Clinical Research: Phase 1 - Phase 4

Rare Disease Trials: The Beginning, the Middle and the End

Speaker: Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research

This session includes a regulatory overview of rare diseases and orphan drug status as well as practical considerations for varying stages of the development process. Viewers will learn more about conducting successful Natural History studies and the presenters will review how to avoid common challenges through lessons learned.

Additionally, there will be a discussion of best practices for running a program of studies in parallel. Helpful hints for preparing for the final regulatory submission will also be provided (including key factors for when studies are ongoing at the time of the submission).