Journal of Biopharmaceutical Statistics

Article	Citations
Applying latent profile analysis to identify adolescents and young adults with chronic conditions at risk for poor health-related quality of life	17
Implementing the meta-analytic approach for the evaluation of surrogate endpoints in SAS and R: a word of caution	16
Comment on ‘statistical consideration and challenges in bridging study of personalized medicine’: a modified variance for sensitivity analysis	15
Retraction: A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	14
A Bayesian framework for safety signal detection from medical device data	12
Multi-stage dose expansion cohort (MSDEC) design with Bayesian stopping rule	11
Rectangular multivariate normal prediction regions for setting reference regions in laboratory medicine	11
A Bayesian three-tier quantitative decision-making framework for single arm studies in early phase oncology	10
Estimands, Handling of Missing Data and Impact on Assumed Effect Size and Power in Pivotal COVID-19 Treatment Trials	9
Optimised point estimators for multi-stage single-arm phase II oncology trials	9
Net benefit of diagnostic tests for multistate diseases: an indicator variables approach	9
Comparison of profile-likelihood-based confidence intervals with other rank-based methods for the two-sample problem in ordered categorical data	9
Biomarker-driven basket trial designs: origins and new methodological developments	9
Re: Transporting survival of an HIV clinical trial to the external target populations	8
A comparison of Bayesian and score methods for interval estimates of positive/negative likelihood ratios in support of diagnostic device performance evaluation	7
Forecasting determinants of recurrence in lung cancer patients exploiting various machine learning models	7
Assess predictive values of a binary diagnostic test under a nested case–control design	7
Small sample adjustment for inference without assuming orthogonality in a mixed model for repeated measures analysis	7
Emerging clinical initiatives in pharmaceutical development: methodology and regulatory perspectives	7
Innovative methods for rare disease drug development	7
In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f 2	6
Leveraging pharmacokinetic parameters as covariate in Bayesian logistic regression model to optimize dose selection in early phase oncology trial	6
Bayesian Hierarchy model for population pharmacokinetics of amikacin in Japanese clinical population	6
Association of the medication protocols and longitudinal change of COVID-19 symptoms: a hospital-based mixed-statistical methods study	6
Power and sample size calculation for the win odds test: application to an ordinal endpoint in COVID-19 trials	6

Bayesian model averaging for randomized dose optimization trials in multiple indications	6
Weighted log-rank tests for censored data under Wei’s urn design: saddlepoint approximation and confidence intervals	6
Applying machine learning-based multiple imputation methods to nonparametric multiple comparisons in longitudinal clinical studies	5
Risk difference, relative risk, and odds ratio for non-inferiority clinical trials with risk rate endpoint	5
Determining the late effect parameter in the Fleming-Harrington test using asymptotic relative efficiency in cancer immunotherapy clinical trials	5
A nonparametric approach to confidence intervals for concordance index and difference between correlated indices	5
Heterogeneous logistic regression for estimation of subgroup effects on hypertension	5
Borrowing historical information to improve phase I clinical trials using meta-analytic-predictive priors	5
Group sequential designs for cancer immunotherapy trial with delayed treatment effect	5
An adaptive seamless 2-in-1 design with biomarker-driven subgroup enrichment	5
Confidence intervals of mean residual life function in length-biased sampling based on modified empirical likelihood	5
Estimands and estimators of two-level methods using return to baseline strategy for longitudinal clinical trials with incomplete daily patient reported outcomes	4
Conditional borrowing external data to establish a hybrid control arm in randomized clinical trials	4
Design of paediatric trials with benefit-risk endpoints using a composite score of adverse events of interest (AEI) and win-statistics	4
Different view of the diagnostics test accuracy measures and optimal cut-off point selection procedure under tree or umbrella ordering	4
A novel approach to augment single-arm clinical studies with real-world data	4
Correction	4
The 2009 FDA PRO guidance, Potential Type I error, Descriptive Statistics and Pragmatic estimation of the number of interviews for item elicitation	4
A stochastically curtailed single‐arm phase II trial design for binary outcomes	4
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US	4
Dynamic borrowing from a single prior data source using the conditional power prior	4
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	4
Propensity score-integrated Bayesian prior approaches for augmented control designs: a simulation study	4
Robust safety monitoring and signal detection using alternatives to the standard poisson distribution	4
Epistemic uncertainty in Bayesian predictive probabilities	4
Step-down multiple testing procedures for the overall population and partitioning subgroups	3
REFEREES FOR VOLUME 31	3
A shrinkage estimator for subgroup analysis without the exchangeability assumption	3
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	3
Latent class analysis of post-acute sequelae of SARS-CoV-2 infection	3
Statistical operating characteristics of current early phase dose finding designs with toxicity and efficacy in oncology	3
Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	3
On F β -score for medical diagnostics tests of binary diseases: proposing new measures of accuracy	3
Evaluation of the lifetime performance index on first failure progressive censored data based on Topp Leone Alpha power exponential model applied on HPLC data	3
Using surrogate information to improve confirmatory platform trial with sample size re-estimation	3
Optimal dose selection in phase I/II dose finding trial with contextual bandits: a case study and practical recommendations	3
How to select the initial dose for a pediatric study?	3
Efficient estimation of the cox model when incorporating the subgroup restricted mean survival time	3
Emerging insights and commentaries – MMRM vs LOCF	3
Exploring the Potential of External Control Arms created from Patient Level Data: A case study in non-small cell lung cancer	3
Guest editors’ note on the special issue International Chinese Statistical Association (ICSA) Applied Statistics Symposium 2021	3
Reflections on estimands for patient-reported outcomes in cancer clinical trials	3
Referees for Volume 33	3
Quantile regression shrinkage and selection via the Lqsso	3
A win ratio-based framework to combine multiple clinical endpoints in exploratory basket trials	3
[Special issue PRO] A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	3
Drug response hysteresis in the concentration-QTc analysis of early clinical trials	3
Adaptive sequential design for phase II single-arm oncology trials: an expansion of Simon’s design	3
Consideration of the adaptive randomization allocation ratio in the presence of treatment group heteroscedasticity in clinical trials	3
Recurrent neural networks and attention scores for personalized prediction and interpretation of patient-reported outcomes	3

The Ci3+3 design for dual-agent combination dose-finding clinical trials	3
Assessment of nonlinear dose–response relationships via nonparametric regression	3
Establishment of RWS guidance reflecting contributions of China to regulatory science	3
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)	3
Conditional bias adjusted estimator of treatment effect in 2-in-1 adaptive design	3
Robust time selection for interim analysis in the Bayesian phase 2 exploratory clinical trial	3
Handbook of meta-analysis	3
Bayesian approaches to benefit-risk assessment for diagnostic tests	3
Statistical methods to control for confounders in rare disease settings that use external control	3
Dynamic incorporation of real world evidence within the framework of adaptive design	2
CORRECTION	2
Random intercept hierarchical linear model for multi-regional clinical trials	2
Propensity score-integrated approach to survival analysis: leveraging external evidence in single-arm studies	2
A win ratio approach for comparing crossing survival curves in clinical trials	2
Up-front matching: an ongoing recruitment method for prospective observational studies that mimics randomization for selected baseline covariates	2
Estimand in benefit-risk assessment	2
Adaptive two-stage seamless sequential design for clinical trials	2
Equivalence tests under the Cox-Aalen model and the partly Aalen model	2
A Bayesian group sequential design for randomized biosimilar clinical trials with adaptive information borrowing from historical data	2
Four-parameter paired response curve for serial dilution assays	2
Inferential properties with a novel two parameter Poisson generalized Lindley distribution with regression and application to INAR(1) process	2
Optimal sample size determination for single-arm trials in pediatric and rare populations with Bayesian borrowing	2
Saddlepoint p-values for a class of location-scale tests	2
Randomization tests in clinical trials with multiple imputation for handling missing data	2
A Bayesian joint bent-cable model for longitudinal measurements and survival time with heterogeneous random-effects distributions	2
Group sequential multi-arm multi-stage survival trial design with treatment selection	2
Dose-finding based on feasibility and late-onset toxicity in adoptive cell therapy trials	2
Novel 3-arm wait-list controlled trial designs together with mixed-effects analysis improve precision of treatment effect estimators	2
Confidence intervals for the common odds ratio based on the inverse sinh transformation	2
A Cholesky-based sparse covariance estimation with an application to genes data	2
Testing the homogeneity of proportions for combined unilateral and bilateral data	2
Diagnostic evaluation of pharmacokinetic features of functional markers	2
Whole-cage randomization for animal studies with unequal cage or group sizes	2
Utilizing stratified generalized propensity score matching to approximate blocked randomized designs with multiple treatment levels	1
Inclusion of unexposed subjects improves the precision and power of self-controlled case series method	1
Applied Meta-Analysis with R and Stata, 2nd Edition	1
A systematic approach to adaptive sequential design for clinical trials: using simulations to select a design with desired operating characteristics	1
Design and analysis of drop-the-losers studies using binary endpoints in the rare disease setting	1
Defective 3-parameter Gompertz model with frailty term for estimating cure fraction in survival data	1
Evidence synthesis analysis with prioritized benefit outcomes in oncology clinical trials	1
Incorporating external real-world data (RWD) in confirmatory adaptive design	1
A confirmatory basket design considering non-inferiority and superiority testing	1
Empower clinical development by harnessing data from diverse sources: methodology, applications and regulatory perspectives	1
Nonparametric inference of the area under ROC curve under two-phase cluster sampling	1
Two-stage response adaptive randomization designs for multi-arm trials with binary outcome	1
Analysis and reporting of pediatric growth and development assessment from clinical trials: overview and challenges	1
Improved confidence estimation for the binomial proportion with applications to clinical studies	1
Response to comment on “Transporting survival of an HIV clinical trial to the external target populations by Lee et al. (2024)”	1
Medical diagnostic accuracy measures: an innovative approach based on the area under predictive values curves	1
Adaptive platform trials: the impact of common controls on type one error and power	1
A composite semiparametric homogeneity test for the distributions of multigroup interval-bounded longitudinal data	1
Simulating survival data when one subgroup lacks information	1
The impact of misclassification errors on the performance of biomarkers based on next-generation sequencing, a simulation study	1
Non-constant mean relative potency for antibody-dependent cellular cytotoxicity assays	1
Bayesian design of clinical trials with multiple time-to-event outcomes subject to functional cure	1
Statistics and machine learning methods for EHR data – from data extraction to data analytics	1
Transporting survival of an HIV clinical trial to the external target populations	1
Real world data (RWD) in pediatrics	1
Utility of real-world evidence in biosimilar development	1
Bayesian Divide-and-Conquer Propensity Score Based Approaches for Leveraging Real World Data in Single Arm Clinical Trials	1
Leveraging real-world data to conduct externally controlled trial for rare diseases with count-type endpoints: utilizing multiple entries – a simulation study	1
Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects	1
Improved automated spot counting and modeling with bias correction	1
Application of estimand framework in ICH E9 (R1) to vaccine trials	1
Evaluating bias control strategies in observational studies using frequentist model averaging	1
A semi-parametric approach for time-dependent ROC curves with nonignorable missing biomarker	1
Response to Comment on ‘Statistical Consideration and Challenges in Bridging Study of Personalized Medicine’: A modified variance for sensitivity	1
Comments on ”Emerging insights and commentaries – MMRM vs LOCF by Naitee Ting”	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 treatment effects in early-phase randomized clinical trials involving external control data	1
On approximate equality of Z-values of the statistical tests for win statistics (win ratio, win odds, and net benefit)	1
