Article,
Re-formulating non-inferiority trials as superiority trials: The case of binary outcomes.
Biometrical journal. Biometrische Zeitschrift, 51 (1): 185-92 (February 2009)4771<m:linebreak></m:linebreak>CP: Copyright © 2009 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim; PN: 0323-3847;.
DOI: 10.1002/bimj.200810499
Abstract
Non-inferiority trials are conducted for a variety of reasons including to show that a new treatment has a negligible reduction in efficacy or safety when compared to the current standard treatment, or a more complex setting of showing that a new treatment has a negligible reduction in efficacy when compared to the current standard yet is superior in terms of other treatment characteristics. The latter reason for conducting a non-inferiority trial presents the challenge of deciding on a balance between a suitable reduction in efficacy, known as the non-inferiority margin, in return for a gain in other important treatment characteristics/findings. It would be ideal to alleviate the dilemma on the choice of margin in this setting by reverting to a traditional superiority trial design where a single p -value for superiority of both the most important endpoint (efficacy) and the most important finding (treatment characteristic) is provided. We discuss how this can be done using the information-preserving composite endpoint (IPCE) approach and consider binary outcome cases in which the combination of efficacy and treatment characteristics, but not one itself, paints a clear picture that the novel treatment is superior to the active control.
Tags
- biometry
- biometry:methods
- controlledclinicaltrialsastopic
- controlledclinicaltrialsastopic:methods
- datainterpretation
- endpointdetermination
- endpointdetermination:methods
- epidemiologicresearchdesign
- humans
- outcomeassessment(healthcare)
- outcomeassessment(healthcare):methods
- statistical
