context of a pivotal trial for regulatory approval in a rare disease	5
PMED: Optimal Bayesian Platform Trial Design with Multiple Endpoints	5
Calibrated dynamic borrowing using capping priors	5
MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design	5
Comparison of continuous, binary, and ordinal endpoints	5
Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect	5
Machine learning approach for detection of MACE events within clinical trial data	5
Adjusted win ratio using the inverse probability of treatment weighting	5
Strategies for successful dose optimization in oncology drug development: a practical guide	5
Integrating real world data and clinical trial results using survival data reconstruction and marginal moment-balancing weights	4
Analysis of continuous monitoring device data	4
genRCT: a statistical analysis framework for generalizing RCT findings to real-world population	4
Joint comparison of the predictive values of multiple binary diagnostic tests: an extension of McNemar’s test	4
Clustering plasma concentration-time curves: applications ofunsupervised learning in pharmacogenomics	4
P-values and confidence intervals for weighted log-rank tests under truncated binomial design based on clustered medical data	4
Assessing the incidence and severity of drug adverse events: a Bayesian hierarchical cumulative logit model	4
Survival stacking with multiple data types using pseudo-observation-based-AUC loss	4
Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect	4
A structured framework for adaptively incorporating external evidence in sequentially monitored clinical trials	4
Sample size determination for a study with variable follow-up time	4
Isotonic design for phase I cancer clinical trials with late-onset toxicities	4
Investigating pharmacokinetic profiles of Centella asiatica using machine learning and PBPK modelling	4
FDA experiences with a centralized statistical monitoring tool	4
Double machine learning methods for estimating average treatment effects: a comparative study	4
Bayesian screening for feature selection	4
Bayesian censored piecewise regression mixture models with skewness	4
Estimation of median survival time and its 95% confidence interval using SAS PROC LIFETEST	4
Guest editors’ note on special issue on application of estimand	4
Assessing the use of GEE methods for analyzing binary outcomes in family studies: the Strong Heart Family Study	3
Group sequential multi-arm multi-stage survival trial design with treatment selection	3
Adaptive two-stage seamless sequential design for clinical trials	3
Emerging insights and commentaries – MMRM vs LOCF	3
Imputation of Missing Covariates in Randomized Controlled Trials with Continuous Outcomes: Simple, Unbiased and Efficient Methods	3
Bayesian method for comparing F1 scores in the absence of a gold standard	3
Using Bayesian hierarchical models for controlled post hoc subgroup analysis of clinical trials: application to smoking cessation treatment in American Indians and Alaska Natives	3
Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease	3
Conditional borrowing external data to establish a hybrid control arm in randomized clinical trials	3
Combination MCP-Mod for two-drug combination dose-ranging studies	3
Reflections on estimands for patient-reported outcomes in cancer clinical trials	3
Random intercept hierarchical linear model for multi-regional clinical trials	3
Step-down multiple testing procedures for the overall population and partitioning subgroups	3

Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	3
Deep historical borrowing framework to prospectively and simultaneously synthesize control information in confirmatory clinical trials with multiple endpoints	3
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	3
The use of real-world data for clinical investigation of effectiveness in drug development	3
Emerging clinical initiatives in pharmaceutical development: methodology and regulatory perspectives	3
A win ratio approach for comparing crossing survival curves in clinical trials	3
Retrieved-Dropout-Based multiple imputation for time-to-event data in cardiovascular outcome trials	3
Real world data (RWD) in pediatrics	3
Dynamic borrowing from a single prior data source using the conditional power prior	3
On F β -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy	3
Assessment of nonlinear dose–response relationships via nonparametric regression	3
Conditional power in vaccine trials with seasonal variations	3
Lessons learned in the Apple Heart Study and implications for the data management of future digital clinical trials	3
Innovative methods for rare disease drug development	3
DOD-Combo: Bayesian dose finding design in combination trials with meta-analytic-predictive prior	3
Equivalence tests under the Cox-Aalen model and the partly Aalen model	3
Meta-analysis application to hERG safety evaluation in clinical trials	2
Identifying the clinical relative importance of each correlated outcome variables in multivariate approach: an exploration using ACCORD trial data	2
A systematic approach to adaptive sequential design for clinical trials: using simulations to select a design with desired operating characteristics	2
Robust time selection for interim analysis in the Bayesian phase 2 exploratory clinical trial	2
Optimum designs for clinical trials in personalized medicine when response variance depends on treatment	2
A basket trial design based on constrained hierarchical Bayesian model for latent subgroups	2
Bayesian design of clinical trials using the scale transformed power prior	2
The value of a two-armed Bayesian response adaptive randomization trial	2
Response to Comment on “Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	2
Novel machine learning approach to differential cell flow cytometry analysis based on projection pursuit	2
Bayesian analyses of multiple random change points in survival models with applications to clinical trials	2
A semi-parametric approach for time-dependent ROC curves with nonignorable missing biomarker	2
Guest editors’ note on the special issue International Chinese Statistical Association (ICSA) Applied Statistics Symposium 2021	2
An improved biomarker-guided adaptive patient enrichment design for oncology trials	2
Use of alternative and confirmatory data in support of rare disease drug development	2
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study	2
Flexible parametric accelerated failure time model	2
Correction	2
Assessing model accuracy using random data split: a simulation study	2
Estimation of treatment effects in early-phase randomized clinical trials involving external control data	2
Establishment of RWS guidance reflecting contributions of China to regulatory science	2
Dynamic incorporation of real world evidence within the framework of adaptive design	2
Homogeneity test of several covariance matrices with high-dimensional data	2
Developing large language models to detect adverse drug events in posts on x	2
Methods and Applications of Sample Size Calculation and Recalculation in Clinical Trials	2
Incorporating patient-reported outcomes in dose-finding clinical trials with continuous patient enrollment	2
A new basket trial design based on clustering of homogeneous subpopulations	2
Improving power in adaptive expansion of biomarker populations in phase 3 clinical trials	1
Optimal dose selection in phase I/II dose finding trial with contextual bandits: a case study and practical recommendations	1
Confidence intervals of mean residual life function in length-biased sampling based on modified empirical likelihood	1
Bayesian hierarchical model for dose-finding trial incorporating historical data	1
Estimands and estimators of two-level methods using return to baseline strategy for longitudinal clinical trials with incomplete daily patient reported outcomes	1
Adaptive promising zone design for cancer immunotherapy with heterogeneous delayed treatment effect	1
Rectangular multivariate normal prediction regions for setting reference regions in laboratory medicine	1
Statistical operating characteristics of current early phase dose finding designs with toxicity and efficacy in oncology	1
Bias and Type I error Control in Correcting Treatment Effect for Treatment Switching Using Marginal Structural Models in Phase III Oncology Trials	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
Investigating the impact of data monitoring committee recommendations on the probability of trial success	1
Reverse graphical approaches for multiple test procedures	1
Reimagining optimization of clinical trials efficiency through use of statistical innovation, technology and non-standard data sources	1
Multi-arm multi-stage survival trial design with arm-specific stopping rule	1
Generalized triple outcome decision-making in basket trials	1
Defective 3-parameter Gompertz model with frailty term for estimating cure fraction in survival data	1
Response to comment on “Transporting survival of an HIV clinical trial to the external target populations by Lee et al. (2024)”	1
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing	1
A promising subgroup identification method based on a genetic algorithm for censored survival data	1
Study design for a three-arm equivalence clinical trial with binomially distributed outcomes	1
Overview of real-world applications of federated learning with NVIDIA FLARE	1
Correction	1
Applied Meta-Analysis with R and Stata, 2nd Edition	1
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	1
Group sequential designs for cancer immunotherapy trial with delayed treatment effect	1
Inverse probability weighted Bayesian dynamic borrowing for estimation of marginal treatment effects with application to hybrid control arm oncology studies	1
DOD-BART: machine learning-based dose optimization design incorporating patient-level prognostic factors via Bayesian additive regression trees	1
