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 2021-05-01 to 2025-05-01.)

Article	Citations
Composite Endpoints in Cardio-Renal Clinical Outcome Trials	26
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	19
Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing	19
U-MET: Utility-Based Dose Optimization Approach for Multiple-Dose Randomized Trial Designs	17
Use of Nonconcurrent Common Control in Master Protocols in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion	15
Statistical Inference of the Probability of Passing the USP Dissolution Test	14
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	13
Modified Simon’s Two-Stage Design for Phase IIA Clinical Trials in Oncology—Dynamic Monitoring and More Flexibility	11
Consistency Based Multiplicity Adjustment Approach—Multiple Doses in Phase III Studies	11
The current landscape of HTA framework and key challenges	9
A Basket Trial Design Based on Power Priors	9
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”	9
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	9
Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective	8
Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction	8
Statistics in Biopharmaceutical Research Best Papers Award 2023	7
Some Group Sequential Trials from Industry over the Last 30 Years	7
On the Implementation of Robust Meta-Analytical-Predictive Prior	7
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	7
Statistical Issues and Challenges in Clinical Trials for COVID-19 Treatments, Vaccines, Medical Devices and Diagnostics	7
Monitoring Ongoing Clinical Trials Under Fractional Brownian Motion With Drift	7
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration	7
Backward Sequential Significance Testing in Survival Trials	7
Joint Analysis of Longitudinal Data and Zero-Inflated Recurrent Events	7
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	7

Bayesian Hierarchical Model with Adaptive Similarity Evaluation of Treatment Effects in Oncology Basket Trials	6
Reference-Based Multiple Imputation—What is the Right Variance and How to Estimate It	6
MT-Keyboard: A Bayesian Model-Assisted Interval Design to Account for Toxicity Grades and Types for Phase I Trials	6
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators	6
A New Centralized Statistical Monitoring Method for Detecting Atypical Distribution of Qualitative Variables in Multicenter Randomized Controlled Trials	6
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	5
Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-Arm Trials	5
A Primer of Quasi-Exact Tests for Randomized Clinical Trials with Binary Endpoints	5
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	5
Our Most Important Discovery: The Question	5
Editor’s Note	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
A Comparison of Different Approaches to Bayesian Hierarchical Models in a Basket Trial to Evaluate the Benefits of Increasing Complexity	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
Adaptive Endpoints Selection with Application in Rare Disease	5
Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial	5
Estimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments	4
Correction	4
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	4
On Variance Estimation for the One-Sample Log-Rank Test	4
Multiarmed Bandit Designs for Phase I Dose-Finding Clinical Trials With Multiple Toxicity Types	4
Treatment-control comparisons in platform trials including non-concurrent controls	4
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	4
The Use of Machine Learning in Regulatory Drug Safety Evaluation	4
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real?	3
Applying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management	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
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
Bayesian Optimal Phase II Design for Randomized Clinical Trials	3
Closed-Form Approximation of Correlation Matrix Among Fleming Harrington Test Statistics in MaxCombo Test: Comments on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A St	3
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies	3
The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions	3
Assessing the Commonly Used Assumptions in Estimating the Principal Causal Effect in Clinical Trials	3
Comment on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group”: The Test Statistic Should Estimate Some Reasonable Meas	3
An Alternative to Traditional Sample Size Determination for Small Patient Populations	3
Random Effects Meta-Analysis of Contingency Tables with Complete and Partially Complete Data, with Application to COVID-19 Research	3
Comment on “Monitoring Overall Survival in Pivotal Trials in Indolent Cancers”	3
Rejoinder to Commentaries on the “Monitoring OS in Pivotal Trials in Indolent Cancers” Article	3
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
Balancing the Objectives of Statistical Efficiency and Allocation Randomness in Randomized Controlled Trials	2
Generalizing Clinical Trial Results to a Target Population	2
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development	2
Chasing Shadows: How Implausible Assumptions Skew Our Understanding of Causal Estimands	2
Rejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	2
Assessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models	2
Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	2
A Quantitative Bias Analysis to Assess Constancy Assumption in Noninferiority Trials Using Bayesian Hierarchical Models	2
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	2
A Rényi-Divergence-Based Family of Metrics for the Evaluation of Surrogate Endpoints in a Causal Inference Framework	2

Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators	2
A Method for Ensuring a Consistent Dose–Response Relationship Between An Entire Population and One Region in Multiregional Dose–Response Studies Using MCP-Mod	2
Trial Design with Win Statistics for Multiple Time-to-Event Endpoints with Hierarchy	2
Value Function Guided Subgroup Identification via Gradient Tree Boosting: A Framework to Handle Multiple Outcomes for Optimal Treatment Recommendation	2
Targeted Learning: Toward a Future Informed by Real-World Evidence	2
The Role of Statistical Thinking in Biopharmaceutical 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
Modified Robust Meta-Analytic-Predictive Priors for Incorporating Historical Controls in Clinical Trials	2
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials	2
Combined Tests Based on Restricted Mean Time Lost for Competing Risks Data	2
Vaccine Development during a Pandemic: General Lessons for Clinical Trial Design	2
Quick Multiple Test Procedures and p-Value Adjustments	1
Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes	1
Editor’s Note	1
Three-Way Mixed Effect ANOVA to Estimate MRMC Limits of Agreement	1
An Unbiased Method to Approximate a Principal Estimand	1
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score	1
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”	1
Sample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial	1
The Choice of a Randomization Procedure in Survival Studies with Nonproportional Hazards	1
Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes	1
Bayesian Approaches for Handling Hypothetical Estimands in Longitudinal Clinical Trials With Gaussian Outcomes	1
Statistical Models for Composite Endpoints of Death and Nonfatal Events: A Review	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Adjusting for Time-Varying Treatment Switches in Randomized Clinical Trials: The Danger of Extrapolation and How to Address It	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
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
Covariate-Adjusted Response Adaptive Designs for Competing Risk Survival Models	1
Bayesian Optimal Designs for Multi-Arm Multi-Stage Phase II Randomized Clinical Trials with Multiple Endpoints	1
Eliciting the Discount Parameter in a Power Prior Method on the Basis of the Type I Error Consideration	1
Editor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes	1
A Statistical Method for Protocol Modifications With Heterogeneous Population Variances	1
Handling Intercurrent Events Through Hypothetical Strategy in Delayed-Start Designs	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
A Sequential Predictive Power Design for a COVID Vaccine Trial	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
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making	1
Bayesian Design of Superiority Trials: Methods and Applications	1
A Bayesian Adaptive Umbrella Trial Design with Robust Information Borrowing for Screening Multiple Combination Therapies	1
Statistical Observations on Vaccine Clinical Development for Pandemic Diseases	1
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
Conditional and Unconditional Treatment Effects in Randomized Clinical Trials: Estimands, Estimation, and Interpretation	1
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development	1
Type I Error Control of an Adaptive Endpoint Selection Procedure	1
A Modification of Location Commensurate Power Prior in Clinical Trials	1
A Composite Endpoint for Treatment Benefit According to Patient Preference	1
Planning for the Next Pandemic: Ethics and Innovation Today for Improved Clinical Trials Tomorrow	1
Remembering Gregory Campbell (1949–2023): An Accomplished Leader, Mentor, and Biostatistical Innovator	1
Bayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers	1
Beyond the Classical Type I Error: Bayesian Metrics for Bayesian Designs Using Informative Priors	1
Shrinkage Estimation Methods for Subgroup Analyses	1
Quantifying Replicability and Consistency in Systematic Reviews	1
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	1
Qualitative versus Quantitative Treatment-by-Subgroup Interaction in Equivalence Studies with Multiple Subgroups	1
Bayesian Shrinkage Estimation of Credible Subgroups for Count Data with Excess Zeros	1
Assessment of Treatment Effect Heterogeneity for Multiregional Randomized Clinical Trials	1