ArticleBMC medical research methodology2019
Evidence-based sizing of non-inferiority trials using decision models.
Article in BMC medical research methodology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.
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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
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Who cites it
3 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Variability of quality-of-life measurements and reporting in randomised controlled trials of pancreatic cancer: a systematic review.BMJ open · 2024Pooled it
- Projected environmental and public health benefits of extended-interval dosing: an analysis of pembrolizumab use in a US national health system.The Lancet. Oncology · 2024Article
- Alternative Trastuzumab Dosing Schedules Are Associated With Reductions in Health Care Greenhouse Gas Emissions.JCO oncology practice · 2023Article
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
backgroundThere are significant challenges to the successful conduct of non-inferiority trials because they require large numbers to demonstrate that an alternative intervention is "not too much worse" than the standard. In this paper, we present a novel strategy for designing non-inferiority trials using an approach for determining the appropriate non-inferiority margin (δ), which explicitly balances the benefits of interventions in the two arms of the study (e.g. lower recurrence rate or better survival) with the burden of interventions (e.g. toxicity, pain), and early and late-term morbidity.
methodsWe use a decision analytic approach to simulate a trial using a fixed value for the trial outcome of interest (e.g. cancer incidence or recurrence) under the standard intervention (p
resultsUsing the European Polyp Surveillance non-inferiority trial as an example, our decision analytic approach suggests an appropriate non-inferiority margin, defined here as the difference between the two study arms in the 10-year risk of being diagnosed with colorectal cancer, of 0.42% rather than the 0.50% used to design the trial. The size of the non-inferiority margin was smaller for higher assumed burden of colonoscopies.
conclusionsThe example demonstrates that applying our proposed method appears feasible in real-world settings and offers the benefits of more explicit and rigorous quantification of the various considerations relevant for determining a non-inferiority margin and associated trial sample size.
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