ArticleJournal of the National Cancer Institute2020
TOP: Time-to-Event Bayesian Optimal Phase II Trial Design for Cancer Immunotherapy.
Article in Journal of the National Cancer Institute, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed.
- Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases.Annals of translational medicine · 2022Trial
- Bayesian Learning to Reduce Cardiac Risk of Patients With Locally Advanced Non-Small Cell Lung Cancer Based on Personalized Radiation Therapy Prescription.International journal of radiation oncology, biology, physics · 2026Article
- Estimands for Early-Phase Dose Optimization Trials in Oncology.Biometrical journal. Biometrische Zeitschrift · 2025Article
- Challenging go/no-go decision scenarios and design recommendations in phase II oncology trials.Journal of the National Cancer Center · 2025Article
- The Win Ratio Approach in Bayesian Monitoring for Two-Arm Phase II Clinical Trial Designs With Multiple Time-To-Event Endpoints.Statistics in medicine · 2024Article
- Bayesian sequential monitoring strategies for trials of digestive cancer therapeutics.BMC medical research methodology · 2024Article
- A generalized calibrated Bayesian hierarchical modeling approach to basket trials with multiple endpoints.Biometrical journal. Biometrische Zeitschrift · 2024Article
- Using short-term endpoints to improve interim decision making and trial duration in two-stage phase II trials with nested binary endpoints.Statistical methods in medical research · 2023Article
- Comparison Between Simultaneous and Sequential Utilization of Safety and Efficacy for Optimal Dose Determination in Bayesian Model-Assisted Designs.Therapeutic innovation & regulatory science · 2023Article
- Bayesian single-arm phase II trial designs with time-to-event endpoints.Pharmaceutical statistics · 2021Article
- Unified exact design with early stopping rules for single arm clinical trials with multiple endpoints.Statistical methods in medical research · 2021Article
- Response adaptive designs for Phase II trials with binary endpoint based on context-dependent information measures.Computational statistics & data analysis · 2021Article
- Advancing Effective Clinical Trial Designs for Myelofibrosis.Hematology/oncology clinics of North America · 2021Review
- BAGS: A Bayesian Adaptive Group Sequential Trial Design With Subgroup-Specific Survival Comparisons.Journal of the American Statistical Association · 2021Article
- BOIN12: Bayesian Optimal Interval Phase I/II Trial Design for Utility-Based Dose Finding in Immunotherapy and Targeted Therapies.JCO precision oncology · 2020Article
- Model-Assisted Designs for Early-Phase Clinical Trials: Simplicity Meets Superiority.JCO precision oncology · 2019Review
Corrections and comments
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Authors and funding
3 authors.
Funding
Abstract
backgroundImmunotherapies have revolutionized cancer treatment. Unlike chemotherapies, immune agents often take longer to show benefit, and the complex and unique mechanism of action of these agents renders the use of multiple endpoints more appropriate in some trials. These new features of immunotherapy make conventional phase II trial designs, which assume a single binary endpoint that is quickly ascertainable, inefficient and dysfunctional.
methodsWe propose a flexible and efficient time-to-event Bayesian optimal phase II (TOP) design. The TOP design is efficient in that it allows real-time "go/no-go" interim decision making in the presence of late-onset responses by using all available data and maximizes statistical power for detecting effective treatments. TOP is flexible in the number of interim looks and capable of handling simple and complicated endpoints under a unified framework. We conduct simulation studies to evaluate the operating characteristics of the TOP design.
resultsIn the considered trial settings, compared to some existing Bayesian designs, the TOP design shortens the trial duration by 4-10 months and improves the power to detect effective treatment up to 90%, with well-controlled type I errors.
conclusionsThe TOP design is transparent and easy to implement, as its decision rules can be tabulated and included in the protocol prior to the conduct of the trial. The TOP design provides a flexible, efficient, and easy-to-implement method to accelerate and improve the development of immunotherapies.
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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.