Open AccessAccounting for the rarity of the disease when designing clinical trials with a focus on pediatric cancers
Author(s) -
Audrey Mauguen
Publication year - 2022
Publication title -
clinical trials
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.559
H-Index - 63
eISSN - 1740-7753
pISSN - 1740-7745
DOI - 10.1177/17407745221080728
Subject(s) - sample size determination , statistics , confidence interval , population , type i and type ii errors , standard error , clinical trial , sample (material) , inference , statistical power , medicine , econometrics , mathematics , computer science , chemistry , environmental health , chromatography , artificial intelligence
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.