Presenter: Pirouz Shamszad, MD - Medical Director, Medpace
Presentation 1 of 3 in Medpace Discovery Series - Rare Disease - Connecting the Dots
Key takeaways from this presentation are:
Planning and executing a study for a Rare Disease is challenging. Major issues exist involving protocol design, patient recruitment and retention, and global regulatory design.
•Best practices to accelerate these novel compounds to commercialization given difficult rare disease and orphan drug studies
•Special pediatric population issues with Rare Disease studies
•Clinical Trial Design Considerations