OOIR: Observatory of International Research

Papers

(The TQCC of Journal of Biopharmaceutical Statistics is 2. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2022-08-01 to 2026-08-01.)

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
Estimating treatment effect in randomized trial after control to treatment crossover using external controls	12
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
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
scRAA: the development of a robust and automatic annotation procedure for single-cell RNA sequencing data	10
Correction	10
Whole-cage randomization for animal studies with unequal cage or group sizes	9
A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes	9
Estimand in benefit-risk assessment	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
Comparison of continuous, binary, and ordinal endpoints	6
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
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
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
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
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
Establishment of RWS guidance reflecting contributions of China to regulatory science	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
Impacts of reducing the number of hERG assay concentration levels on IC50 estimation	3
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

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
Statistical considerations for some issues in clinical bridging studies evaluating companion diagnostic devices	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
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
A win ratio approach for comparing crossing survival curves in 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
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
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