Clinical Trials

Article	Citations
Industry payments and brand-name tyrosine kinase inhibitor use amid generic entry	65
Treatment selection in multi-arm multi-stage designs: With application to a postpartum haemorrhage trial	54
A critique on “A randomized evaluation of on-site monitoring nested in a multinational randomized trial”	50
Stakeholder views about the responsibilities of principal investigators in multicenter randomized controlled trials	48
Perspectives of adolescents with neurofibromatosis 1 and cutaneous neurofibromas: Implications for clinical trials	28
Dose finding in early-phase human immunodeficiency virus type 1 prevention monoclonal antibody clinical trials	26
A review of current practice in the design and analysis of extremely small stepped-wedge cluster randomized trials	26
Topic-specific living databases of clinical trials: A scoping review of public databases	24
Performance of Cox regression models for composite time-to-event endpoints with component-wise censoring in randomized trials	22
Characterization of studies considered and required under Medicare’s coverage with evidence development program	22
A Bayesian adaptive design approach for stepped-wedge cluster randomized trials	22
Importance of patient engagement in the conduct of pragmatic multicenter randomized controlled trials: The ADAPTABLE experience	21
Training the next generation of clinical trial biostatisticians	20
How is missing data handled in cluster randomized controlled trials? A review of trials published in the NIHR Journals Library 1997–2024	20
Modeling impact of inflation reduction act price negotiations on new drug pipeline considering differential contributions of large and small biopharmaceutical companies	19
Partner engagement for planning and development of non-pharmacological care pathways in the AIM-Back trial	18
Unresolved issues with noninferiority pragmatic trials: Results of a literature survey	16
Sixteenth Annual University of Pennsylvania conference on statistical issues in clinical trial/optimizing dose-finding across the clinical trials spectrum (morning panel discussion)	15
Controlling the false-discovery rate when identifying the subgroup benefiting from treatment	15
Evaluating the use of text-message reminders and personalised text-message reminders on the return of participant questionnaires in trials, a systematic review and meta-analysis	15
Bayesian analysis in confirmatory clinical trials: A narrative review and discussion of current practice	14
Using the consolidated framework for implementation research to identify recruitment barriers and targeted strategies for a shared decision-making randomized clinical trial in pediatric sickle cell di	14
On what basis did Health Canada approve OxyContin in 1996? A retrospective analysis of regulatory data	14
Adaptive trial design and interim decision-making using incomplete longitudinal measurements: Methods and application to myasthenia gravis	13
Covariate adjustment in randomized trials: An overview	13

The patient perspective on dose optimization for anticancer treatments: A new era of cancer drug dosing—Challenging the “more is better” dogma	13
Commentary on DeMets et al: The need for greater transparency regarding data monitoring committee charters	12
A randomized control trial of re-designed and shorter research informed consent forms to improve comprehension	12
Over-accrual in Bayesian adaptive trials with continuous futility stopping	11
Data monitoring committees in pediatric randomized controlled trials registered in ClinicalTrials.gov	11
Sample size estimation for the averted events ratio	11
Policy recommendations for implementing registries to minimize over-volunteering in Phase I clinical trials	10
Sequential monitoring of time-to-event safety endpoints in clinical trials	9
Assessing the current utilization status of wearable devices in clinical research	9
Commentary: Multi-stage consent for time-sensitive clinical trials	9
Reconsidering stepped wedge cluster randomized trial designs with implementation periods: Fewer sequences or the parallel-group design with baseline and implementation periods are potentially more eff	9
Challenges in conducting efficacy trials for new COVID-19 vaccines in developed countries	8
A Bayesian adaptive design for clinical trials of rare efficacy outcomes with multiple definitions	8
Assessing institutional responsibility in scientific misconduct: A case study of enoximone research by Joachim Boldt	8
A hybrid approach to comparing parallel-group and stepped-wedge cluster-randomized trials with a continuous primary outcome when there is uncertainty in the intra-cluster correlation	8
Heterogeneity of surrogate outcome measures used in critical care studies: A systematic review	8
Response	8
The symbolic two-step method applied to cancer care delivery research: Safeguarding against designing an underpowered cluster randomized trial with a continuous outcome by accounting for the imprecisi	8
Comparison of outcomes of the 50-year follow-up of a randomized trial assessed by study questionnaire and by data linkage: The CONCUR study	8
The evolution of Data and Safety Monitoring Boards	8
What influences trust in and understanding of clinical trials? An analysis of information and communication technology use and online health behavior from the Health Information National Trends Survey	8
Scaling and interpreting treatment effects in clinical trials using restricted mean survival time	8
Salvaging information from paused or stopped clinical studies	7
Handling intercurrent events and missing data in non-inferiority trials using the estimand framework: A tuberculosis case study	7
Analysis of composite time-to-event endpoints in cardiovascular outcome trials	7
UK paediatric clinical trial protocols: A review of guidance for participant management and care in the event of premature termination	7
Premarket and postmarket real-world evidence studies supporting U.S. Food and Drug Administration regulatory decision-making, 2016–2024	7
Trial informed consent forms, the Declaration of Helsinki and the SPIRIT 2025 statement	7
Commentary on Wittes et al: Aspirin for primary prevention of CV events – Rationally robust? Statistically significant? Clinically convincing?	7
A hybrid automated event adjudication system for clinical trials	7
Proceedings of the University of Pennsylvania 17th annual conference on statistical issues in clinical trials: Covariate adjustment in randomized clinical trials: new methods and applications	7
Book Review – For the common good	7
Patient notification about pragmatic clinical trials conducted with a waiver of consent: A qualitative study	7
The Support, Educate, Empower personalized glaucoma coaching trial design	7
Estimating clinical trial hazard functions	7
Adjusting for covariates in randomized clinical trials for drugs and biological products	6
Desirability of outcome ranking (DOOR) analysis for multivariate survival outcomes with application to ACTT-1 trial	6
Estimands in clinical trials of complex disease processes	6
Impact of differences between interim and post-interim analysis populations on outcomes of a group sequential trial: Example of the MOVe-OUT study	6
The roles and professional competencies of clinical study coordinators and data managers in clinical trials: A systematic review	6
Causal interpretation of the hazard ratio in randomized clinical trials	6
Thirteenth annual UPenn conference on statistical issues in clinical trials: Cluster-randomized clinical trials—opportunities and challenges (afternoon panel session)	6
Reconsidering registration requirements for trials randomising health care providers	6
Adherence to key recommendations for design and analysis of stepped-wedge cluster randomized trials: A review of trials published 2016–2022	6
Developing a research coordinator workforce: A case study of a hospital and university collaboration	6
In-Conference Workshop	6
Optimizing the doses of cancer drugs after usual dose finding	5
Dynamic data-enabled stratified sampling for trial invitations with application in NHS-Galleri	5
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials	5
Public involvement in Australian clinical trials: A systematic review	5

A pilot recruitment strategy to enhance ethical and equitable access to Covid-19 pediatric vaccine trials	5
The improving Medication Adherence in Adolescents and young adults following Liver Transplantation (iMALT) multisite trial: Design and trial implementation considerations	5
Evidence supporting European Medicines Agency drug approvals (2020–2023): A cross-sectional study of trial design and outcomes	5
Shaping the future of clinical trials through strategic foresight	5
Lessons learned from conducting the first cancer care delivery trial in the Alliance for Clinical Trials in Oncology (Alliance A191402CD)	5
Site staff perspectives on communicating trial results to participants: Cost and feasibility results from the Show RESPECT cluster randomised, factorial, mixed-methods trial	5
Phase I design for partially ordered groups with late-onset toxicity	5
BASIC: A Bayesian adaptive synthetic-control design for phase II clinical trials	5
Covariate adjustment in randomized controlled trials: General concepts and practical considerations	5
Accounting for non-adherence: A re-analysis of the Liraglutide Effect and Action in Diabetes: Evaluation of Cardiovascular Outcome Results trial	5
Comparison of adaptive seamless Phase 2/3 designs for dose selection in clinical trials with multiple endpoints	5
Individualized clinical decisions within standard-of-care pragmatic clinical trials: Implications for consent	5
Now is the time to fix the evidence generation system	5
Estimating treatment effects in trials with outcome data truncated by death: A case study on aligning estimators with estimands	4
Factors associated with enrollment in a randomized clinical trial of docosahexaenoic supplementation in toddlers born preterm	4
Commentary on van Lancker et al	4
Demographic diversity of US-based participants in GSK-sponsored interventional clinical trials	4
Do recruitment SWAT interventions have an impact on participant retention in randomised controlled trials? A systematic review	4
A randomized study comparing patient portal and email communications for trial recruitment	4
Overall average treatment effects from clinical trials, one-variable-at-a-time subgroup analyses and predictive approaches to heterogeneous treatment effects: Toward a more patient-centered evidence-b	4
Afternoon discussion: Statistical issues in clinical trials conference on dose finding	4
Determining a risk-proportionate approach to the validation of statistical programming for clinical trials	4
Determining the sample size for a cluster-randomised trial using knowledge elicitation: Bayesian hierarchical modelling of the intracluster correlation coefficient	4
When should factorial designs be used for late-phase randomised controlled trials?	4
Chronic pain trials often exclude people with comorbid depressive symptoms: A secondary analysis of 346 randomized controlled trials	4
Optimising adherence to inpatient rehabilitation trial protocols: A mixed-methods systematic review	4
Standardising management of consent withdrawal and other clinical trial participation changes: The UKCRC Registered Clinical Trials Unit Network’s PeRSEVERE project	4
Designing a childhood obesity preventive intervention using the multiphase optimization strategy: The Healthy Bodies Project	4
From the ASPREE investigators: Response to Wittes et al.	4
Implementing a suicide risk management protocol as part of a multisite clinical trial: Findings and lessons learned	4
Machine learning to optimize precision in the analysis of randomized trials: A journey in pre-specified, yet data-adaptive learning	3
Approaches to ensure quality of information provision and consent processes for vaccine clinical trial participation in Sub-Saharan Africa: A scoping review	3
Aspirin in primary prevention: Undue reliance on an uninformative trial led to misinformed clinical guidelines	3
A single item for overall side effect impact: Association with clinician-reported adverse events and global health	3
Taking clinical decisions seriously in standard-of-care pragmatic clinical trials	3
Strategies to promote contraception use by female volunteers in Alzheimer’s Prevention Initiative Autosomal-Dominant Alzheimer’s Disease (API ADAD) Colombia trial	3
Statistical approaches for component-wise censored composite endpoints	3
Regulatory compliance and readability of informed consent forms in industry-sponsored drug development clinical trials	3
Overview of modern approaches for identifying and evaluating heterogeneous treatment effects from clinical data	3
The influence of political ideology on clinical trial knowledge, invitation, and participation among adults in the United States	3
Consent to recontact for future research using linked primary healthcare data: Outcomes and general practice perceptions from the ATHENA COVID-19 study	3
Precision medicine evaluation of heterogeneity of treatment effect for a time-to-event outcome with application in a trial of Initial treatment for people living with HIV	3
Identification and mitigation of a systematic analysis error in a multicenter dual-energy x-ray absorptiometry study	3
Concordance between clinical trial data use request proposals and corresponding publications: A cross-sectional study	3
Analysis of adaptive platform trials using a network approach	3
Algorithmic benchmark modulation: A novel method to develop success rates for clinical studies	3
An open-source SQL database schema for integrated clinical and translational data management in clinical trials	3
Critical importance of correctly defining and reporting secondary endpoints when assessing the ethics of research biopsies	3
Design and implementation of community consultation for research conducted under exception from informed consent regulations for the PreVent and the PreVent 2 trials: Changes over time and during the	3
The net benefit for time-to-event outcome in oncology clinical trials with treatment switching	3
Inference on subgroups identified based on a heterogeneous treatment effect in a post hoc analysis of a clinical trial	3
Design and logistical considerations for the randomized adaptive non-inferiority storage-duration-ranging CHIlled Platelet Study	3
Advancing neurofibromatosis and schwannomatosis clinical trial design: Consensus recommendations from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaborati	3
Defining estimand for the win ratio: Separate the true effect from censoring	3
Seamless monotherapy-combination phase I dose-escalation model-based design	2
Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1	2
