ReviewJournal of the National Cancer Institute2019
A Review of Perspectives on the Use of Randomization in Phase II Oncology Trials.
Review in Journal of the National Cancer Institute, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
28 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Bayesian Approaches for Confirmatory Trials in Rare Diseases: Opportunities and Challenges.International journal of environmental research and public health · 2021Pooled it
- Efficacy outcomes in phase 2 and phase 3 randomized controlled trials in rheumatology.Nature medicine · 2020Pooled it
- Bayesian sample size determination in basket trials borrowing information between subsets.Biostatistics (Oxford, England) · 2023Trial
- Sources of bias for single-arm phase II cancer clinical trials.Annals of translational medicine · 2022Trial
- A stochastically curtailed two-arm randomised phase II trial design for binary outcomes.Pharmaceutical statistics · 2021Trial
- Information borrowing in phase II randomized dose-ranging clinical trials in oncology.BMC medical research methodology · 2026Article
- A Bayesian Treatment Selection Design for Phase II Randomised Cancer Clinical Trials.Statistics in medicine · 2026Article
- Sieving for gold: external comparator studies to prioritize drugs for late-stage clinical trial.Journal of comparative effectiveness research · 2026Review
- An Improved Bayesian Pick-the-Winner (IBPW) Design for Randomized Phase II Clinical Trials.Statistics in medicine · 2026Article
- Review
- Clinical study design strategies to mitigate confounding effects of time-dependent clearance on dose optimization of therapeutic antibodies.CPT: pharmacometrics & systems pharmacology · 2025Article
- Eftilagimod Alpha (a Soluble LAG-3 Protein) Combined With Pembrolizumab in Second-Line Metastatic NSCLC Refractory to Anti-Programmed Cell Death Protein 1/Programmed Death-Ligand 1-Based Therapy: Final Results from a Phase 2 Study.JTO clinical and research reports · 2024Article
- Completion Rate and Positive Results Reporting Among Immunotherapy Trials in Breast Cancer, 2004-2023.JAMA network open · 2024Article
- Selection Bias Risk in Randomized Controlled Trials Rated as Low Bias Using Risk of Bias, Version 2 (RoB2) Tool.Cureus · 2024Article
- Comparison of Pocock and Simon's covariate-adaptive randomization procedures in clinical trials.BMC medical research methodology · 2024Article
- BASIC: A Bayesian adaptive synthetic-control design for phase II clinical trials.Clinical trials (London, England) · 2023Article
- Can a propensity score matching method be applied to assessing efficacy from single-arm proof-of-concept trials in oncology?CPT: pharmacometrics & systems pharmacology · 2023Article
- The Trial within Cohorts (TwiCs) study design in oncology: experience and methodological reflections.BMC medical research methodology · 2023Article
- A single-arm study design with non-inferiority and superiority time-to-event endpoints: a tool for proof-of-concept and de-intensification strategies in breast cancer.Frontiers in oncology · 2023Article
- Is it feasible and ethical to randomize patients between surgery and non-surgical treatments for gastrointestinal cancers?Frontiers in oncology · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Historically, phase II oncology trials assessed a treatment's efficacy by examining its tumor response rate in a single-arm trial. Then, approximately 25 years ago, certain statistical and pharmacological considerations ignited a debate around whether randomized designs should be used instead. Here, based on an extensive literature review, we review the arguments on either side of this debate. In particular, we describe the numerous factors that relate to the reliance of single-arm trials on historical control data and detail the trial scenarios in which there was general agreement on preferential utilization of single-arm or randomized design frameworks, such as the use of single-arm designs when investigating treatments for rare cancers. We then summarize the latest figures on phase II oncology trial design, contrasting current design choices against historical recommendations on best practice. Ultimately, we find several ways in which the design of recently completed phase II trials does not appear to align with said recommendations. For example, despite advice to the contrary, only 66.2% of the assessed trials that employed progression-free survival as a primary or coprimary outcome used a randomized comparative design. In addition, we identify that just 28.2% of the considered randomized comparative trials came to a positive conclusion as opposed to 72.7% of the single-arm trials. We conclude by describing a selection of important issues influencing contemporary design, framing this discourse in light of current trends in phase II, such as the increased use of biomarkers and recent interest in novel adaptive designs.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.