TL04: Challenges Facing Observational Studies Based on Rare Disease Registry Data
*Mohammad Bsharat, Vertex Pharmaceuticals 

Keywords: registries, rare diseases, big data

The use of patient registries is a very useful tool for a number of purposes. Registries embedded observational studies differ from clinical trials in a number of substantive ways; in evidence development, design, operations, and evaluation. This roundtable will discuss some of the most important issues facing study team working on rare disease observational studies that are based on registries, and the rules and guidelines that may differentiate such observational studies from regular clinical trials such as guidelines regarding ethical committee review, submission of data to governmental websites, role of steering or scientific committees, …etc.

Should and when observational studies be reviewed by IRBs? What is the role of the scientific or the steering committee of an observational study, and how and when study team should ask such a scientific or a steering committee for advice?

Some real life examples on the different challenges facing a statistician while working with EU registries will also be discussed.