OOIR: Observatory of International Research

Papers

(The median citation count of Statistics in Biopharmaceutical Research is 1. 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 2022-01-01 to 2026-01-01.)

Article	Citations
Treatment Selection at Interim Analysis Based on a Correlated Early Endpoint in Adaptive Two-Stage Trials with a Primary Endpoint Based on Count Data	36
U-MET: Utility-Based Dose Optimization Approach for Multiple-Dose Randomized Trial Designs	23
Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing	16
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	16
A landscape assessment of key evidence needs in study design and statistical methodologies for HTA submissions	14
Consistency Based Multiplicity Adjustment Approach—Multiple Doses in Phase III Studies	12
Considerations on Interim Evaluation of OS in Pivotal Oncology Trials	12
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	11
Modified Simon’s Two-Stage Design for Phase IIA Clinical Trials in Oncology—Dynamic Monitoring and More Flexibility	11
We Need Subject Matter Expertise to Choose and Identify Causal Estimands: Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	10
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	10
Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction	10
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	9
Some Group Sequential Trials from Industry over the Last 30 Years	9
Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective	9
The Current Landscape of HTA Framework and Key Challenges	9
A Basket Trial Design Based on Power Priors	9
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration	8
LORDs: Locally Optimal Restricted Designs for Phase I/II Dose-Finding Studies	8
Joint Analysis of Longitudinal Data and Zero-Inflated Recurrent Events	8
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	8
Augmenting the Control Arm of Randomized Trials by Incorporating Multiple External Data Sources Using Propensity Score Stratification and Data-Driven Mixture Prior	8
Optimizing Pediatric Dose Finding: A Phase I/II Design Integrating Adult Data	8
Monitoring Ongoing Clinical Trials Under Fractional Brownian Motion With Drift	8
Closing a Chapter: A Farewell from the Editor of Statistics in Biopharmaceutical Research	7

MT-Keyboard: A Bayesian Model-Assisted Interval Design to Account for Toxicity Grades and Types for Phase I Trials	7
Backward Sequential Significance Testing in Survival Trials	7
A New Centralized Statistical Monitoring Method for Detecting Atypical Distribution of Qualitative Variables in Multicenter Randomized Controlled Trials	6
Pre-Specified Safety Analysis of OS Data for Trials in Indolent or Early-Stage Cancers	6
Exact Matching as an Alternative to Propensity Score Matching	6
Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-Arm Trials	6
Bayesian Hierarchical Model with Adaptive Similarity Evaluation of Treatment Effects in Oncology Basket Trials	6
Statistics in Biopharmaceutical Research Best Papers Award 2023	6
Novel Statistical Designs and Considerations to Support Diversity and Inclusion in Clinical Trials	6
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	5
Treatment-Control Comparisons in Platform Trials Including Non-Concurrent Controls	5
Bayesian Basket Trial Design Accounting for Multiple Cutoffs of an Ambiguous Biomarker	5
Nonconcurrent Controls in Platform Trials: Can We Borrow Their Concurrent Observation Data?	5
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	5
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	5
Editor’s Note	5
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	5
Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial	4
On Variance Estimation for the One-Sample Log-Rank Test	4
Adaptive Endpoints Selection with Application in Rare Disease	4
Rejoinder to Commentaries on the “Monitoring OS in Pivotal Trials in Indolent Cancers” Article	4
Statistical Thinking and Innovation with Global Impact—Special Issue for the 2023 Regulatory-Industry Statistics Workshop	4
A Primer of Quasi-Exact Tests for Randomized Clinical Trials with Binary Endpoints	4
Estimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments	4
Evaluating the Impact of Outcome Delay on the Efficiency of Two-Arm Group-Sequential Trials	3
Assessing the Commonly Used Assumptions in Estimating the Principal Causal Effect in Clinical Trials	3
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	3
Comment on “Monitoring Overall Survival in Pivotal Trials in Indolent Cancers”	3
The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions	3
A Rényi-Divergence-Based Family of Metrics for the Evaluation of Surrogate Endpoints in a Causal Inference Framework	3
An Alternative to Traditional Sample Size Determination for Small Patient Populations	3
The Use of Machine Learning in Regulatory Drug Safety Evaluation	3
A Multi-Arm Multi-Stage Group Sequential Phase 2/3 Design with Dose Selection for Oncology Trials	3
Bayesian Optimal Phase II Design for Randomized Clinical Trials	3
A Simulation Study on the Estimation of the Effect in the Hypothetical Scenario of No Use of Symptomatic Treatment in Trials for Disease-Modifying Agents for Alzheimer’s Disease	3
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies	3
Correction	3
Rejoinder to Letter to the Editor—Selecting an Optimal Design for a Nonrandomized Comparative Study: A Commentary on “Some Considerations on Design and Analysis Plan on a Nonrandomized Comparative Stu	3
A Comprehensive Bayesian Approach to Quantify and Compare Benefit-Risk of Medical Products	3
Use of Common Control Arms Across Substudies in a Master Protocol via Ridge Estimation	3
Random Effects Meta-Analysis of Contingency Tables with Complete and Partially Complete Data, with Application to COVID-19 Research	2
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials	2
Non-Inferiority Testing on the Accuracy of Two Qualitative Microbiological Methods on Paired Binary Data	2
Three-Way Mixed Effect ANOVA to Estimate MRMC Limits of Agreement	2
Monitoring Overall Survival in Phase 3 Indolent Cancers: An Extended Framework and an Example	2
Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	2
Coping with Information Loss and the Use of Auxiliary Sources of Data: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	2
Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators	2
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development	2
Dionne Price: An Esteemed Colleague, Inspirational Leader, Trailblazer Statistician and a Dear Friend	2

Vaccine Development during a Pandemic: General Lessons for Clinical Trial Design	2
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials	2
A Modification of Location Commensurate Power Prior in Clinical Trials	2
Targeted Learning: Toward a Future Informed by Real-World Evidence	2
Chasing Shadows: How Implausible Assumptions Skew Our Understanding of Causal Estimands	2
Balancing the Objectives of Statistical Efficiency and Allocation Randomness in Randomized Controlled Trials	2
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	2
The Role of Statistical Thinking in Biopharmaceutical Research	2
Subgroup Analyses in Confirmatory Clinical Trials Using Bayesian Hierarchical Models	2
Modified Robust Meta-Analytic-Predictive Priors for Incorporating Historical Controls in Clinical Trials	2
Assessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models	2
Trial Design with Win Statistics for Multiple Time-to-Event Endpoints with Hierarchy	2
A Quantitative Bias Analysis to Assess Constancy Assumption in Noninferiority Trials Using Bayesian Hierarchical Models	2
Beyond the Classical Type I Error: Bayesian Metrics for Bayesian Designs Using Informative Priors	1
Quantifying Replicability and Consistency in Systematic Reviews	1
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
Comment on “Non-Proportional Hazards – an Evaluation of the MaxCombo Test in Cancer Clinical Trials” by the Cross-Pharma Non-Proportional Hazards Working Group	1
A Method for Ensuring a Consistent Dose–Response Relationship Between An Entire Population and One Region in Multiregional Dose–Response Studies Using MCP-Mod	1
A Bayesian Adaptive Umbrella Trial Design with Robust Information Borrowing for Screening Multiple Combination Therapies	1
Rejoinder: Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Bayesian Optimal Designs for Multi-Arm Multi-Stage Phase II Randomized Clinical Trials with Multiple Endpoints	1
The Statistical and Practical Implications of Mis-Stratified Randomization in Clinical Trials	1
Good Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder)	1
Multiple Surrogates in the Meta-Analytic Setting for Normally Distributed Endpoints	1
Generalized Likelihood Ratios for Designing Dose Optimization Studies of Targeted Therapies	1
Adjusting for Time-Varying Treatment Switches in Randomized Clinical Trials: The Danger of Extrapolation and How to Address It	1
Comparison of g-Estimation Approaches for Handling Symptomatic Medication at Multiple Timepoints in Alzheimer’s Disease with a Hypothetical Strategy	1
Bayesian Design of Superiority Trials: Methods and Applications	1
Shrinkage Estimation Methods for Subgroup Analyses	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Hybrid Control Design with Commensurate Prior Constructed Using Propensity Score-Matched External Controls: A Simulation Study	1
Bayesian Shrinkage Estimation of Credible Subgroups for Count Data with Excess Zeros	1
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development	1
Conditional and Unconditional Treatment Effects in Randomized Clinical Trials: Estimands, Estimation, and Interpretation	1
Editor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes	1
Rejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	1
Editor’s Note	1
Improved Trimmed Weighted Hochberg Procedures With Two Endpoints and Sample Size Optimization	1
Bayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers	1
Using Randomization Tests to Address Disruptions in Clinical Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes	1
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score	1
Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes	1
Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	1
Evaluation of Current Statistical Methods for Implementing Quality Tolerance Limits	1
Meta-Analysis of Moxifloxacin Concentration-QTc Effects with Application to Assay Sensitivity Assessment	1
Covariate-Adjusted Response Adaptive Designs for Competing Risk Survival Models	1
A Composite Endpoint for Treatment Benefit According to Patient Preference	1
Type I Error Control of an Adaptive Endpoint Selection Procedure	1
An Unbiased Method to Approximate a Principal Estimand	1
Eliciting the Discount Parameter in a Power Prior Method on the Basis of the Type I Error Consideration	1
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making	1
Assessing the Harm and Benefit of Cancer Therapies with an Overall Survival Endpoint in Comparative Clinical Studies	1
Sample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial	1
Systematic Comparison of Bayesian Basket Trial Designs with Unequal Sample Sizes and Proposal of a New Method Based on Power Priors	1
Remembering Gregory Campbell (1949–2023): An Accomplished Leader, Mentor, and Biostatistical Innovator	1
Assessment of Treatment Effect Heterogeneity for Multiregional Randomized Clinical Trials	1
A Statistical Method for Protocol Modifications With Heterogeneous Population Variances	1
Bayesian Dose Finding Model using Ordinal Endpoints	1
Qualitative versus Quantitative Treatment-by-Subgroup Interaction in Equivalence Studies with Multiple Subgroups	1