RESUMO
The most common Bayesian methods for sample size determination (SSD) are reviewed in the non-sequential context of a confirmatory phase III trial in drug development. After recalling the regulatory viewpoint on SSD, we discuss the relevance of the various priors applied to the planning of clinical trials. We then investigate whether these Bayesian methods could compete with the usual frequentist approach to SSD and be considered as acceptable from a regulatory viewpoint.
Assuntos
Teorema de Bayes , Ensaios Clínicos Fase III como Assunto/legislação & jurisprudência , Ensaios Clínicos Fase III como Assunto/métodos , Tamanho da Amostra , Humanos , Projetos de Pesquisa/legislação & jurisprudênciaRESUMO
Recently, two CPMP Points to Consider, one on adjustment for baseline covariates and the other on multiplicity issues in clinical trials, have included recommendations on the use of subgroup analysis for regulatory purposes. However, despite their regular use and regulatory attention, the validity and nature of subgroup analyses are still frequently questioned. This article provides guidance on when subgroup analyses can be done, when they should be done, and their interpretation. The validity of common regulatory claims based on subgroup analyses is then discussed.