OOIR: Observatory of International Research

Papers

(The TQCC of Statistics in Biopharmaceutical Research is 3. 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
Statistical Models for Composite Endpoints of Death and Nonfatal Events: A Review	14
Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event	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
Quantifying Efficiency Gains of Innovative Designs of Two-Arm Vaccine Trials for COVID-19 Using an Epidemic Simulation Model	10
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
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
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators	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
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
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
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
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
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
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
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
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
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
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