Operating characteristics of unequal allocation ratios in platform trials with the staggered addition of drugs using binary endpoints.

ABSTRACT:

Background

One recommendation for the allocation ratio between multiple drugs and a shared placebo control group in platform trials (PTs) is to use a k :1 allocation ratio for the placebo group relative to the drug group, where k is the number of drug groups with ongoing patient enrollment during the trials. However, the practical utility of such unequal allocation ratios in PTs lacks adequate study.

Methods

We compared the performances of equal and unequal allocation ratios through simulations to imitate practical PTs using only concurrent controls and binary endpoints for hospitalized patients with infectious diseases. The operating characteristics, including the type I error rate, power of hypothesis testing, and total sample size, were evaluated.

Results

In PTs, u

SUBMITTER: Shimizu Y 

PROVIDER: S-EPMC11889599 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature