Assessing noninferiority: Evaluating efficacy of a new treatment without complete data.
Pharm Stat
; 18(5): 546-554, 2019 10.
Article
em En
| MEDLINE
| ID: mdl-30977258
ABSTRACT
The FDA released the final guidance on noninferiority trials in November 2016. In noninferiority trials, validity of the assessment of the efficacy of the test treatment depends on the control treatment's efficacy. Therefore, it is critically important that there be a reliable estimate of the control treatment effect-which is generally obtained from historical trials, and often assumed to hold in the current setting (the assay constancy assumption). Validating the constancy assumption requires clinical data, which are typically lacking. The guidance acknowledges that "lack of constancy can occur for many reasons." We clarify the objectives of noninferiority trials. We conclude that correction for bias, rather than assay constancy, is critical to conducting valid noninferiority trials. We propose that assay constancy not be assumed and discounting or thresholds be used to address concern about loss of historical efficacy. Examples are provided for illustration.
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Base de dados:
MEDLINE
Assunto principal:
Projetos de Pesquisa
/
Estudos de Equivalência como Asunto
Idioma:
En
Ano de publicação:
2019
Tipo de documento:
Article