The DOI matches its Crossref record; 28 of the 29 references it cites were found in a registry or an index.
Methodology of superiority vs. equivalence trials and non-inferiority trials
Abstract
The randomized clinical trial (RCT) is generally accepted as the best method of comparing effects of therapies. Most often the aim of an RCT is to show that a new therapy is superior to an established therapy or placebo, i.e. they are planned and performed as superiority trials. Sometimes the aim of an RCT is just to show that a new therapy is not superior but equivalent to or not inferior to an established therapy, i.e. they are planned and performed as equivalence trials or non-inferiority trials. Since the types of trials have different aims, they differ significantly in various methodological aspects. The awareness of the methodological differences is generally quite limited. This paper reviews the methodology of these types of trials with special reference to differences in respect to planning, performance, analysis and reporting of the trial. In this context the relevant basal statistical concepts are reviewed. Some of the important points are illustrated by examples.
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“All but one of the trials were powered to test superiority, not equivalence or non-inferiority41, so it would be premature to construe our findings as proof of equivalent effects.”
From The effectiveness of virtual reality based interventions for symptoms of anxiety and depression: A meta-analysis (2018) · cites this paper as [41]
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Cites
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Machine learning for medical imaging: methodological failures and recommendations for the future
Varoquaux, G. P., & Cheplygina, V. (2022) · npj Digital Medicine · doi:10.1038/s41746-022-00592-y · Cited by 815
Sample size calculations: basic principles and common pitfalls
Noordzij, M., Tripepi, G. L., Dekker, F. W., Zoccali, C., Tanck, M. W. T., & Jager, K. J. (2010) · Nephrology Dialysis Transplantation · doi:10.1093/ndt/gfp732 · Cited by 440
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Intention to treat and per protocol analysis in clinical trials
Tripepi, G., Chesnaye, N. C., Dekker, F. W., Zoccali, C., & Jager, K. J. (2020) · Nephrology · doi:10.1111/nep.13709 · Cited by 284
Metadata
- Authors
- Christensen, E.
- Affiliations
- Bispebjerg Hospital (DK)
- Corresponding author
- Christensen, Erik
- ORCID
- Christensen, Erik
- Venue
- Journal of Hepatology
- Publisher
- Elsevier BV
- Published
- 2007-05
- Volume
- 46
- Issue
- 5
- Pages
- 947-954
- ISSN
- 0168-8278 (ISSN-L)
- OpenAlex
- W2018758420
- Cited by
- 205
- References
- 29
- Citations by year
- 2026: 9 · 2025: 9 · 2024: 16 · 2023: 19 · 2022: 12 · 2021: 6 · 2020: 14 · 2019: 13 · 2018: 14 · 2017: 15 · 2016: 8 · 2015: 6 · 2014: 12 · 2013: 11 · 2012: 5
- FWCI
- 8.08
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Hepatology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Social Sciences › Decision Sciences › Statistics, Probability and Uncertainty › Meta-analysis and systematic reviews
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
- Keywords
- superiority trials, equivalence trials, randomized controlled trials, trial design, statistical analysis, trial reporting, statistical concepts, treatment comparison
- MeSH terms
- Animals, Clinical Protocols, Clinical Trials as Topic, Data Interpretation, Statistical, Humans, Patient Compliance, Research Design, Therapeutic Equivalency, Confidence Intervals, Randomized Controlled Trials as Topic, Sample Size, Risk Assessment
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