Accounting for the rarity of the disease when designing clinical trials with a focus on pediatric cancers.

ABSTRACT:

Background

Clinical trials are challenging in rare diseases like pediatric cancers, where the accrual is limited. In these trials, inference assumptions are the same as in common diseases, that is the sample comes from a quasi-infinite population. This leads to overestimating the variance of the mean treatment effect. The finite-population correction factor correcting this bias is often used in surveys, but not in clinical trials. With few assumptions, the use of this correction factor can improve trials efficiency, showing that the power of those trials is sometimes higher than it appears.

Methods

First, a simulation study assesses the standard error of the mean treatment effect and coverage of the 95% confidence interval with and without the correction. Second, the analyti

SUBMITTER: Mauguen A 

PROVIDER: S-EPMC9232871 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature