Journal of Biopharmaceutical Statistics

Article	Citations
Estimands, Handling of Missing Data and Impact on Assumed Effect Size and Power in Pivotal COVID-19 Treatment Trials	19
Optimised point estimators for multi-stage single-arm phase II oncology trials	19
Heterogeneous logistic regression for estimation of subgroup effects on hypertension	19
Comparison of profile-likelihood-based confidence intervals with other rank-based methods for the two-sample problem in ordered categorical data	17
An adaptive seamless 2-in-1 design with biomarker-driven subgroup enrichment	16
A stochastically curtailed single‐arm phase II trial design for binary outcomes	13
Different view of the diagnostics test accuracy measures and optimal cut-off point selection procedure under tree or umbrella ordering	12
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	12
In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f 2	10
Propensity score-integrated Bayesian prior approaches for augmented control designs: a simulation study	9
Whole-cage randomization for animal studies with unequal cage or group sizes	9
Power and sample size calculation for the win odds test: application to an ordinal endpoint in COVID-19 trials	9
A practical analysis procedure on generalizing comparative effectiveness in the randomized clinical trial to the real-world trial-eligible population	9
Quasi-Empirical Bayes methods for parameter estimation involving many small samples	9
A shrinkage estimator for subgroup analysis without the exchangeability assumption	8
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	8
[Special issue PRO] A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	7
Testing the homogeneity of proportions for combined unilateral and bilateral data	7
A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions	7
Estimand in benefit-risk assessment	7
Using surrogate information to improve confirmatory platform trial with sample size re-estimation	7
Optimal sample size determination for single-arm trials in pediatric and rare populations with Bayesian borrowing	7
Propensity score-integrated approach to survival analysis: leveraging external evidence in single-arm studies	7
Propensity score-integrated power prior approach for augmenting the control arm of a randomized controlled trial by incorporating multiple external data sources	6
Statistical innovation for next generation pharmaceutical development	6

Evaluating bias control strategies in observational studies using frequentist model averaging	6
An enriched approach to combining high-dimensional genomic and low-dimensional phenotypic data	6
Non-constant mean relative potency for antibody-dependent cellular cytotoxicity assays	6
Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects	6
Optimal designs for phase II clinical trials with heterogeneous patient populations	5
Machine learning approach for detection of MACE events within clinical trial data	5
Case study using RWD in the context of a pivotal trial for regulatory approval in a rare disease	5
Implementation of statistical features of a Bayesian two-armed responsive adaptive randomization trial with post hoc analysis of time trend drift	5
Optimization of EWOC principle in BLRM design for phase 1 oncology trials	5
Calibrated dynamic borrowing using capping priors	5
PMED: Optimal Bayesian Platform Trial Design with Multiple Endpoints	5
Analysis of innovative two-stage seamless adaptive design with different endpoints and population shift	5
Strategies for successful dose optimization in oncology drug development: a practical guide	5
Directed Acyclic Graph Assisted Method For Estimating Average Treatment Effect	5
Comparison of continuous, binary, and ordinal endpoints	4
A structured framework for adaptively incorporating external evidence in sequentially monitored clinical trials	4
Analysis of continuous monitoring device data	4
Double machine learning methods for estimating average treatment effects: a comparative study	4
Sample size determination for a study with variable follow-up time	4
A collection of parametric modal regression models for bounded data	4
FDA experiences with a centralized statistical monitoring tool	4
MOVER tests for non-inferiority of the difference between two binary-outcome treatments in the matched-pairs design	4
Integrating real world data and clinical trial results using survival data reconstruction and marginal moment-balancing weights	4
Deep historical borrowing framework to prospectively and simultaneously synthesize control information in confirmatory clinical trials with multiple endpoints	4
Joint comparison of the predictive values of multiple binary diagnostic tests: an extension of McNemar’s test	4
Conditional power in vaccine trials with seasonal variations	4
Adjusted win ratio using the inverse probability of treatment weighting	4
Bayesian screening for feature selection	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
genRCT: a statistical analysis framework for generalizing RCT findings to real-world population	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
Conditional borrowing external data to establish a hybrid control arm in randomized clinical trials	3
Guest editors’ note on the special issue International Chinese Statistical Association (ICSA) Applied Statistics Symposium 2021	3
Bayesian method for comparing F1 scores in the absence of a gold standard	3
Investigating pharmacokinetic profiles of Centella asiatica using machine learning and PBPK modelling	3
Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease	3
Assessing the incidence and severity of drug adverse events: a Bayesian hierarchical cumulative logit model	3
