The Challenges Posed By Orphan & Rare Disease Trials

As chief editor for Clinical Leader, I speak to a lot of executives at companies working in the rare disease space. Clinical trials always present challenges to sponsor companies. Those challenges can be magnified greatly when dealing with rare and orphan diseases. The lack of a natural history of the disease is the first hurdle many companies have to navigate, but patient recruitment, data collection, and trial design can also present additional challenges that must be overcome.

In this eBook, we present the stories of four companies attempting to overcome these challenges and bring effective therapies to patients in need. Dr. Brian Schwartz, CMO for ArQule, discusses the challenges of an ultra-rare disease trial; Dr. Pushkal Garg, Chief Medical Officer at Alnylam Pharmaceuticals, discusses new approaches to patient-centric drug development; Mallory Factor, chairman of IntraBio discusses what government can do to help promote orphan disease research, and a panel of experts discuss what progress is being made to bring treatments to rare disease patients.