Assess predictive values of a binary diagnostic test under a nested case–control design	1
Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time	1
Miettinen and Nurminen score statistics revisited	1
Epistemic uncertainty in Bayesian predictive probabilities	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
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials	1
Large-scale dependent multiple testing via higher-order hidden Markov models	1
The role of regulatory flexibility in the review and approval process of rare disease drug development	1
Interval estimation of relative risks for combined unilateral and bilateral correlated data	1

Non-inferiority testing for qualitative microbiological methods:Assessing and improving the approach in USP <1223>	1
A composite semiparametric homogeneity test for the distributions of multigroup interval-bounded longitudinal data	1
Two-stage response adaptive randomization designs for multi-arm trials with binary outcome	1
Estimation of the selected treatment mean in two stage drop-the-losers design	1
On a Holm-related MTP for rejecting at least k hypotheses: general validity, optimality property, confidence regions, and applications	1
Statistical methods of indirect comparison with real-world data for survival endpoint under non-proportional hazards	1
Transporting survival of an HIV clinical trial to the external target populations	1
Optimal two-phase sampling for comparing correlated areas under the ROC curves of two screening tests in the presence of verification bias	1
Medical diagnostic accuracy measures: an innovative approach based on the area under predictive values curves	1
Bayesian model averaging of longitudinal dose-response models	1
Determining the late effect parameter in the Fleming-Harrington test using asymptotic relative efficiency in cancer immunotherapy clinical trials	1
Four-parameter paired response curve for serial dilution assays	1
Direct estimation of volume under the ROC surface with verification bias	1
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US	1
Applying latent profile analysis to identify adolescents and young adults with chronic conditions at risk for poor health-related quality of life	1
Statistical learning in preclinical drug proarrhythmic assessment	1
A novel approach to augment single-arm clinical studies with real-world data	1
Propensity score stratified MAP prior and posterior inference for incorporating information across multiple potentially heterogeneous data sources	1
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	1
Correction	1
A randomized Bayesian optimal phase II design with binary endpoint	1
Comments on ”Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	1
Statistical considerations for using tolerance interval to set product specification for normally distributed attribute	1
Bayesian design of clinical trials with multiple time-to-event outcomes subject to functional cure	1
Response to Comment on ‘Statistical Consideration and Challenges in Bridging Study of Personalized Medicine’: A modified variance for sensitivity	1
Power priors with entropy balancing weights in data augmentation of partially controlled randomized trials	1
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	0
Net benefit of diagnostic tests for multistate diseases: an indicator variables approach	0
Multiple test procedures of disease prevalence based on stratified partially validated series in the presence of a gold standard	0
Re: Transporting survival of an HIV clinical trial to the external target populations	0
Joint modeling of longitudinal endpoints and its applications to trial planning, monitoring and analysis	0
Parameter estimation of Weibull distribution for the number of days between drug administration and early onset adverse event	0
Providing meaningful interpretation of performance outcome measures by co-calibration with patient-reported outcomes through the Rasch model: illustration with multiple sclerosis measures	0
Comment on ‘statistical consideration and challenges in bridging study of personalized medicine’: a modified variance for sensitivity analysis	0
The win odds: statistical inference and regression	0
A Bayesian model for combining standardized mean differences and odds ratios in the same meta-analysis	0
Composite Likelihoods with Bounded Weights in Extrapolation of Data	0
Utility of propensity score-based Bayesian borrowing of external adult data in pediatric trials: A pragmatic evaluation through a case study in acute lymphoblastic leukemia (ALL)	0
Regional consistency assessment in multiregional clinical trials	0
On variance estimation of target population created by inverse probability weighting	0
Modelling non-linear patterns of time-varying intervention effects on recurrent events in infectious disease prevention studies	0
Randomized two-stage optimal design for interval-censored data	0
Study duration prediction for clinical trials with time-to-event endpoints accounting for heterogeneous population	0
On the use of RWD in support of regulatory submission in drug development	0
MIDAS-2: an enhanced Bayesian platform design for immunotherapy combinations with subgroup efficacy exploration	0
A Bayesian phase I–II clinical trial design to find the biological optimal dose on drug combination	0
Confidence interval construction for proportion difference from partially validated series with two fallible classifiers	0
Randomization tests in clinical trials with multiple imputation for handling missing data	0
Statistical methods to control for confounders in rare disease settings that use external control	0
Small sample adjustment for inference without assuming orthogonality in a mixed model for repeated measures analysis	0
Pediatric needs should be considered early in the clinical development lifecycle	0
Recurrent neural networks and attention scores for personalized prediction and interpretation of patient-reported outcomes	0
Generalized exponentiated unit Gompertz distribution for modeling arthritic pain relief times data: classical approach to statistical inference	0
Assessing adverse events in clinical trials during the era of the COVID-19 pandemic	0
A Bayesian framework for safety signal detection from medical device data	0
Response to letter to editor	0
Clinical trial considerations for pediatric cancer drug development	0
On the feasibility of pediatric dose-finding trials in small samples with information from a preceding trial in adults	0
On Reference-based Imputation for Analysis of Incomplete Repeated Binary Endpoints	0
Equivalence tests before end of follow-up under the class of log transformation model	0
Variable selection for longitudinal zero-inflated power series transition model	0
Challenges and potential strategies utilizing external data for efficacy evaluation in small-sized clinical trials	0
Two-stage designs with small sample sizes	0
Pharmacometrics-Enabled DOse OPtimization (PEDOOP) for seamless phase I-II trials in oncology	0
Weighted log-rank tests for left-truncated data under Wei’s urn design: saddlepoint p-values and confidence intervals	0
Modelling alternately recurring events using subject specific hazard estimation approach	0
On dependence assumption in p-value based multiple test procedures	0
Enhancement of Bayesian optimal interval design by accounting for overdose and underdose errors trade-offs	0
Latent class analysis of post-acute sequelae of SARS-CoV-2 infection	0
How to select the initial dose for a pediatric study?	0
Adaptive sequential design for phase II single-arm oncology trials: an expansion of Simon’s design	0
Missing data in the eligibility criteria of synthetic controls from real-world data	0
Weighted log-rank tests for censored data under Wei’s urn design: saddlepoint approximation and confidence intervals	0
A Two-Stage Decision Making Approach for Safety Studies	0
Applying machine learning-based multiple imputation methods to nonparametric multiple comparisons in longitudinal clinical studies	0
Association of the medication protocols and longitudinal change of COVID-19 symptoms: a hospital-based mixed-statistical methods study	0
The Ci3+3 design for dual-agent combination dose-finding clinical trials	0
Improved group sequential Holm procedures for testing multiple correlated hypotheses over time	0
Implementing the meta-analytic approach for the evaluation of surrogate endpoints in SAS and R: a word of caution	0
Perspectives on informative Bayesian methods in pediatrics	0
Application of estimand framework to the design and analysis of multi-regional clinical trials	0
Revolutionizing cardiovascular disease classification through machine learning and statistical methods	0
A Bayesian approach for studying COVID-19 contagion dynamics in Algeria using a Poisson autoregressive (PAR) model	0
Valid overall odds ratio estimators using different stratified sampling schemes	0
The score-goldilocks design for phase 3 clinical trials	0
Bayesian sample size determination in a three-arm non-inferiority trial with binary endpoints	0
Statistical Considerations and Software for Designing Sequential, Multiple Assignment, Randomized Trials (SMART) with a Survival Final Endpoint	0
Application of estimand framework in ICH E9 (R1) to safety evaluation	0
Adaptive Multiple Comparison Sequential Design (AMCSD) for clinical trials	0
Bayesian efficient safety monitoring: a simple and well-performing framework to continuous safety monitoring of adverse events in randomized clinical trials	0
Quantile regression shrinkage and selection via the Lqsso	0
Adaptively leverage multiple real-world data sources for treatment effect estimation based on similarity	0
CORRECTION	0
Assessing clinical response in early oncology development with a predictive biomarker	0
Robust safety monitoring and signal detection using alternatives to the standard poisson distribution	0