Determination of the optimal sample size for a clinical trial accounting for the population size.

ABSTRACT: The problem of choosing a sample size for a clinical trial is a very common one. In some settings, such as rare diseases or other small populations, the large sample sizes usually associated with the standard frequentist approach may be infeasible, suggesting that the sample size chosen should reflect the size of the population under consideration. Incorporation of the population size is possible in a decision-theoretic approach either explicitly by assuming that the population size is fixed and known, or implicitly through geometric discounting of the gain from future patients reflecting the expected population size. This paper develops such approaches. Building on previous work, an asymptotic expression is derived for the sample size for single and two-arm clinical trials in the general

SUBMITTER: Stallard N 

PROVIDER: S-EPMC5516263 | biostudies-literature | 2017 Jul

REPOSITORIES: biostudies-literature