ArticleStatistics in medicine2015
Building efficient comparative effectiveness trials through adaptive designs, utility functions, and accrual rate optimization: finding the sweet spot.
Article in Statistics in medicine, 2015. 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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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.
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Who cites it
16 citing papers in PubMed, 25 citations in OpenAlex.
- Trial
- Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLS): Bayesian Adaptive Comparative Effectiveness Randomized Trial.JAMA neurology · 2021Trial
- Trial
- A case study in teaching and learning the design, conduction, and analysis of Bayesian adaptive trials through an application.Journal of clinical and translational science · 2026Article
- Conducting a bayesian multi-armed trial with response adaptive randomization for comparative effectiveness of medications for CSPN.Contemporary clinical trials communications · 2023Article
- The value of a two-armed Bayesian response adaptive randomization trial.Journal of biopharmaceutical statistics · 2023Article
- Comparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants.Statistics in biopharmaceutical research · 2023Article
- Designing and analyzing clinical trials for personalized medicine via Bayesian models.Pharmaceutical statistics · 2021Article
- Bayesian accrual modeling and prediction in multicenter clinical trials with varying center activation times.Pharmaceutical statistics · 2020Article
- A new model for clinical trials to address the COVID-19 emergency.Breathe (Sheffield, England) · 2020Article
- Using Adaptive Designs to Avoid Selecting the Wrong Arms in Multiarm Comparative Effectiveness Trials.Statistics in biopharmaceutical research · 2019Article
- Using an onset-anchored Bayesian hierarchical model to improve predictions for amyotrophic lateral sclerosis disease progression.BMC medical research methodology · 2018Article
- A novel evaluation of optimality for randomized controlled trials.Journal of biopharmaceutical statistics · 2017Article
- A post hoc evaluation of a sample size re-estimation in the Secondary Prevention of Small Subcortical Strokes study.Clinical trials (London, England) · 2016Article
- Article
- Commentary on Hey and Kimmelman.Clinical trials (London, England) · 2015Article
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
The time is right for the use of Bayesian Adaptive Designs (BAD) in comparative effectiveness trials. For example, Patient Centered Outcomes Research Institute has joined the Food and Drug Administration and National Intitutes of Health in adopting policies/guidelines encouraging their use. There are multiple aspects to BAD that need to be considered when designing a comparative effectiveness design. First, the adaptation rules can determine the expected size of the trial. Second, a utility function can be used to combine extremely important co-endpoints (e.g., efficacy and tolerability) and is a valuable tool for incorporating clinical expertise and potentially patient preference. Third, accrual rate is also very, very important. Specifically, there is a juxtaposition related to accrual and BAD. If accrual rate is too fast we never gain efficient information for adapting. If accrual rate is too slow we never finish the clinical trial. We propose methodology for finding the 'sweet spot' for BAD that addresses these as design parameters. We demonstrate the methodology on a comparative effectiveness BAD of pharmaceutical agents in cryptogenic sensory polyneuropathy. The study has five arms with two endpoints that are combined with a utility function. The accrual rate is assumed to stem from multiple sites. We perform simulations from which the composite accrual rates across sites result in various piecewise Poisson distributions as parameter inputs. We balance both average number of patients needed and average length of time to finish the study.
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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.