Project R-12748

Innovative Statistical Methodologies to Improve Rare Diseases Clinical Trials in Limited Populations (Research)

Rare diseases in limited populations are challenging for empirical research, whether of an epidemiological or clinical trial type. Specifically for clinical trials, there may be lack of power to demonstrate a treatment effect. This project aims to explore various statistical routes to maximize the amount of information that can be extracted from such trials, including the use of prior information, the use of longitudinal data, and appropriate handling of incomplete data.The use of generalized pairwise comparisons will be explored.

01 December 2021 - 31 August 2024