14th Annual University of Pennsylvania Conference on statistical issues in clinical trials/subgroup analysis in clinical trials: Opportunities and challenges (afternoon panel discussion)	2
A survey on UK researchers’ views regarding their experiences with the de-identification, anonymisation, release methods and re-identification risk estimation for clinical trial datasets	2
Evaluating group-sequential non-inferiority clinical trials following interim stopping: The HIV Prevention Trials Network 083 trial	2
Building a professionally recognised clinical trial workforce: Is it time for an education and accreditation strategy?	2
Adaptive phase I–II clinical trial designs identifying optimal biological doses for targeted agents and immunotherapies	2
Causal inference in randomized trials with partial clustering	2
Abstracts from the Society for Clinical Trials 46th Annual Meeting (2025)	2
Strategies to facilitate adolescent access to medicines: Improving regulatory guidance	2
Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2–related schwannomatosis: Recommendations for clinical trials	2
Assessing the representativeness of cluster randomized trials: Evidence from two large pragmatic trials in United States nursing homes	2
The use of linked administrative data in Australian randomised controlled trials: A scoping review	2
Poster Presentations	2
Minimisation for the design of parallel cluster-randomised trials: An evaluation of balance in cluster-level covariates and numbers of clusters allocated to each arm	2
Covariate adjustment in randomized clinical trials: From general theory to practical insights	2
Central statistical monitoring in clinical trial management: A scoping review	2
Partially clustered designs for clinical trials: Unifying existing designs using consistent terminology	2
A comparison of different population-level summary measures for randomised trials with time-to-event outcomes, with a focus on non-inferiority trials	2
Practical inference for a complier average causal effect in cluster randomised trials with a binary outcome	2
Comparison of Bayesian and frequentist monitoring boundaries motivated by the Multiplatform Randomized Clinical Trial	2
Considerations for identifying the “right” subgroup in adaptive enrichment trials	2
The Bayesian Time Machine: Accounting for temporal drift in multi-arm platform trials	2
Hierarchical Bayesian modeling of heterogeneous outcome variance in cluster randomized trials	2
Subgroup analyses and pre-specification	2
Adaptive promising zone design for sequential parallel comparison design with continuous outcomes	2

Comparing meta-analysis and linear mixed model-based approaches for the analysis of continuous outcomes from batched stepped wedge trials	2
The impact of feedback training on prediction of cancer clinical trial results	2
New strategies for confirmatory testing of secondary hypotheses on combined data from multiple trials	2
Additional crossovers in cluster randomised crossover trials do not always increase statistical power	2
Comment on: Shaping the future of clinical trials through strategic foresight	1
Comment on “Causal interpretation of the hazard ratio in randomized clinical trials” by Fay and Li	1
The ethical value of consulting community members in non-emergency trials conducted with waivers of informed consent for research	1
Covariate-constrained randomization with cluster selection and substitution	1
Session Proposals	1
Meta-analytic evaluation of surrogate endpoints at multiple time points in randomized controlled trials with time-to-event endpoints	1
Design considerations for randomized comparisons of neoadjuvant–adjuvant versus adjuvant-only cancer immunotherapy when tumor measurement schedules do not align (SWOG S1801)	1
Navigating COVID-19 and related challenges to completing clinical trials: Lessons from the PATRIOT and STEP-UP randomized prevention trials	1
Bringing data monitoring committee charters into the sunlight	1
Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1	1
Bayesian design and analysis of two-arm cluster randomised trials using assurance: Extension to binary outcomes and comparison of Markov chain Monte Carlo and Integrated Nested Laplace Approximations	1
Ethical considerations for sharing aggregate results from pragmatic clinical trials	1
A comparison of alternative ranking methods in two-stage clinical trials with multiple interventions: An application to the anxiolysis for laceration repair in children trial	1
Mild-to-moderate depressive symptoms impact on self-reported outcome measures in clinical trials for neurodegenerative diseases	1
17th Annual University of Pennsylvania Conference on statistical issues in clinical trials - Covariate adjustment in randomized clinical trials: New methods and applications (Morning panel discussion)	1
Scoping review of family caregiver roles in cancer clinical trial decision-making	1
