Statistical Considerations in Bayesian Approaches to Clinical Trials: The Zoster Eye Disease Study

Music City Center 

Clinical trials often rely on frequentist statistical methods for design, monitoring, and analysis, emphasizing type I and II errors, p-values, and confidence intervals. While this approach is widely accepted in research and regulatory frameworks, Bayesian methods provide an alternative that incorporates prior knowledge and expert judgment and more importantly characterizes the entire posterior distribution of treatment effects. By basing decisions on a "minimal clinically worthwhile benefit," Bayesian approaches enhance decision-making in clinical practice based on trial data.

We will present and discuss frequentist and Bayesian analyses in the Zoster Eye Disease Study, a multicenter, double-masked, placebo-controlled randomized trial conducted in 95 sites from 2017 to 2024. A total of 527 participants were randomized to receive 12 months of daily valacyclovir or placebo, followed for an additional 6 months, stratified by age at onset (<60 vs ≥60 years) and disease duration (<6 vs ≥6 months). Bayesian analysis provided the probability of a clinically meaningful effect and the probability of various absolute event rate differences, making results more intuitive for clinicians.

Bayesian analyses

Survival analysis

Clinical Trials 

Lifetime Data Science Section