An enriched approach to combining high-dimensional genomic and low-dimensional phenotypic data	1
Bayesian analyses of multiple random change points in survival models with applications to clinical trials	1
Covariate adjusted meta-analytic predictive (CA-MAP) prior for historical borrowing using patient-level data	1
Bigger and bigger circles - the expanding biopharmaceutical statistician’s toolbox	1
Sample size estimation for recurrent event data using multifrailty and multilevel survival models	1
Optimization of EWOC principle in BLRM design for phase 1 oncology trials	1
Beyond p < .05: a critical review of new Bayesian proposals for assessing the p-value	1
RESTART trial design: two-stage seamless transition design with operational considerations	1
Do strict decision criteria hamper productivity in the pharmaceutical industry?	1

Optimum designs for clinical trials in personalized medicine when response variance depends on treatment	1
Adaptively leverage multiple real-world data sources for treatment effect estimation based on similarity	0
Bayesian censored piecewise regression mixture models with skewness	0
Assessing adverse events in clinical trials during the era of the COVID-19 pandemic	0
Isotonic design for phase I cancer clinical trials with late-onset toxicities	0
Comparison of statistical methodologies used to estimate the treatment effect on time-to-event outcomes in observational studies	0
Pediatric needs should be considered early in the clinical development lifecycle	0
An investigation to improve a nonlinear mixed-effects approach for EC50 estimation based on multi-donor dose–response data	0
Deep historical borrowing framework to prospectively and simultaneously synthesize control information in confirmatory clinical trials with multiple endpoints	0
A multiple imputation approach in enhancing causal inference for overall survival in randomized controlled trials with crossover	0
Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease	0
Integrating real world data and clinical trial results using survival data reconstruction and marginal moment-balancing weights	0
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials	0
Two-stage designs with small sample sizes	0
Missing data in the eligibility criteria of synthetic controls from real-world data	0
Statistical learning in preclinical drug proarrhythmic assessment	0
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing	0
Response to letter to editor	0
Assessing the hierarchical beta-binomial model as a basic information sharing tool in basket trials	0
Reweighting estimators to extend the external validity of clinical trials: methodological considerations	0
The effect of misclassification on sample size for one and two-sample tests with binary endpoints	0
Considerations for master protocols using external controls	0
Modelling non-linear patterns of time-varying intervention effects on recurrent events in infectious disease prevention studies	0
Bayesian design of clinical trials using the scale transformed power prior	0
Prediction of vedolizumab treatment outcomes by machine learning	0
DOD-BART: machine learning-based dose optimization design incorporating patient-level prognostic factors via Bayesian additive regression trees	0
Retrieved-Dropout-Based multiple imputation for time-to-event data in cardiovascular outcome trials	0
Bayesian hierarchical model for dose-finding trial incorporating historical data	0
Non-inferiority testing for qualitative microbiological methods:Assessing and improving the approach in USP <1223>	0
Analysis of continuous monitoring device data	0
Meta inference of heterogeneous data streams	0
Revolutionizing cardiovascular disease classification through machine learning and statistical methods	0
Equivalence tests before end of follow-up under the class of log transformation model	0
Joint comparison of the predictive values of multiple binary diagnostic tests: an extension of McNemar’s test	0
Challenges and potential strategies utilizing external data for efficacy evaluation in small-sized clinical trials	0
Estimation of median survival time and its 95% confidence interval using SAS PROC LIFETEST	0
A Two-Stage Decision Making Approach for Safety Studies	0
Conditional power in vaccine trials with seasonal variations	0
Issues in cox proportional hazards model with unequal randomization	0
Valid overall odds ratio estimators using different stratified sampling schemes	0
The weighted log-rank tests based on stratified clustered survival data: saddle-point p-values and confidence intervals	0
Composite Likelihoods with Bounded Weights in Extrapolation of Data	0
A randomized Bayesian optimal phase II design with binary endpoint	0
Homogeneity test of several covariance matrices with high-dimensional data	0
The use of real-world data for clinical investigation of effectiveness in drug development	0
Survival stacking with multiple data types using pseudo-observation-based-AUC loss	0
Sequential monitoring of cancer immunotherapy trial with random delayed treatment effect	0
On variance estimation of target population created by inverse probability weighting	0
Statistical considerations for using tolerance interval to set product specification for normally distributed attribute	0
Bayesian sample size determination in a three-arm non-inferiority trial with binary endpoints	0
The win odds: statistical inference and regression	0
Multiple test procedures of disease prevalence based on stratified partially validated series in the presence of a gold standard	0
FDA experiences with a centralized statistical monitoring tool	0
P-values and confidence intervals of linear rank tests for left-truncated data under truncated binomial design	0
Flexible seamless 2-in-1 design with sample size adaptation	0
Pharmacometrics-Enabled DOse OPtimization (PEDOOP) for seamless phase I-II trials in oncology	0
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	0
Lessons learned in the Apple Heart Study and implications for the data management of future digital clinical trials	0
Correction	0
Generalized exponentiated unit Gompertz distribution for modeling arthritic pain relief times data: classical approach to statistical inference	0
Regional consistency assessment in multiregional clinical trials	0
Clustering plasma concentration-time curves: applications ofunsupervised learning in pharmacogenomics	0
Miettinen and Nurminen score statistics revisited	0
Generalized triple outcome decision-making in basket trials	0
On dependence assumption in p-value based multiple test procedures	0
Assessing the incidence and severity of drug adverse events: a Bayesian hierarchical cumulative logit model	0
Imputation of Missing Covariates in Randomized Controlled Trials with Continuous Outcomes: Simple, Unbiased and Efficient Methods	0
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	0
Application of estimand framework to the design and analysis of multi-regional clinical trials	0
Confidence interval construction for proportion difference from partially validated series with two fallible classifiers	0
Assessing the use of GEE methods for analyzing binary outcomes in family studies: the Strong Heart Family Study	0
Dissecting the restricted mean time in favor of treatment	0
Guest editors’ note on special issue on application of estimand	0
A Bayesian joint model for multivariate longitudinal and time-to-event data with application to ALL maintenance studies	0
Bayesian method for comparing F1 scores in the absence of a gold standard	0
What is the expected benefit of patient-centric clinical development in oncology?	0
Combination MCP-Mod for two-drug combination dose-ranging studies	0
The value of a two-armed Bayesian response adaptive randomization trial	0
A Bayesian phase I–II clinical trial design to find the biological optimal dose on drug combination	0
Investigating the impact of data monitoring committee recommendations on the probability of trial success	0
Developing large language models to detect adverse drug events in posts on x	0
Sample size determination for a study with variable follow-up time	0
Characterization of a credibility index	0
Letter to the editor: Inconsistencies between example data and calculation results in Bland, J. M., & Altman, D. G. (2007). Agreement between methods of measurement with multiple observations per	0
Robust estimates of regional treatment effects in multiregional randomized clinical trials with ordinal responses	0
genRCT: a statistical analysis framework for generalizing RCT findings to real-world population	0
On the feasibility of pediatric dose-finding trials in small samples with information from a preceding trial in adults	0
On the use of RWD in support of regulatory submission in drug development	0
Drug safety assessment by machine learning models	0
Investigating pharmacokinetic profiles of Centella asiatica using machine learning and PBPK modelling	0
Adaptive Multiple Comparison Sequential Design (AMCSD) for clinical trials	0
Leverage multiple real-world data sources in single-arm medical device clinical studies	0
Defective regression models for cure rate data with competing risks	0
BOP2-TE: Bayesian optimal phase 2 design for jointly monitoring efficacy and toxicity with application to dose optimization	0
Using Bayesian hierarchical models for controlled post hoc subgroup analysis of clinical trials: application to smoking cessation treatment in American Indians and Alaska Natives	0