A framework for sequential monitoring of individual N-of-1 trials and combining results across a series of sequentially monitored N-of-1 trials	1
Adjudication of cause of death in older adults: Learnings for death certification from the ASPirin in Reducing Events in the Elderly study	1
Response to Cleland and Anzar	1
Informative cluster size in cluster-randomised trials: A case study from the TRIGGER trial	1
Examining the bias-efficiency tradeoff from incorporation of nonconcurrent controls in platform trials: A simulation study example from the adaptive COVID-19 treatment trial	1
Feasibility of recruiting in prisons during a randomized controlled trial with people with serious mental illness	1
Optimizing accrual to a large-scale, clinically integrated randomized trial in anesthesiology: A 2-year analysis of recruitment	1
Experiences with recruitment and retention of adolescents and emerging adults in a weight loss intervention trial	1
Evaluating the impact of stratification on the power and cross-arm balance of randomized phase 2 clinical trials	1
Leveraging generative AI to transform statistical analysis plan authoring in clinical trials	1
Electronic patient-reported adverse event monitoring in academic early-phase clinical trials: A feasibility study	1
Successful completion of large, low-cost randomized cancer trials at an academic cancer center	1
Society for Clinical Trials Data Monitoring Committee initiative website: Closing the gap	1
Composite endpoints in COVID-19 randomized controlled trials: a systematic review	1
On the proposed use of a finite-population correction factor in clinical trials	1
Randomized controlled dose-escalation design to evaluate the safety of a novel pharmacological cardiopulmonary resuscitation strategy	1
2024 Peer Reviewers	1
Abstracts from the society for clinical trials 44th annual meeting (2023)	1
Corrigendum	1
Success of blinding a procedural intervention in a randomised controlled trial in preterm infants receiving respiratory support	1
Now is the time to fix the clinical research workforce crisis	1
Some drawbacks of variable-weight composite endpoints	1
A statistical evaluation of decision-making methods and the efficiency of Bayesian multi-arm multi-stage trials	1
From RAGs to riches: Utilizing large language models to write documents for clinical trials	1
15th Annual University of Pennsylvania conference on statistical issues in clinical trial/advances in time-to-event analyses in clinical trials (afternoon panel discussion)	1
Design of a clinical trial using generalized pairwise comparisons to test a less intensive treatment regimen	1
Cognitive screening considerations for psychosocial clinical trials in HIV, aging, and cognition	1
Electronic common technical document submission with analysis using R	1
Pragmatic guidance for embedding pragmatic clinical trials in health plans: Large simple trials aren’t so simple	1
Proceedings of the University of Pennsylvania 15th annual conference on statistical issues in clinical trials: Advances in time-to-event analyses in clinical trials—challenges and opportunities	1
Confidence interval estimation for the win probability in cluster randomized trials with hierarchical composite endpoints using win fractions	1
A review of patient recruitment in randomised controlled trials of preoperative exercise	1
A note on rank-preserving structural failure time models to account for crossover	1
Statistical properties of items and summary scores from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE ®	1
Automated data collection from an electronic medical record for a prospective real-world study in patients with retinal disease (VOYAGER)	1
On flexible covariate adjustment under covariate-constrained randomization	1
Use of comprehensive recruitment strategies in the glycemia reduction approaches in diabetes: A comparative effectiveness study (GRADE) multi-center clinical trial	1
Pragmatic monitoring of emerging efficacy data in randomized controlled trials	1
The 3 + 3 design in dose-finding studies with small sample sizes: Pitfalls and possible remedies	1
Dose optimization for cancer treatments with considerations for late-onset toxicities	1
Mobilizing the clinical trial ecosystem to drive adoption of master protocols	1
Detecting irregularities in randomized controlled trials using machine learning	1
Ethics challenges in sharing data from pragmatic clinical trials	1
Incorporating data from multiple ongoing trials for Bayesian two-stage phase II single-arm studies	1
Charting the content of data monitoring committee charters for clinical trials	1
Using the Delphi process to determine the minimum clinically important effect size for the Balanced-2 randomised controlled trial	1
Evaluating whether the proportional odds models to analyse ordinal outcomes in COVID-19 clinical trials is providing clinically interpretable treatment effects: A systematic review	1