Sample size considerations for single-arm clinical trials with time-to-event endpoint using the gamma distribution.

ABSTRACT:

Background

Time-to-event (TTE) endpoints are evaluated as the primary endpoint in single-arm clinical trials; however, limited options are available in statistical software for sample size calculation. In single-arm trials with TTE endpoints, the non-parametric log-rank test is commonly used. Parametric options for single-arm design assume survival times follow exponential distribution or Weibull distribution.

Methods

The exponential- or Weibull-distributed survival time assumption does not always reflect hazard pattern of real-life diseases. We therefore propose gamma distribution as an alternative parametric option for designing single-arm studies with TTE endpoints. We outline a sample size calculation approach using gamma distribution with a known shape parameter and exp

SUBMITTER: Dai J 

PROVIDER: S-EPMC11369365 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature