ReviewJCO precision oncology2019
Model-Assisted Designs for Early-Phase Clinical Trials: Simplicity Meets Superiority.
Review in JCO precision oncology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
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
36 citing papers in PubMed.
- Trial
- Strategic roadmap for delivery of clinical trials in rare childhood central nervous system (CNS) tumours: a multi-stakeholder consensus.British journal of cancer · 2026Article
- An evaluation of designs for Phase I/IIa dose-finding studies in Tuberculosis.Statistical methods in medical research · 2026Article
- Patient Retreat in Dose Escalation for Phase I Clinical Trials With Rare Diseases.Statistics in medicine · 2026Article
- FDA-AACR Strategies for Optimizing Dosages for Oncology Drug Products: Early-Phase Trials Using Innovative Trial Designs and Biomarkers.Clinical cancer research : an official journal of the American Association for Cancer Research · 2025Review
- Posterior Predictive Design for Phase I Clinical Trials.Journal of the American Statistical Association · 2025Article
- SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols.EClinicalMedicine · 2025Article
- CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trials.EClinicalMedicine · 2025Article
- Targeted-Agent Continual Reassessment Method: A Novel Bayesian Enrichment Design for Phase I Trials of Molecularly Targeted Therapies.JCO precision oncology · 2024Article
- Determining doses for backfill cohorts based on patient-reported outcome.BMC medical research methodology · 2024Article
- Robustness Assessment of Oncology Dose-Finding Trials Using the Modified Fragility Index.Cancers · 2024Article
- Toxicity Adaptive Lists Design: A Practical Design for Phase I Drug Combination Trials in Oncology.JCO precision oncology · 2024Article
- MC-Keyboard: A Practical Phase I Trial Design for Targeted Therapies and Immunotherapies Integrating Multiple-Grade Toxicities.Journal of immunotherapy and precision oncology · 2024Article
- Statistical and practical considerations in planning and conduct of dose-optimization trials.Clinical trials (London, England) · 2024Review
- Backfilling Patients in Phase I Dose-Escalation Trials Using Bayesian Optimal Interval Design (BOIN).Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Article
- Phase I clinical trial designs in oncology: A systematic literature review from 2020 to 2022.Journal of clinical and translational science · 2024Article
- Novel Clinical Trial Designs with Dose Optimization to Improve Long-term Outcomes.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023Review
- Enhancing reporting quality and impact of early phase dose-finding clinical trials: CONSORT Dose-finding Extension (CONSORT-DEFINE) guidance.BMJ (Clinical research ed.) · 2023Article
- Improving the efficiency of clinical trials in multiple sclerosis.Multiple sclerosis (Houndmills, Basingstoke, England) · 2023Review
- Dose optimization during drug development: whether and when to optimize.Journal of the National Cancer Institute · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Drug development enterprise is struggling because of prohibitively high costs and slow progress. There is urgent need for adoption of novel adaptive designs to improve the efficiency and success of clinical trials. A major barrier is that many conventional designs are inadequate for modern drug development, yet most novel adaptive designs are difficult to understand, require complicated statistical modeling, demand complex computation, and need expensive infrastructure for implementation. The objective of this article is to introduce and review a class of novel adaptive designs, known as model-assisted designs, to remove this barrier and increase the use of novel adaptive designs. Model-assisted designs enjoy superior performance comparable to more complicated, model-based adaptive designs, but their decision rule can be pretabulated and included in the protocol-thus implemented as simply as the conventional designs. We review state-of-the-art model-assisted designs for phase I clinical trials for single-agent, drug-combination and late-onset toxicity scenarios. We also briefly introduce model-assisted designs for phase II trials to handle binary, coprimary endpoints and delayed response. Freely available user-friendly software and trial examples (trialdesign.org) facilitate the adoption of model-assisted designs.
Identifiers
What OpenQuestion holds
Registered trials
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.