Sample size calculation for recurrent event data with additive rates models.
Pharm Stat
; 21(1): 89-102, 2022 01.
Article
em En
| MEDLINE
| ID: mdl-34309179
ABSTRACT
This paper discusses the design of clinical trials where the primary endpoint is a recurrent event with the focus on the sample size calculation. For the problem, a few methods have been proposed but most of them assume a multiplicative treatment effect on the rate or mean number of recurrent events. In practice, sometimes the additive treatment effect may be preferred or more appealing because of its intuitive clinical meaning and straightforward interpretation compared to a multiplicative relationship. In this paper, new methods are presented and investigated for the sample size calculation based on the additive rates model for superiority, non-inferiority, and equivalence trials. They allow for flexible baseline rate function, staggered entry, random dropout, and overdispersion in event numbers, and simulation studies show that the proposed methods perform well in a variety of settings. We also illustrate how to use the proposed methods to design a clinical trial based on real data.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Modelos Estatísticos
Tipo de estudo:
Clinical_trials
/
Risk_factors_studies
Limite:
Humans
Idioma:
En
Revista:
Pharm Stat
Assunto da revista:
FARMACOLOGIA
Ano de publicação:
2022
Tipo de documento:
Article
País de afiliação:
Estados Unidos