Bayesian Disease Progression Modeling in Clinical Trials; Scott Berry, PhD, Berry Consultants

Abstract:

Frequently primary analyses in clinical trials of progressive diseases use change-from-baseline type analyses, such as the MMRM.  These analyses of the absolute changes at different time points ignores that the disease is progressive, and the mechanism of the intervention is to slow progression.

In this talk I’ll present several example in which a Bayesian primary analysis is created that analyzes whether an intervention slows the rate of progression of a disease, rather than the absolute change.  The analysis can create a great deal of improved precision — as well as improved clinical interpretations.  Multiple examples will be discussed, including dominantly inherited Alzheimer’s, GNE Myopathy, and Batten’s Disease.