Estimation of median survival time and its 95% confidence interval using SAS PROC LIFETEST	3
Innovative methods for rare disease drug development	3
Assessment of nonlinear dose–response relationships via nonparametric regression	3
DOD-Combo: Bayesian dose finding design in combination trials with meta-analytic-predictive prior	3
On F β -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy	3
Robust time selection for interim analysis in the Bayesian phase 2 exploratory clinical trial	3
Dynamic borrowing from a single prior data source using the conditional power prior	3
Combination MCP-Mod for two-drug combination dose-ranging studies	3
P-values and confidence intervals for weighted log-rank tests under truncated binomial design based on clustered medical data	3
Retrieved-Dropout-Based multiple imputation for time-to-event data in cardiovascular outcome trials	3
Bayesian censored piecewise regression mixture models with skewness	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

Lessons learned in the Apple Heart Study and implications for the data management of future digital clinical trials	3
Dynamic incorporation of real world evidence within the framework of adaptive design	3
Emerging insights and commentaries – MMRM vs LOCF	3
Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	3
Emerging clinical initiatives in pharmaceutical development: methodology and regulatory perspectives	3
Guest editors’ note on special issue on application of estimand	3
Imputation of Missing Covariates in Randomized Controlled Trials with Continuous Outcomes: Simple, Unbiased and Efficient Methods	3
Clustering plasma concentration-time curves: applications ofunsupervised learning in pharmacogenomics	3
Isotonic design for phase I cancer clinical trials with late-onset toxicities	3
Assessing the use of GEE methods for analyzing binary outcomes in family studies: the Strong Heart Family Study	3
The use of real-world data for clinical investigation of effectiveness in drug development	3
Reflections on estimands for patient-reported outcomes in cancer clinical trials	3
Step-down multiple testing procedures for the overall population and partitioning subgroups	3
Adaptive two-stage seamless sequential design for clinical trials	3
Optimum designs for clinical trials in personalized medicine when response variance depends on treatment	2
Establishment of RWS guidance reflecting contributions of China to regulatory science	2
Equivalence tests under the Cox-Aalen model and the partly Aalen model	2
A win ratio approach for comparing crossing survival curves in clinical trials	2
A new basket trial design based on clustering of homogeneous subpopulations	2
Methods and Applications of Sample Size Calculation and Recalculation in Clinical Trials	2
A basket trial design based on constrained hierarchical Bayesian model for latent subgroups	2
An improved biomarker-guided adaptive patient enrichment design for oncology trials	2
A systematic approach to adaptive sequential design for clinical trials: using simulations to select a design with desired operating characteristics	2
Identifying the clinical relative importance of each correlated outcome variables in multivariate approach: an exploration using ACCORD trial data	2
Group sequential multi-arm multi-stage survival trial design with treatment selection	2
Random intercept hierarchical linear model for multi-regional clinical trials	2
Assessing model accuracy using random data split: a simulation study	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
Estimation of the selected treatment mean in two stage drop-the-losers design	2
Meta-analysis application to hERG safety evaluation in clinical trials	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
Real world data (RWD) in pediatrics	2
Estimation of treatment effects in early-phase randomized clinical trials involving external control data	2
Novel machine learning approach to differential cell flow cytometry analysis based on projection pursuit	2
Homogeneity test of several covariance matrices with high-dimensional data	2
Correction	2
Flexible parametric accelerated failure time model	2
Use of alternative and confirmatory data in support of rare disease drug development	2
Design and analysis of drop-the-losers studies using binary endpoints in the rare disease setting	2
Generalized triple outcome decision-making in basket trials	1
Inverse probability weighted Bayesian dynamic borrowing for estimation of marginal treatment effects with application to hybrid control arm oncology studies	1
Improving power in adaptive expansion of biomarker populations in phase 3 clinical trials	1
A randomized Bayesian optimal phase II design with binary endpoint	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
Response to comment on “Transporting survival of an HIV clinical trial to the external target populations by Lee et al. (2024)”	1
Correction	1
Defective 3-parameter Gompertz model with frailty term for estimating cure fraction in survival data	1
Reimagining optimization of clinical trials efficiency through use of statistical innovation, technology and non-standard data sources	1
Comments on ”Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	1
Non-inferiority testing for qualitative microbiological methods:Assessing and improving the approach in USP <1223>	1
Statistical operating characteristics of current early phase dose finding designs with toxicity and efficacy in oncology	1
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing	1
Evaluation of the lifetime performance index on first failure progressive censored data based on Topp Leone Alpha power exponential model applied on HPLC data	1
Bigger and bigger circles - the expanding biopharmaceutical statistician’s toolbox	1
Bayesian design of clinical trials using the scale transformed power prior	1
Determining the late effect parameter in the Fleming-Harrington test using asymptotic relative efficiency in cancer immunotherapy clinical trials	1
Assess predictive values of a binary diagnostic test under a nested case–control design	1
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US	1
On a Holm-related MTP for rejecting at least k hypotheses: general validity, optimality property, confidence regions, and applications	1
Rectangular multivariate normal prediction regions for setting reference regions in laboratory medicine	1
Transporting survival of an HIV clinical trial to the external target populations	1
A novel approach to augment single-arm clinical studies with real-world data	1
Bayesian design of clinical trials with multiple time-to-event outcomes subject to functional cure	1
The role of regulatory flexibility in the review and approval process of rare disease drug development	1
Reverse graphical approaches for multiple test procedures	1
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials	1
Optimal dose selection in phase I/II dose finding trial with contextual bandits: a case study and practical recommendations	1
Investigating the impact of data monitoring committee recommendations on the probability of trial success	1
Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time	1
Study design for a three-arm equivalence clinical trial with binomially distributed outcomes	1
Optimal two-phase sampling for comparing correlated areas under the ROC curves of two screening tests in the presence of verification bias	1
Applied Meta-Analysis with R and Stata, 2nd Edition	1
Statistical methods of indirect comparison with real-world data for survival endpoint under non-proportional hazards	1
The value of a two-armed Bayesian response adaptive randomization trial	1
Epistemic uncertainty in Bayesian predictive probabilities	1
Propensity score stratified MAP prior and posterior inference for incorporating information across multiple potentially heterogeneous data sources	1
Group sequential designs for cancer immunotherapy trial with delayed treatment effect	1

A promising subgroup identification method based on a genetic algorithm for censored survival data	1
Confidence intervals of mean residual life function in length-biased sampling based on modified empirical likelihood	1
Two-stage response adaptive randomization designs for multi-arm trials with binary outcome	1
Estimands and estimators of two-level methods using return to baseline strategy for longitudinal clinical trials with incomplete daily patient reported outcomes	1
A composite semiparametric homogeneity test for the distributions of multigroup interval-bounded longitudinal data	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
Response to Comment on ‘Statistical Consideration and Challenges in Bridging Study of Personalized Medicine’: A modified variance for sensitivity	1
Addressing sequential and concurrent treatment regimens in a small n sequential, multiple assignment, randomized trial (snSMART) in the MISTIC study	1
Four-parameter paired response curve for serial dilution assays	1
Statistical considerations for using tolerance interval to set product specification for normally distributed attribute	1
Inferential properties with a novel two parameter Poisson generalized Lindley distribution with regression and application to INAR(1) process	1
Developing large language models to detect adverse drug events in posts on x	1
Medical diagnostic accuracy measures: an innovative approach based on the area under predictive values curves	1
Power priors with entropy balancing weights in data augmentation of partially controlled randomized trials	1
Confidence interval construction for proportion difference from partially validated series with two fallible classifiers	0
Bayesian spatial cluster signal learning with application to adverse event (AE)	0
Adaptive Multiple Comparison Sequential Design (AMCSD) for clinical trials	0
BOP2-TE: Bayesian optimal phase 2 design for jointly monitoring efficacy and toxicity with application to dose optimization	0
A pragmatic approach for dynamically incorporating predicate device data in prospective diagnostic test studies	0
Comment on ‘statistical consideration and challenges in bridging study of personalized medicine’: a modified variance for sensitivity analysis	0
Bayesian efficient safety monitoring: a simple and well-performing framework to continuous safety monitoring of adverse events in randomized clinical trials	0
Referees for Volume 32	0
Application of estimand framework in ICH E9 (R1) to safety evaluation	0
On variance estimation of target population created by inverse probability weighting	0
Bayesian sample size determination in a three-arm non-inferiority trial with binary endpoints	0
A Two-Stage Decision Making Approach for Safety Studies	0
Exploring the Potential of External Control Arms created from Patient Level Data: A case study in non-small cell lung cancer	0
Empower clinical development by harnessing data from diverse sources: methodology, applications and regulatory perspectives	0
Joint modeling of longitudinal endpoints and its applications to trial planning, monitoring and analysis	0
A Bayesian approach for studying COVID-19 contagion dynamics in Algeria using a Poisson autoregressive (PAR) model	0
A Bayesian framework for safety signal detection from medical device data	0
Latent class analysis of post-acute sequelae of SARS-CoV-2 infection	0
On the use of RWD in support of regulatory submission in drug development	0
Net benefit of diagnostic tests for multistate diseases: an indicator variables approach	0
Recurrent neural networks and attention scores for personalized prediction and interpretation of patient-reported outcomes	0
A Bayesian model for combining standardized mean differences and odds ratios in the same meta-analysis	0
On dependence assumption in p-value based multiple test procedures	0
Bayesian Divide-and-Conquer Propensity Score Based Approaches for Leveraging Real World Data in Single Arm Clinical Trials	0
Generalized exponentiated unit Gompertz distribution for modeling arthritic pain relief times data: classical approach to statistical inference	0
Composite Likelihoods with Bounded Weights in Extrapolation of Data	0
Perspectives on informative Bayesian methods in pediatrics	0
Modelling non-linear patterns of time-varying intervention effects on recurrent events in infectious disease prevention studies	0
Missing data in the eligibility criteria of synthetic controls from real-world data	0
Pharmacometrics-Enabled DOse OPtimization (PEDOOP) for seamless phase I-II trials in oncology	0
Statistical Considerations and Software for Designing Sequential, Multiple Assignment, Randomized Trials (SMART) with a Survival Final Endpoint	0
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	0
Study duration prediction for clinical trials with time-to-event endpoints accounting for heterogeneous population	0
Guest Editors’ Note on the Special Issue Real-World Evidence	0
Assessing adverse events in clinical trials during the era of the COVID-19 pandemic	0
MIDAS-2: an enhanced Bayesian platform design for immunotherapy combinations with subgroup efficacy exploration	0
Clinical trial considerations for pediatric cancer drug development	0
Adaptively leverage multiple real-world data sources for treatment effect estimation based on similarity	0
Pediatric needs should be considered early in the clinical development lifecycle	0
The win odds: statistical inference and regression	0
Quantile regression shrinkage and selection via the Lqsso	0
The Ci3+3 design for dual-agent combination dose-finding clinical trials	0
How to select the initial dose for a pediatric study?	0
Re: Transporting survival of an HIV clinical trial to the external target populations	0
Incorporating propensity scores for evidence synthesis under bayesian framework: review and recommendations for clinical studies	0
Weighted log-rank tests for censored data under Wei’s urn design: saddlepoint approximation and confidence intervals	0
CORRECTION	0
Variable selection for longitudinal zero-inflated power series transition model	0
Small sample adjustment for inference without assuming orthogonality in a mixed model for repeated measures analysis	0
Bayesian phase II adaptive randomization by jointly modeling efficacy and toxicity as time-to-event outcomes	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
Revolutionizing cardiovascular disease classification through machine learning and statistical methods	0
Two-stage designs with small sample sizes	0
Regional consistency assessment in multiregional clinical trials	0
On the feasibility of pediatric dose-finding trials in small samples with information from a preceding trial in adults	0
Randomized two-stage optimal design for interval-censored data	0
Application of estimand framework to the design and analysis of multi-regional clinical trials	0
Parameter estimation of Weibull distribution for the number of days between drug administration and early onset adverse event	0
A machine learning case study to predict rare clinical event of interest: imbalanced data, interpretability, and practical considerations	0
A Bayesian phase I–II clinical trial design to find the biological optimal dose on drug combination	0
Modelling alternately recurring events using subject specific hazard estimation approach	0
Optimal dose escalation methods using deep reinforcement learning in phase I oncology trials	0
Enhancement of Bayesian optimal interval design by accounting for overdose and underdose errors trade-offs	0
Multiple test procedures of disease prevalence based on stratified partially validated series in the presence of a gold standard	0
Response to letter to editor	0
Saddlepoint p-values for a class of location-scale tests	0
Randomization tests in clinical trials with multiple imputation for handling missing data	0
Applying machine learning-based multiple imputation methods to nonparametric multiple comparisons in longitudinal clinical studies	0
Statistical methods to control for confounders in rare disease settings that use external control	0
Improved group sequential Holm procedures for testing multiple correlated hypotheses over time	0
Association of the medication protocols and longitudinal change of COVID-19 symptoms: a hospital-based mixed-statistical methods study	0
On Reference-based Imputation for Analysis of Incomplete Repeated Binary Endpoints	0
Robust safety monitoring and signal detection using alternatives to the standard poisson distribution	0
Incorporating external real-world data (RWD) in confirmatory adaptive design	0
The score-goldilocks design for phase 3 clinical trials	0
Implementing the meta-analytic approach for the evaluation of surrogate endpoints in SAS and R: a word of caution	0
Multi-arm multi-stage clinical trials for time-to-event outcomes	0
Adaptive sequential design for phase II single-arm oncology trials: an expansion of Simon’s design	0
Valid overall odds ratio estimators using different stratified sampling schemes	0
Weighted log-rank tests for left-truncated data under Wei’s urn design: saddlepoint p-values and confidence intervals	0
Challenges and potential strategies utilizing external data for efficacy evaluation in small-sized clinical trials	0