Dynamic use of historical controls in clinical trials for rare disease research: A re-evaluation of the MILES trial.

ABSTRACT:

Background

Randomized controlled trials offer the best design for eliminating bias in estimating treatment effects but can be slow and costly in rare disease research. Additionally, an equal randomization approach may not be optimal in studies in which prior evidence of superiority of one or more treatments exist. Supplementing prospectively enrolled, concurrent controls with historical controls can reduce recruitment requirements and provide patients a higher likelihood of enrolling in a new and possibly superior treatment arm. Appropriate methods need to be employed to ensure comparability of concurrent and historical controls to minimize bias and variability in the treatment effect estimates and reduce the chances of drawing incorrect conclusions regarding treatment benefit.

SUBMITTER: Harun N 

PROVIDER: S-EPMC10257755 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature