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 2020-11-01 to 2024-11-01.)

Article	Citations
The Current Landscape in Biostatistics of Real-World Data and Evidence: Causal Inference Frameworks for Study Design and Analysis	34
Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group	31
Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment	25
The Current Landscape in Biostatistics of Real-World Data and Evidence: Clinical Study Design and Analysis	21
Use of Nonconcurrent Common Control in Master Protocols in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion	16
Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event	14
Statistical Models for Composite Endpoints of Death and Nonfatal Events: A Review	14
Reference-Based Multiple Imputation—What is the Right Variance and How to Estimate It	13
Developing a Targeted Learning-Based Statistical Analysis Plan	13
Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods	12
Using Surrogate Endpoints in Adaptive Designs with Delayed Treatment Effect	11
Deep Learning Based Multimodal Progression Modeling for Alzheimer’s Disease	11
Efficiency Comparison of Analysis Methods for Recurrent Event and Time-to-First Event Endpoints in the Presence of Terminal Events—Application to Clinical Trials in Chronic Heart Failure	10
Quantifying Efficiency Gains of Innovative Designs of Two-Arm Vaccine Trials for COVID-19 Using an Epidemic Simulation Model	10
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators	9
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	9
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	8
Targeted Learning: Toward a Future Informed by Real-World Evidence	8
The Strong Null Hypothesis and the MaxCombo Test: Comment on “Robust Design and Analysis of Clinical Trials with Nonproportional Hazards: A Straw Man Guidance form a Cross-Pharma Working Group.”	7
Type I Error Considerations in Master Protocols With Common Control in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion	7
Nonproportional Hazards—An Evaluation of the MaxCombo Test in Cancer Clinical Trials	7
A Seamless Adaptive 2-in-1 Design Expanding a Phase 2 Trial for Treatment or Dose Selection Into a Phase 3 Trial	7
Incorporating Surrogate Information for Adaptive Subgroup Enrichment Design with Sample Size Re-Estimation	6
A Comparison of Different Approaches to Bayesian Hierarchical Models in a Basket Trial to Evaluate the Benefits of Increasing Complexity	6
Quantifying Replicability and Consistency in Systematic Reviews	6

Missing Data Imputation With Baseline Information in Longitudinal Clinical Trials	6
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development	6
A Generalized Framework of Optimal Two-Stage Designs for Exploratory Basket Trials	6
A Phase 3 Trial Analysis Proposal for Mitigating the Impact of the COVID-19 Pandemic	6
Editorial: Roles of Hypothesis Testing, p-Values and Decision Making in Biopharmaceutical Research	6
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials	6
Statistical Observations on Vaccine Clinical Development for Pandemic Diseases	6
Bayesian Optimal Phase II Design for Randomized Clinical Trials	6
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	5
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	5
Testing the Equality of Proportions for Combined Unilateral and Bilateral Data Under Equal Intraclass Correlation Model	5
Predicting the COVID-19 Pandemic Impact on Clinical Trial Recruitment	5
Planning for the Next Pandemic: Ethics and Innovation Today for Improved Clinical Trials Tomorrow	5
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”	5
Statistical Opportunities to Accelerate Development for COVID-19 Therapeutics	5
Application of Group Sequential Methods to the 2-in-1 Design and Its Extensions for Interim Monitoring	5
Estimands in Real-World Evidence Studies	4
Our Most Important Discovery: The Question	4
Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation	4
Bootstrap Cross-validation Improves Model Selection in Pharmacometrics	4
Regulatory Guidance on Randomization and the Use of Randomization Tests in Clinical Trials: A Systematic Review	4
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	4
Generalizing Clinical Trial Results to a Target Population	4
Selecting an Optimal Design for a Non-randomized Comparative Study: A Comment on “Some Considerations on Design and Analysis Plan on a Nonrandomized Comparative Study Utilizing Propensity Score Method	4
Combining the Target Trial and Estimand Frameworks to Define the Causal Estimand: An Application Using Real-World Data to Contextualize a Single-Arm Trial	4
Testing and Interpreting the “Right” Hypothesis—Comment on “Non-proportional Hazards — An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	4
Radical Thinking: Scientific Rigor and Pragmatism	4
Discrete Time Multistate Model With Regime Switching for Modeling COVID-19 Disease Progression and Clinical Outcomes	4
Methods for Informative Censoring in Time-to-Event Data Analysis	3
Mitigating Study Power Loss Caused by Clinical Trial Disruptions Due to the COVID-19 Pandemic: Leveraging External Data via Propensity Score-Integrated Approaches	3
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials	3
Hierarchical Generalized Linear Models for Multiregional Clinical Trials	3
Statistical Inference on the Estimators of the Adherer Average Causal Effect	3
Examples of Applying RWE Causal-Inference Roadmap to Clinical Studies	3
Some Biostatistical Considerations About Real-World Data and Evidence in Clinical Studies, Especially for Regulatory Purposes	3
On Identification of the Principal Stratum Effect in Patients Who Would Comply If Treated	3
Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators	3
Simulation-Based Pharmacokinetics Sampling Design for Evaluating Correlates of Prevention Efficacy of Passive HIV Monoclonal Antibody Prophylaxis	3
Bayesian Change-Point Joint Models for Multivariate Longitudinal and Time-to-Event Data	3
Multivariate Shared-Parameter Mixed-Effects Location Scale Model for Analysis of Intensive Longitudinal Data	3
Quick Multiple Test Procedures and p-Value Adjustments	3
A Composite Endpoint for Treatment Benefit According to Patient Preference	3
Value Function Guided Subgroup Identification via Gradient Tree Boosting: A Framework to Handle Multiple Outcomes for Optimal Treatment Recommendation	3
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”	3
Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes	3
What Can Be Achieved with the Estimand Framework?	3
Applying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management	3
Bayesian Approaches for Handling Hypothetical Estimands in Longitudinal Clinical Trials With Gaussian Outcomes	3
Post-Test Diagnostic Accuracy Measures of a Continuous Test With a Disease of Ordinal Multistages	3
It’s the Selection’s Fault—Not the p-Values’: A Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	3

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”	2
An Extended Youden Design in Biological Assays	2
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real?	2
The Use of Machine Learning in Regulatory Drug Safety Evaluation	2
Adaptive Endpoints Selection with Application in Rare Disease	2
Beyond the Classical Type I Error: Bayesian Metrics for Bayesian Designs Using Informative Priors	2
The Choice of a Randomization Procedure in Survival Studies with Nonproportional Hazards	2
Least Conservative Critical Boundaries of Multiple Hypothesis Testing in a Range of Correlation Values	2
Conditional and Unconditional Treatment Effects in Randomized Clinical Trials: Estimands, Estimation, and Interpretation	2
How Many Cohorts Should Be Considered in an Exploratory Master Protocol?	2
Waking up to p: Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	2
Optimal Spiking Experiment for Noninferiority of Qualitative Microbiological Methods on Accuracy With Multiple Microorganisms	2
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	2
Three-Way Mixed Effect ANOVA to Estimate MRMC Limits of Agreement	2
Using Randomization Tests to Address Disruptions in Clinical Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	2
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	2
Efficient Multiple Imputation for Sensitivity Analysis of Recurrent Events Data with Informative Censoring	2
Comment on “Non-Proportional Hazards – an Evaluation of the MaxCombo Test in Cancer Clinical Trials” by the Cross-Pharma Non-Proportional Hazards Working Group	2
Dynamic Monitoring of Ongoing Clinical Trials	2
Bayesian Design of Superiority Trials: Methods and Applications	2
An Empirical Comparison of Segmented and Stochastic Linear Mixed Effects Models to Estimate Rapid Disease Progression in Longitudinal Biomarker Studies	2
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	2
Qualitative versus Quantitative Treatment-by-Subgroup Interaction in Equivalence Studies with Multiple Subgroups	2
Dose Optimization for Novel Oncology Agents: Design Options and Strategies	2
p-Values and Replicability: A Commentary on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	2
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	2
A Sequential Predictive Power Design for a COVID Vaccine Trial	2
Are p-values Useful to Judge the Evidence Against the Null Hypotheses in Complex Clinical Trials? A Comment on “The Role of p-values in Judging the Strength of Evidence and Realistic Rep	2
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score	2
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Sequential Bayes Factors for Sample Size Reduction in Preclinical Experiments with Binary Outcomes	1
The Targeted Virtual Control Approach for Single-Arm Clinical Trials with External Controls	1
Bayesian Basket Trial Design Accounting for Multiple Cutoffs of an Ambiguous Biomarker	1
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
Balancing the Objectives of Statistical Efficiency and Allocation Randomness in Randomized Controlled Trials	1
Comment on “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
A Test of the Dependence Assumptions for the Simes-Test-based Multiple Test Procedures	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
Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	1
Similarity-Principle-Based Machine Learning Method for Clinical Trials and Beyond	1
A Closer Look at the Kernels Generated by the Decision and Regression Tree Ensembles	1
A Comparison of Randomization Methods for Multi-Arm Clinical Trials	1
Statistical Issues and Challenges in Clinical Trials for COVID-19 Treatments, Vaccines, Medical Devices and Diagnostics	1
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	1
Treatment Comparisons in Adaptive Platform Trials Adjusting for Temporal Drift	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	1
Trigger Strategy in Repeated Tests on Multiple Hypotheses	1
Blinded Sample Size Re-Estimation in Comparative Clinical Trials With Overdispersed Count Data: Incorporation of Misspecification of the Variance Function	1
Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	1
Comparison of Adverse Event Risks in Randomized Controlled Trials with Varying Follow-Up Times and Competing Events: Results from an Empirical Study	1
Strategy for Combining Information from Real World Data Sources When Individual Patient Data Are Not Simultaneously Accessible	1
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	1
The Role of Statistical Thinking in Biopharmaceutical Research	1
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	1
Survey and Recommendations on the Use ofP-Values Driving Decisions in Nonclinical Pharmaceutical Applications	1
Sequential Multiple Assignment Randomized Trials for COMparing Personalized Antibiotic StrategieS (SMART COMPASS): Design Considerations	1
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making	1
Evaluating Misclassification Effects on Single Sequential Treatment in Sequential Multiple Assignment Randomized Trial (SMART) Designs	1
Bayesian Borrowing Methods for Count Data: Analysis of Incontinence Episodes in Patients with Overactive Bladder	1
On the Implementation of Robust Meta-Analytical-Predictive Prior	1
Validity of Tests for Time-to-Event Endpoints in Studies with the Pocock and Simon Covariate-Adaptive Randomization	1
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	1
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development	1
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	1
Teasing Out the Overall Survival Benefit With Adjustment for Treatment Switching to Multiple Treatments	1
Statement on P-values	1
The Role of FDA CDER Statisticians in Response Efforts to the COVID-19 Pandemic	1
Copula-Based Model for Incorporating Single-Agent Historical Data into Dual-Agent Phase I Cancer Trials	1
An Expected Score Approach to Ordinal Outcomes in a Bayesian, Response Adaptive, Randomized Trial	1
A Bayesian Decision-Theoretic Design for Simultaneous Biomarker-Based Subgroup Selection and Efficacy Evaluation	1
A Test for Treatment Effects Based on the Exact Distribution of an Ordinary Least-Square Estimator in Sequential Parallel Comparison Design	1
Rejoinder to Letter to the Editor on “A New Principal Stratum Estimand Investigating the Treatment Effect in Patients Who Would Comply, If Treated With a Specific Treatment”	1
Combined Tests Based on Restricted Mean Time Lost for Competing Risks Data	1
A Novel Approach for Modeling Biphasic Dose–Response Curves	1
Comment on “Good Data Science Practice: Moving towards a Code of Practice for Drug Development”	1
A Flexible Analytical Framework for Reference-Based Imputation, Delta Adjustment and Tipping-Point Stress-Testing	1
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	1