Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles.

ABSTRACT:

Background

Design and analysis of clinical trials for rare and ultra-rare disease pose unique challenges to the practitioners. Meeting conventional power requirements is infeasible for diseases where sample sizes are inherently very small. Moreover, rare disease populations are generally heterogeneous and widely dispersed, which complicates study enrollment and design. Leveraging all available information in rare and ultra-rare disease trials can improve both drug development and informed decision-making processes.

Main text

Bayesian statistics provides a formal framework for combining all relevant information at all stages of the clinical trial, including trial design, execution, and analysis. This manuscript provides an overview of different Bayesian methods applicable to

SUBMITTER: Kidwell KM 

PROVIDER: S-EPMC9077995 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature